Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
This panel will explore the critical role of CDMOs in modern biomanufacturing, covering topics from partner selection and relationship management to navigating regulatory challenges and optimizing for success in a rapidly changing environment
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at Cell and Gene Therapy International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at Cell and Gene Therapy International.
• Developing formulations that enhance the stability of biologics
• Include the use of excipients, lyophilization, and novel delivery systems
The Biophorum ATMP Visible Particles workstream has proposed a holistic, lifecycle approach to reduce and de-risk visible particulates in cell therapy (CT) drug formulations. This involves characterizing and detecting particulates in the manufacturing process and formulation, then improving material quality and process controls to minimize them.
CTs face unique challenges in particulate control and detection compared to other injectables, including difficult-to-inspect formulations and containers. CTs, with inherent cell-related particulates, complicate the detection of other particles. Terminal sterilizing filtration isn't applicable due to cell size and formulation needs. Small batch volumes, especially in autologous therapies, make rejecting units with particle defects critical. Regulatory guidance and health authority expectations are not aligned with the unique characteristics of cell and gene therapies, posing challenges for sponsors in meeting particulate specifications.
A Representative from Single Use Support
- Establishing clear communication channels and quality agreements that foster a shared commitment to quality.
- Implementing effective oversight and monitoring systems for CDMO operations.
- Navigating differences in quality cultures and ensuring alignment.
- Leveraging technology and data to enhance quality and compliance in external manufacturing.
- Addressing challenges related to investigations, deviations, and CAPAs across organizations.
- Building trust and transparency as foundational elements of a strong quality partnership.
Patient convenience and the need for self-administration are fueling the demand for prefilled syringes and autoinjectors for biologics, requiring specialized formulation and fill finish capabilities.
- Anticipating the needs on the work streams
- Process Understanding and Design
- Technology Transfer
- Dealing with changes and Comparability
- Frameworks for evaluating make vs. buy decisions in biopharma.
- Identifying core competencies and areas ripe for outsourcing.
- Balancing control and flexibility in manufacturing choices.
- Case studies illustrating successful outsourcing strategies.
- Review of the main streams and initial status
- Broad requirements for Tox and for First in Human Studies
- Cell banking
- Other Raw materials
- Drug Substance Process and Manufacture
- Minimum requirements
- Understanding your process :
- Impurities : identity, clearance, control
- First steps towards a control strategy
- Adventitious contamination and Viral Clearance Studies
- Drug formulation and Drug Product Processing
- Analytical package
- Release methods definition and development
- From method performance to method validation
- In Process Controls (else cover under process?)
- Batch data in the submission
- Product Characterisation and Reference standard
- Stability ( DS and DP)
- Forced degradation studies : necessity and importance
- Why is stability important ?
- Different type of stability studies and typical package for PhI
- Shelf life assignment
This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.
Understanding the Tech Transfer Process
Gain a clear roadmap for transitioning projects from laboratory research to large-scale clinical manufacturing.
Identify common challenges and best practices to ensure a smooth and efficient transfer.
Optimizing Scale-Up and Clinical Trial Readiness
Learn strategies for scaling up manufacturing processes efficiently.
Discover methods for ensuring reproducibility and overcoming obstacles in clinical trial preparation.
Navigating Regulatory Compliance and Tech Transfer Challenges
Explore key regulatory considerations in tech transfer.
Understand compliance challenges and how to effectively navigate regulatory requirements.
Enhancing Efficiency and Speed in Tech Transfer
Modernize processes with innovative approaches and new technologies.
Implement strategies to reduce development-to-manufacturing time and accelerate the tech transfer timeline.
Leveraging Automation and Cloud-Based Solutions
Explore the integration of automated tech transfer systems across platforms.
Learn how cloud-based solutions can enhance data transfer and collaboration.
Expert Insights and Practical Advice
Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.
Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.
• Advances in transposon technology for targeted integration and enhanced stability
• Stability improvements through transposon innovations: what works and what doesn’t
- What to start early in the process?
- How early is too early to consider strategy for scaling up?
- How to define the right commercial scale early in development
- Best practices for locking in scalable processes from the start
- Designing therapies with long-term success in mind—beyond Phase 1
- Key considerations for commercial scale up, reimbursement, raw material availability, and regulatory approvals
- Pathways to developing safe, reimbursable therapies with broad patient access
• Addressing supply chain disruptions: strategies for procurement, digitalization, and raw material management
• Sustainability challenges and cost-saving innovations for large-scale media production
- How to ensure safety and compliance while accelerating clinical and manufacturing timelines
- Common pitfalls and best practices
- Strategies to address changing regulatory requirements during rapid development
- Strategic approaches to transitioning from clinical development to manufacturing readiness
- Aligning speed with regulatory expectations for product quality and safety
- Key FDA regulations and guidelines relevant to CDMO partnerships (e.g., 21 CFR Parts 210 & 211).
- Understanding and implementing robust quality agreements.
- Navigating supplier qualification and audit processes.
- Strategies for maintaining compliance throughout the partnership lifecycle.
Round Table Session 1: Host Cell Proteins (HCPs): The Unseen Challenge
- Focus: Managing HCPs throughout the bioprocessing lifecycle.
- Key Discussion Points:
- HCP Characterization and Detection: Advancements in analytical methods for identifying and quantifying HCPs, including challenges with low-level detection and diverse HCP populations. Discuss the use of orthogonal methods.
- Impact of HCPs on Product Quality: Explore the mechanisms by which HCPs can affect product stability, efficacy, and immunogenicity. Focus on specific examples and case studies.
- HCP Removal Strategies: Evaluate current HCP removal technologies (chromatography, filtration) and discuss emerging approaches (e.g., novel resins, targeted removal). Consider the challenges of removing specific "high-risk" HCPs (lipases, proteases).
- HCP-Related Challenges in Different Product Modalities: Discuss specific HCP challenges associated with different types of biologics (e.g., antibodies, proteins, vaccines, cell therapies).
- Setting HCP Acceptance Criteria: How do we determine appropriate levels of HCPs, and what factors influence these decisions? Discuss the role of regulatory guidance.
- Future Directions in HCP Management: Explore innovative technologies and strategies for improved HCP control and monitoring. Discuss the role of AI/ML in predicting and managing HCP-related risks.
Round Table Session 2: Extractables and Leachables (E&L): Ensuring Patient Safety
- Focus: Understanding, controlling, and mitigating the risks associated with E&L.
- Key Discussion Points:
- E&L Sources and Identification: Discuss common sources of E&L in bioprocessing and packaging, including materials of construction, processing equipment, and packaging components. Explore advanced analytical techniques for identifying and characterizing E&L.
- Risk Assessment and Toxicological Evaluation: How do we assess the potential toxicity of identified E&L? Discuss the use of toxicological studies and risk-based approaches to determine acceptable levels.
- E&L Control Strategies: Explore strategies for minimizing E&L, including material selection, process optimization, and container closure system design. Discuss the importance of supplier qualification and change control.
- Analytical Method Development and Validation: Discuss the challenges of developing and validating robust analytical methods for E&L testing, especially for trace-level contaminants.
- Regulatory Requirements for E&L: Review current regulatory guidelines and expectations for E&L characterization and control. Discuss differences in global regulatory requirements.
- Emerging Trends in E&L Management: Explore new technologies and approaches for E&L analysis and control, including the use of predictive modeling and simulation.
Roundtable Session 3: Polysorbate Degradation: Protecting Product Stability
- Focus: Understanding the mechanisms of polysorbate degradation and developing strategies to mitigate it.
- Key Discussion Points:
- Mechanisms of Polysorbate Degradation: Discuss the various pathways of polysorbate degradation, including hydrolysis, oxidation, and enzymatic degradation (with a focus on lipase contamination).
- Factors Influencing Degradation: Explore the factors that can influence polysorbate degradation, such as pH, temperature, oxygen levels, and the presence of specific HCPs (especially lipases).
- Analytical Methods for Monitoring Degradation: Discuss analytical techniques for detecting and quantifying polysorbate degradation products.
- Strategies for Mitigating Degradation: Explore strategies for preventing or minimizing polysorbate degradation, including process optimization, excipient selection, and the use of inhibitors.
- Impact of Polysorbate Degradation on Product Quality: Discuss the potential consequences of polysorbate degradation on product stability, efficacy, and safety.
- Case Studies of Polysorbate Degradation: Share real-world examples of polysorbate degradation challenges and the solutions implemented.
- Future Directions in Polysorbate Management: Explore new approaches for stabilizing polysorbates and preventing degradation, including the use of novel excipients and formulation strategies. Discuss the potential of more sensitive analytical methods.
Round Table Session 1: Host Cell Proteins (HCPs): The Unseen Challenge
- Focus: Managing HCPs throughout the bioprocessing lifecycle.
- Key Discussion Points:
- HCP Characterization and Detection: Advancements in analytical methods for identifying and quantifying HCPs, including challenges with low-level detection and diverse HCP populations. Discuss the use of orthogonal methods.
- Impact of HCPs on Product Quality: Explore the mechanisms by which HCPs can affect product stability, efficacy, and immunogenicity. Focus on specific examples and case studies.
- HCP Removal Strategies: Evaluate current HCP removal technologies (chromatography, filtration) and discuss emerging approaches (e.g., novel resins, targeted removal). Consider the challenges of removing specific "high-risk" HCPs (lipases, proteases).
- HCP-Related Challenges in Different Product Modalities: Discuss specific HCP challenges associated with different types of biologics (e.g., antibodies, proteins, vaccines, cell therapies).
- Setting HCP Acceptance Criteria: How do we determine appropriate levels of HCPs, and what factors influence these decisions? Discuss the role of regulatory guidance.
- Future Directions in HCP Management: Explore innovative technologies and strategies for improved HCP control and monitoring. Discuss the role of AI/ML in predicting and managing HCP-related risks.
Round Table Session 2: Extractables and Leachables (E&L): Ensuring Patient Safety
- Focus: Understanding, controlling, and mitigating the risks associated with E&L.
- Key Discussion Points:
- E&L Sources and Identification: Discuss common sources of E&L in bioprocessing and packaging, including materials of construction, processing equipment, and packaging components. Explore advanced analytical techniques for identifying and characterizing E&L.
- Risk Assessment and Toxicological Evaluation: How do we assess the potential toxicity of identified E&L? Discuss the use of toxicological studies and risk-based approaches to determine acceptable levels.
- E&L Control Strategies: Explore strategies for minimizing E&L, including material selection, process optimization, and container closure system design. Discuss the importance of supplier qualification and change control.
- Analytical Method Development and Validation: Discuss the challenges of developing and validating robust analytical methods for E&L testing, especially for trace-level contaminants.
- Regulatory Requirements for E&L: Review current regulatory guidelines and expectations for E&L characterization and control. Discuss differences in global regulatory requirements.
- Emerging Trends in E&L Management: Explore new technologies and approaches for E&L analysis and control, including the use of predictive modeling and simulation.
Roundtable Session 3: Polysorbate Degradation: Protecting Product Stability
- Focus: Understanding the mechanisms of polysorbate degradation and developing strategies to mitigate it.
- Key Discussion Points:
- Mechanisms of Polysorbate Degradation: Discuss the various pathways of polysorbate degradation, including hydrolysis, oxidation, and enzymatic degradation (with a focus on lipase contamination).
- Factors Influencing Degradation: Explore the factors that can influence polysorbate degradation, such as pH, temperature, oxygen levels, and the presence of specific HCPs (especially lipases).
- Analytical Methods for Monitoring Degradation: Discuss analytical techniques for detecting and quantifying polysorbate degradation products.
- Strategies for Mitigating Degradation: Explore strategies for preventing or minimizing polysorbate degradation, including process optimization, excipient selection, and the use of inhibitors.
- Impact of Polysorbate Degradation on Product Quality: Discuss the potential consequences of polysorbate degradation on product stability, efficacy, and safety.
- Case Studies of Polysorbate Degradation: Share real-world examples of polysorbate degradation challenges and the solutions implemented.
- Future Directions in Polysorbate Management: Explore new approaches for stabilizing polysorbates and preventing degradation, including the use of novel excipients and formulation strategies. Discuss the potential of more sensitive analytical methods.
The biopharmaceutical industry is moving towards greater flexibility and efficiency in manufacturing. This session will explore the concept of integrated, product-agnostic biomanufacturing facilities – those designed to produce a range of biologics using common equipment and platforms. Attendees will learn how this approach can reduce capital costs, accelerate development timelines, and improve responsiveness to market demands.
This session will take a grounded approach to the application of AI in biomanufacturing, exploring practical use cases, addressing common misconceptions, and discussing the limitations of current AI technologies.
- Approaching the challenges and considerations when scaling-up ADC production
- Process Optimization
- Quality Control
- Regulatory compliance
- Strategies to incorporate when scaling up conjugation reactions, purification processing, formulation development
- Implementation of robust quality control measures to ensure purity, potency & stability
- Case study on overcoming scale-up challenges
- Introduction
- Objectives and outline of course
- Main development stages and regulatory cadre
- The main streams of activity from cell line to drug product
- The CTD for Regulatory filings. Quality Module – overview of main sections
- The importance of CMC : Quality (and Safety and Efficacy)
- Supply
- The pillars of pharmaceutical development
- Criticality Assessment
- Control strategy & Validation
- QBD Principles
- Most relevant regulatory Guidance and where to find it MH
Quiz
- The evolution of biopharmaceutical modality
- Analytical methods and their purpose in biopharmaceutical development and manufacturing
- Analytical method development and validation
- Product physicochemical characterization - high-performance liquid chromatography (HPLC), capillary electrophoresis (CE), spectroscopy, imaging, and post-translational modification (PTM)
- Product biological assays - cell-based assays (CBA), enzyme-linked immunosorbent assays (ELISA), and potency assays
- Microbiological contaminants - sterility testing, endotoxin testing, and microbial limits testing
- Process impurity testing - host cell DNA, host cell proteins, chromatography ligand
- Role of quality control (QC) and quality assurance (QA) in biopharma
- Case studies and industry examples
- Latest and future advancements in analytical methods and quality assurance
In order to achieve development timelines, rapid process development can result in the sacrifice of capsid recovery in order to meet specifications. By establishing a strategy focused on critical process parameters, leveraging design of experiments strategies, and using statistically sound analytics, genome-containing capsid recovery can be optimized in recombinant AAV downstream unit operations.
In order to achieve development timelines, rapid process development can result in the sacrifice of capsid recovery in order to meet specifications. By establishing a strategy focused on critical process parameters, leveraging design of experiments strategies, and using statistically sound analytics, genome-containing capsid recovery can be optimized in recombinant AAV downstream unit operations.
The CGT Supply Chain forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences between supply chain executives of 20-30 CGT therapeutic development companies.
An exclusive invite-only session, the forum will include 2-3 case studies and panel discussions focussed on brainstorming solutions to navigate the complexities of supply chain management for CGTs. Offering comprehensive insights into innovations, tools and trends to enhance efficiency, visibility, and responsiveness across the entire network and ultimately increasing CGT access to patients.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Discover cutting-edge analytical strategies for characterizing complex next-generation biologics, including LNPs, hypoimmune cell therapies, and CRISPR-based treatments. As cell and gene therapies rapidly evolve, innovative analytical techniques are needed to ensure product quality, accelerate development, and navigate the evolving regulatory landscape. This session explores new tools for characterizing engineered immune cells and gene-edited therapies, the growing role of Next-Generation Sequencing (NGS) and Mass Spectrometry (MS), and the integration of emerging bioanalysis techniques. Attendees will gain actionable insights to tackle the analytical challenges posed by complex biologics and learn about innovations in analytical methodologies for biopharmaceutical characterization, process analytics, and advanced automated peptide synthesis and purification methods
- Optimizing bioconjugation processes for dual-payload ADCs
- E.g. Cysteine conjugation, amino acid incorporation, for payload attachment
- Varying payload incorporation
- Manufacturing considerations for site-specific conjugation
- Analytical method development for complex ADCs
- CMC aspects of regulatory submissions for novel ADCs
Process intensification (PI) is a strategy to maximize throughput and reduce operational costs by improving the efficiency of each step in the downstream process. This session will cover the latest techniques in PI, such as multi-column chromatography, hybrid filtration systems, and integrating various purification steps. Attendees will learn how these innovations are driving faster and more cost-effective biologics manufacturing.
- How to effectively adopt AI tools in the CGT space
- Opportunities for AI and machine learning in process development & manufacturing
- Real-world applications of AI in CGT pipeline development
- Case study insights on AI-driven innovation
- How to effectively adopt AI tools in the CGT space
- Opportunities for AI and machine learning in process development & manufacturing
- Real-world applications of AI in CGT pipeline development
- Case study insights on AI-driven innovation
Automation is rapidly transforming downstream processing by improving reproducibility, reducing labor costs, and accelerating timelines. This talk will focus on the integration of automation technologies in the downstream workflow, from automated chromatography systems to robotic liquid handling and filtration. The speaker will discuss how automated systems enhance productivity, improve process control, and allow for more consistent and scalable processes.
BioPhorum members, through an industry-wide collaboration, have devised a proof-of-concept for controlling product collection during cation exchange (CIEX) bind-and-elute chromatography. Our approach to this task, using real-time multi-angle light scattering (RT-MALS) technology, involves directly monitoring aggregate content - a critical quality attribute (CQA) - and applying a defined breakthrough or cumulative threshold to stop pooling.
The intent of this partnership is to show that direct monitoring of a suitable CQA enables recovery of good material, currently lost due to conservative pooling practices. In addition to yield improvements for this polishing unit operation, the resulting pools should be more consistent batch-to-batch with respect to aggregate content. In this presentation, we share results from Phase I of the project.
MALS technology measures the intensity of scattered light produced by a sample at different angles. The scattered intensity relates to the molar mass and size. Averaged over a sample, the reported value is the weight-average solution molar mass (Mw) which will increase with increasing aggregate content. In our work, the in-line MALS detector reports the change in Mw in real-time allowing us to “see” when the aggregate population starts to break through. This is already an improvement over traditional UV which does not differentiate between protein populations. We could have set a reasonable threshold limit to the Mw signal and used it to trigger stop pooling and moved on with our work; however, the team took a more ambitious approach to use the Mw measurement in a more accurate way to calculate the cumulative aggregate content in the pool and use a cumulative aggregate threshold to trigger stop pooling. The more ambitious approach ensures tighter consistency of aggregate content in pools batch-to-batch, irrespective of peak shape. This approach also reduces the need for conservative pooling since stop pooling is triggered by the actual CQA (pool % HMW).
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
An acceleration in the speed at which therapies are breaking through from basic scientific discovery to development is placing a premium on innovative business development models. Biotech innovators, Pharma, and VCs alike are competing for successful win-win deal-making models in the face of novel personalized applications of gene therapies, gene editing, regenerative medicines, vaccines, therapies, and companion biomarkers. This session will cover best practices and strategies in finding the right partnerships to engage.
Spotlight Presentation – Calling all Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Examples of working with biopharma customers to optimize raw material formulations for specific applications.
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
The CGT C-Level Forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences between executives of ~20 CGT therapeutic development companies. The forum is designed to foster new ideas and create new relationships between attendees so that they can leave with tangible outcomes and that will drive their businesses and the sector forward.
An exclusive invite-only session, in a closed room setting, the forum will include 2-3 case studies discussing the right approach to engage and partner with pharma and industry. It will also include general discussion focussed on brainstorming solutions to the most pressing and current challenges of CEO/CSO/CBO/CMOs in the CGT sector.
Process intensification is a key strategy to increase the productivity of cell culture systems while reducing footprint and cost. This session will discuss methods such as increased cell density, optimized feeding strategies, and higher perfusion rates. The speaker will highlight examples of successful process intensification in mammalian cell cultures and how this approach can lead to higher product titers and more efficient use of resources.
Delivery of plasmid DNA enables systemic expression of therapeutic molecules, including but not limited to monoclonal antibodies, proteins and peptides. However conventional delivery techniques are limited by constraints of redosability and toxicity in their ability to delivery DNA effectively. Polymeric systems can overcome these constraints but have a very large design space. This presentation will describe how machine learning can leverage large design spaces for the design of polymeric delivery vehicles for a broad range of therapeutically relevant molecules in vivo.
Delivery of plasmid DNA enables systemic expression of therapeutic molecules, including but not limited to monoclonal antibodies, proteins and peptides. However conventional delivery techniques are limited by constraints of redosability and toxicity in their ability to delivery DNA effectively. Polymeric systems can overcome these constraints but have a very large design space. This presentation will describe how machine learning can leverage large design spaces for the design of polymeric delivery vehicles for a broad range of therapeutically relevant molecules in vivo.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
- The evolution of biopharmaceutical modality
- Analytical methods and their purpose in biopharmaceutical development and manufacturing
- Analytical method development and validation
- Product physicochemical characterization - high-performance liquid chromatography (HPLC), capillary electrophoresis (CE), spectroscopy, imaging, and post-translational modification (PTM)
- Product biological assays - cell-based assays (CBA), enzyme-linked immunosorbent assays (ELISA), and potency assays
- Microbiological contaminants - sterility testing, endotoxin testing, and microbial limits testing
- Process impurity testing - host cell DNA, host cell proteins, chromatography ligand
- Role of quality control (QC) and quality assurance (QA) in biopharma
- Case studies and industry examples
- Latest and future advancements in analytical methods and quality assurance
• Recent advancements in Raman spectroscopy, near-infrared (NIR) spectroscopy, and IR spectroscopy
• Case studies on implementing high-throughput PAT for upstream process development
• Challenges and opportunities in scaling PAT for GMP bioreactors and downstream processes.
- Gap analyses of the studies available to prepare a Phase 1 submission.
- 4 teams (Analytics, DS, DP, Materials) to cover different parts of case
- 30 – 45 min work in groups : Brief :
- do the existing studies support a Phase 1 file and what are the risks involved?
- Recommendation on eventual additional or superfluous studies
- Map studies / data onto CTD modules (if time)
- 60 min debrief (15 min / group)
- General Conclusions and End of Session
This session will delve into the complex regulatory landscape surrounding the use of Artificial Intelligence (AI) and Machine Learning (ML) in the development, manufacturing, and quality control of biologics. Experts will discuss evolving guidelines from regulatory agencies, including best practices for validating AI/ML models, ensuring data integrity, and demonstrating the reliability and transparency of AI/ML-driven decisions, panelists will deliver strategies, improvements in processes, and lower costs of operations through the proper application of the data. Key focus areas include strategies for generating trust and confidence in AI/ML-based analytics for regulatory submissions and maintaining compliance throughout the product lifecycle with specific actionable strategies
Join industry experts as we explore the cutting edge of non-viral gene delivery. This dynamic discussion will cover:
- The Non-Viral Advantage – Where does it shine over viral vectors?
- Beyond the Liver – Innovative delivery routes (brain, tissue-specific targeting).
- Next-Gen Delivery Vehicles – How to utilize LNP alternatives (EVs, VLPs etc) and machine learning-powered targeting
- Optimizing Payloads – Single vs. combination payload strategies
- Analytical & Regulatory Insights – Ensuring safety, efficacy, and consistency
- Future Trends – Will in vivo LNP therapies overtake ex vivo approaches?
- Collaboration & Acceleration – How can companies drive non-viral innovation?
Where is non-viral delivery headed? Let’s discuss the breakthroughs shaping the future!
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Through a series of presentations, case studies, interactive discussion and exercises this workshop will delve deeply into the underlying science of developing potency assays.
Some of the topics to be discussed include:
- Overview of what potency is, and what it is not
- Considerations when qualifying and validating bioassays
- From initial ideas, through characterisation to the final potency method
- Clinical correlation
- Regulator objections and why
- Examples of potency assays for approved products
- Examples of a cell and gene therapy potency assays for various product types
- Targeting ligand selection and impact on isotope selection
- Advancements in radiochemistry for improving the safety & efficacy of radiopharmaceuticals
- E.g. use of chelators to attach radioligands to targeting vehicle
- Impacts on manufacturing and quality
- Dealing with variability in radioisotope production
- Implementing robust quality control measures for radioactive materials
- Optimizing manufacturing processes to account for isotope decay
- Strategies for consistent batch-to-batch production
- Considerations for new radioisotopes and indications
- Biodistribution
- Metabolism, Excretions and Decay
- CMC considerations
- Growing importance of digitalization, AI, and machine learning in the biopharma industry.
- Key pillars of digital transformation in biopharma.
Key Areas of Digitalization
- Data Management and Integration (from Development to Manufacturing).
- Automation and Robotics in bioprocess workflows.
- Real-time Monitoring and Advanced Analytics for process optimization.
Applications in Bioprocessing
- Use of digital twins and AI to optimize upstream and downstream unit operations.
- Role of ML/AI-driven tools for Advanced Therapy Medicinal Products (ATMP) manufacturing.
- AI-driven real-time monitoring, predictive maintenance, and anomaly detection in production lines.
- Simulation-based process development for rapid scale-up.
Challenges and Considerations
- Overcoming data silos and ensuring system interoperability.
- Addressing regulatory requirements for AI and digital tools in GMP environments.
- Ensuring data quality, integrity, and security in digitalized workflows.
- Bridging talent gaps and fostering a digitally skilled workforce.
Case Studies
- Real-world examples of digital transformation in bioprocessing.
- Lessons learned from integrating AI-driven tools in ATMP production.
Future Trends and Directions
- Adoption of Industry 4.0 principles in biopharma manufacturing.
- Emerging technologies such as edge computing and IoT for bioprocessing.
- Sustainability and digitalization: How to?
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
A Representative from Solvias
A Representative from Solvias
- Explain what downstream bioprocessing involves: the purification and formulation of the biological product after cell culture and fermentation.
- Outline the key objectives: ensuring product purity, quality, and stability.
- Describe the process of harvesting cells or extracellular products from the bioreactor.
- Explain the methods used for cell separation (e.g., centrifugation, filtration).
- Introduce the main purification methods: chromatography, filtration, and precipitation.
- Describe different types of chromatography (e.g., affinity, ion-exchange, size-exclusion) and their applications.
- Explain the principles and applications of ultrafiltration and diafiltration.
- Introduction to Biopharmaceutical Life Cycle.
- Explain what upstream bioprocessing involves: the early stages of production, including cell culture and fermentation.
- Outline the key objectives: generating the desired biological product through cell growth and expression.
- Discuss the selection of cell lines (e.g., CHO cells, microbial cells).
- Introduce bioreactors and their role in providing a controlled environment for cell growth.
- Discuss different types of bioreactors (e.g., stirred-tank, wave, single-use) and their applications.
- Explain the fermentation process and its parameters (e.g., pH, temperature, oxygen levels).
- Explain the importance of culture media in supporting cell growth and productivity.
- Describe the process of scaling up from lab-scale to commercial-scale production.
- Highlight current trends in upstream bioprocessing (e.g., single-use technologies, continuous processing).
- Discuss future directions and innovations in the field.
Some of the topics to be discussed include:
- Overview of what potency is, and what it is not
- Considerations when qualifying and validating bioassays
- From initial ideas, through characterisation to the final potency method
- Clinical correlation
- Regulator objections and why
- Examples of potency assays for approved products
- Examples of a cell and gene therapy potency assays for various product types
Traditional QC methods often cause bottlenecks due to their slowness, labor intensity, and variability. This session explores cutting-edge automation solutions, high-throughput analytics, and real-time release testing (RTRT) designed to dramatically enhance efficiency and data integrity in biopharmaceutical QC, streamline workflows, and accelerate testing. As gene and cell therapies move to commercial manufacturing, you'll learn how to scale QC processes, ensure assay transferability, and maintain data integrity across production sites. The session will cover implementing RTRT and high-throughput analytics, strategies for harmonizing QC across multiple manufacturing locations, and the rise of plug-and-play analytical tools. Attendees will gain actionable insights for optimizing QC operations, improving process control, ensuring compliance, and reducing risk during commercial expansion. Learn about next-gen QC strategies that increase efficiency, improve consistency, and ensure regulatory compliance while reducing product release bottlenecks.
With AI amd Machine Learning gainig traction in analytics and process control, many companies are still unclear on the actual benefits of implementation. This session critically examines the real-world applications, separating genuine advancements from overhyped claims. Bringing together industry leaders, AI pioneers, and regulatory experts, the session will discuss successes, failures, and where AI is truly delivering value. It will explore successful AI/ML implementations for process optimization, predictive quality control, streamlined data analysis, and the automation of flow cytometry, how AI-driven computational modeling is reshaping biomanufacturing, and the next five years of AI in analytical sciences and QC. Attendees will gain insights into AI’s current applications and future potential, how it redefines quality control, data analysis, and process optimization, and hurdles that need to be overcome.
Round Table 1: Scaling CP: From Bench to Plant
- What breaks between lab-scale CP and full-scale implementation?
- What scale counts as “commercially viable” for CP?
- What's missing in current tech to make this easier
Round Table 2: The Business Case for Hybrid and CP Models
- How do you model ROI on continuous or hybrid investments?
- What incentives drive adoption (speed, cost of goods, flexibility)?
- When is it worth revisiting old filings to modernize an existing process?
Round Table 3: Continuous But Flexible: Connected vs Fully Continuous Systems
- Is connected processing a stepping stone or an endpoint?
- When is hybrid actually better than true CP?
This session will provide practical advice for bioprocess programs navigating post-approval submissions and late-stage commercialization. A key focus will be post-approval comparability, including strategies for managing situations where materials fall outside of established acceptance criteria. The talk will explore the use of risk assessment.
• Case study on challenges with formulating high concentration biologics – particularly emerging biologics
• Improve viscosity to avoid issues with syringe ability and injectability - development of specialized delivery devices
• Optimising buffer systems and excipient usage
• Maintaining homogeneity
• Storage and temperature challenges
- What is required for product characterization by regulatory authorities?
- Use of risk-based assessment to identify product CQAs
- In-process controls, stability, and release testing expectations from authorities
- How analytics can be used to support the move to “the product is the product”
- Development of rapid process characterization assays
- What is required for product characterization by regulatory authorities?
- Use of risk-based assessment to identify product CQAs
- In-process controls, stability, and release testing expectations from authorities
- How analytics can be used to support the move to “the product is the product”
- Development of rapid process characterization assays
Scalability is critical to ensure that bioprocesses developed at small scales translate to larger scales without compromising product quality, yield, or consistency in cell growth kinetics. Maintaining key culture conditions, such as nutrient supply, mass transfer, and mixing, is essential for achieving consistent cell growth, metabolic behavior, and viral vector production. This study explores how intensified cell perfusion systems can work hand in hand with cell culture media development to address these challenges. Our focus for this work is on scale-up strategies that ensure reproducible cell growth and viral vector production across different scales in HEK293 cell cultures.
Join us for a session to discover how to:
- Enable optimization of nutrient balance, execute efficient waste management, and achieve consistent and scalable HEK293 cell growth via a specialized HEK293 media perfusion process
- Intensify viral vector production by transiently transfecting cells across various perfusion processes and bioreactors
- Achieve consistent HEK293 cell metabolic profiles in different cell densities, demonstrating a robust and versatile bioprocess with precise control of bioprocess parameters
- Reduce operational costs while enhancing titer yield with the combination of automated control systems and advanced perfusion techniques
Small group discussions focused on specific challenges in CDMO selection, such as:
- Phase-appropriate CDMO selection & finding CDMOs with niche capabilities (e.g., specific cell lines, advanced therapies).
- Balancing cost, quality, and timelines in the selection process.
- Building trust and transparency in the initial stages of partnership.
30min table discussion
10 minute findings feedback to the group for each table
This talk will address the critical challenges of technology transfer, particularly the data-related bottlenecks that often hinder the scaling of projects from lab to clinical trial and manufacturing. It will explore the role of software solutions in streamlining this process and discuss how modernizing tech transfer practices can improve efficiency, data handling, QA, and overall speed. The presentation will offer practical strategies for overcoming these hurdles and provide insights into what an optimized, modernized tech transfer workflow could entail.
- Defining point-of-care manufacturing and decentralized manufacturing
- Scaling with consistency: key elements for global standardization
- Tech & innovation: cutting-edge solutions for safe, efficient, and cost-effective decentralized manufacturing
- Overcoming obstacles to decentralised manufacturing to enable access to approved CGTs
- Differences in systems across decentralized cell therapy manufacturing models - how can we harmonize?
- Standardization vs a modular approach
- Equipment, data, and technologies
- Dialogue and collaboration between developer/practitioner to streamline eventual implementation
- Improving worldwide access: how can local manufacturers and supplier help?
- Real world experience with a decentralized cell therapy manufacturing platforms supporting Phase I/II clinical trials
UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene for the treatment of Wilson Disease. UX701 leverages Ultragenyx’s proprietary producer cell line platform, Pinnacle PCL™, to produce rAAV at 2000L scale.
Process characterization of UX701 is an essential aspect of the process validation lifecycle, aimed at defining manufacturing process ranges to maintain the target product profile (TPP). A risk-based approach was applied to the late-stage development activities for UX701 upstream and downstream process including process parameter and raw materials risk assessment, scale-down model (SDM) qualification, and process characterization (PC) studies, to establish process controls and inform parameter criticality for the upstream and downstream unit operations in the 2000L manufacturing process. We assessed over 400 upstream and downstream process parameters for their impacts to process performance indicators (PIs) and critical quality attributes (CQAs) and classified them into high, medium or low risk parameters. Of these we studied 11 high and medium risk upstream process parameters and 2 raw materials, and 14 high and medium risk downstream process parameters in PC studies.
We identified 8 process parameters in upstream and 3 downstream process parameters to be critical (CPP) or key (KPP). In the upstream process, temperature and pH, seeding density, and helper virus concentration were all found to be CPPs during production. In the downstream process, viral heat inactivation step, temperature and time affect the inactivation kinetics of the helper virus. Characterization of our anion-exchange (AEX) polishing chromatography step identified an upper threshold which the load conductivity should stay below to ensure binding onto the AEX column.
In this work, we have characterized the cell culture and purification manufacturing process and demonstrate a robust and high yielding process for the manufacture of UX701. This is the first PC campaign for our Pinnacle PCL™ rAAV gene therapy manufacturing platform.
In an effort to access cutting edge research and stay ahead of the curve during this unprecedented time, investors and pharma companies are taking less risks. What long term trends have fizzled out and what is on the rise to increase innovation? Are new strategies sustainable as increasing investment rounds requiring more and more funds to obtain new treatments, therapies and technologies?
Leading biopharma experts and VCs will share their insights on how to engage investors, which trends to watch for, how to position technologies and discoveries to attract investors, and navigate this evolving landscape.
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Round off your day at BPI School with an interactive and insightful discussion:
- Learnings of the day and a chance to recap:
- Key trends and challenges in biopharmaceutical manufacturing, upstream and downstream.
- The impact of digitalization on the future of biopharma.
- Emerging therapies: Opportunities and obstacles.
- How to utilize and implement analytics tools
- Q&A and interactive discussion with industry experts.
- Bridging the gap between theory and practice in biopharma.
- Understanding emerging therapies: distinctions between cell therapy, gene therapy, etc.
- Therapeutic potential and current clinical landscape of different emerging therapies, unique challenges and opportunities presented.
- Differences and similarities from ‘traditional’ biologics:
- What learnings can we take from traditional modalities to approach novel modalities?
- Understanding the Cell Therapy and Gene Therapy manufacturing processes.
- Best practices when entering/transitioning into the advanced therapy industry.
- Leveraging experiences from your background into industry.
- Strategies and approaches to best utilise available technologies in the development & production of emerging therapies.
- Moving and translating research from academia, to start up, industry, and beyond.
- Understanding the difference between these, how to transition, pros and cons.
- Lessons and experiences from our panellists.
- Defining clear requirements and objectives in your RFP.
- Key criteria for evaluating potential CDMO partners (e.g., technical capabilities, quality systems, financial stability, cultural fit).
- Best practices for conducting thorough due diligence (technical, quality, financial).
- Utilizing site visits and audits effectively.
- Novel radiolabelling techniques and their impact on manufacturing
- Microfluidics in radiopharmaceutical production
- Scaling up production for solid tumor therapies
- Site-specific conjugation methods to enhance stability and targeting of radioligand-based therapies
- Enzymatic conjugation
- Click chemistry
Label-free optical microscopy enables non-perturbative profiling of biological samples based on their intrinsic molecular properties. We developed a multimodal label-free optical imaging system to identify biopharmaceutical CHO cell lines with favorable process performance at early stages. Combined with a machine learning-assisted single-cell analysis pipeline, the system achieved over 95% classification accuracy in early passages (0–5). This approach offers a promising solution to accelerate cell line selection, reducing time and resource demands in biopharmaceutical development.
- Improving the precision and control of drug attachment in ADC conjugation with site-specific conjugation techniques
- The use of engineered antibodies (e.g. THIOMABs)
- Implementing selenocysteine-containing antibodies
Development of novel conjugation techniques to enhance efficiency and specificity for improved patient outcomes
- Which CGT modalities, disease areas, and technologies are top priorities for big pharma investment?
- How major players are integrating CGT into wider R&D pipelines
- Key breakthroughs shaping pharma’s next moves in CGT
- Allocating R&D investment: Autologous vs. Allogeneic programmes
Through a series of case studies this session will focus on the latest innovations and advances in next-generation therapies.
- Explore cutting-edge preclinical and clinical case studies driving the future of cell and gene therapy.
- Discover how data-driven innovation is transforming therapy development, from control and targeting to tackling undruggable diseases.
- Unpack the challenges vs. traditional approaches, advancements in new modalities, and the manufacturing needs shaping commercialization of future therapies.
Key Case Studies Areas Include:
- Gamma Delta T Cells & T Cell Engagers
- mRNA, RNA, & NK Therapies
- Multiplex & Combination Cell Therapies
- 3D Bioprinting & Scaffold Development
- Solid Tumours & Expanding Disease Areas (Autoimmune, Neurology, Cardiometabolic)
- Early-Stage Considerations: How can formulation scientists and process development teams collaborate early in the development lifecycle to ensure seamless transition to fill finish? What are the key formulation attributes that need to be considered from an aseptic processing perspective?
- Handling Challenging Formulations: What are the best practices for aseptically processing complex formulations (e.g., high concentration, viscous, sensitive biologics)? What innovative technologies or approaches can be employed?
- Impact of Excipients on Aseptic Processing: How do different excipients influence the aseptic process? Are there any excipients that pose particular challenges for sterilization or filtration?
- Single-Use Systems and Formulation Compatibility: How does the increasing adoption of single-use systems in fill finish interact with different biologic formulations? Are there any compatibility considerations?
- Real-World Case Studies: Panelists could share anonymized examples of challenges encountered at the intersection of formulation and aseptic processing and the solutions implemented.
- Future Trends and Innovations: What emerging formulation strategies or fill finish technologies hold promise for improving the integration of these two critical areas?
Traditional analytical methods offer incomplete insights into biologic products. Multi-omics approaches, integrating proteomics, genomics, and metabolomics data, provide a holistic view of product quality and batch consistency. As the industry seeks deeper root cause analysis and process optimization, companies must leverage advanced analytical frameworks for thorough characterization. This session will explore high-resolution mass spectrometry for integrating multi-omics data, next-generation sequencing (NGS) for transforming process characterization, and innovative omics-based analytics for enhanced biologics development. Attendees will gain a clear strategy for leveraging multi-omics for deeper process insights, ensuring data integrity and regulatory compliance, and streamlining process development, with insights from Sartorius on applying omics-based technologies for root cause analytics and overall process understanding.
- Case Studies: Success stories of regulatory approvals and commercialization
- Overcoming Hurdles: Key challenges from R&D to approval—and how to tackle them
- Scaling Up: Strategies for manufacturing, validation, and CMC data packages
- Regulatory Insights: Feedback from authorities on characterization, specifications, and approval pathways
- Post-Approval Management: Long-term data tracking, durability studies, and lifecycle strategies
- End-to-End Logistics: Mastering supply chain and global distribution
- Pathways to approval –Case study on the journey from discovery to commercial development and beyond
This talk will focus on specific strategies for removing high-risk HCPs, such as lipases, chromatin, and other species that bind to proteins or cause other processing issues. It will discuss the use of functionalized resins and membranes.
• Techniques for maintaining uniform cell suspension and minimizing damage
• Innovative bioreactor designs to enhance consistency in mixing and mechanical forces
• Case studies showcasing successful implementation in large-scale biomanufacturing
As gene therapy evolves beyond AAV-based delivery systems, new analytical challenges arise in ensuring genome integrity, optimizing therapeutic dosing, and mitigating immunogenicity risks. The industry is shifting toward alternative delivery methods such as CRISPR-based therapies, lipid nanoparticles (LNPs), plasmids, and synthetic vectors, requiring advanced analytical approaches to maintain product quality, efficacy, and regulatory compliance. This session will explore: Genome integrity analysis beyond traditional capsid characterization, leveraging long-read sequencing and multiplex digital PCR to assess payload stability and completeness, optimizing viral vectors for complete genomes, reducing dosing requirements, improving patient outcomes, and lowering production costs, second-dose strategies for non-AAV gene therapies, addressing durability, re-dosing challenges, and immunogenicity considerations, emerging analytical approaches for non-AAV delivery systems, including mRNA-based CRISPR therapies, LNP formulations, and synthetic gene vectors, key regulatory considerations for characterizing non-viral gene therapy platforms, ensuring alignment with evolving global standards.
This talk could address the increasing demand for biologics and discuss innovative solutions for capacity expansion, including modular facilities, process intensification, and improved resource utilization.
This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.
Understanding the Tech Transfer Process
Gain a clear roadmap for transitioning projects from laboratory research to large-scale clinical manufacturing.
Identify common challenges and best practices to ensure a smooth and efficient transfer.
Optimizing Scale-Up and Clinical Trial Readiness
Learn strategies for scaling up manufacturing processes efficiently.
Discover methods for ensuring reproducibility and overcoming obstacles in clinical trial preparation.
Navigating Regulatory Compliance and Tech Transfer Challenges
Explore key regulatory considerations in tech transfer.
Understand compliance challenges and how to effectively navigate regulatory requirements.
Enhancing Efficiency and Speed in Tech Transfer
Modernize processes with innovative approaches and new technologies.
Implement strategies to reduce development-to-manufacturing time and accelerate the tech transfer timeline.
Leveraging Automation and Cloud-Based Solutions
Explore the integration of automated tech transfer systems across platforms.
Learn how cloud-based solutions can enhance data transfer and collaboration.
Expert Insights and Practical Advice
Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.
Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.
- Snapshot into the investment landscape for CGT products and enabling technologies for 2025/ 2026 and beyond
- How to drive investment in CGT?
- Positioning for success in a competitive funding environment: What do investors need to see?
- “Ask the Investor” interactive Q&A open discussion with our investor panel to get tips and tricks when pitching and their views on where innovation lies in the CGT field
- Initiatives to reduce their carbon footprint and implement recycling programs
- Defining point-of-care manufacturing and decentralized manufacturing
- Scaling with consistency: key elements for global standardization
- Tech & innovation: cutting-edge solutions for safe, efficient, and cost-effective decentralized manufacturing
- Overcoming obstacles to decentralised manufacturing to enable access to approved CGTs
- Differences in systems across decentralized cell therapy manufacturing models - how can we harmonize?
- Standardization vs a modular approach
- Equipment, data, and technologies
- Dialogue and collaboration between developer/practitioner to streamline eventual implementation
- Improving worldwide access: how can local manufacturers and supplier help?
- Real world experience with a decentralized cell therapy manufacturing platforms supporting Phase I/II clinical trials
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Some of the topics to be discussed include:
- CDMO Selection: Models and Process
- Contract and Quality Agreement Negotiation
- Tech Transfer: Best practices and risk management for external tech transfers
- Digital Transformation in Tech Transfer
- Quality Assurance and Quality Control
- Life Cycle & Relationship Management
A Representative from Intertek
- Case study examples on potency assay development and validation strategies – What factors/ studies to choose for potency?
- How far do you have to go with regards to potency?
- Functional assays for late-stage programmes
- Requirements for development vs BLA – How to account for BLA requirements from the start?
- Specification requirements to meet regulatory expectations
Feedback received from regulatory authorities on expectations for potency assays
Onechain Immunotherapeutics developed a stromal-free bioprocess for the novo generation of γδ T cells from cord blood (CB) CD34+ HSPC or iPSC-derived CD34+, using recombinant Notch ligands, eliminating the need for expanding these cells from peripheral blood or other sources. The process can yield functional γδ T cells that can be armed with CARs with cytotoxic activity against multiple cancer cells. This strategy is highly scalable, consistent with GMP guidelines, and represents a step forward in the field of allogeneic, off-the-shelf CAR-T cell therapies.
- Leveraging CDMO expertise for specialized technologies and capabilities.
- Mitigating risks and enhancing supply chain resilience through external partners.
- Accelerating speed to market and managing capacity constraints.
- The role of external manufacturing in supporting innovation pipelines.
This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.
Understanding the Tech Transfer Process
Gain a clear roadmap for transitioning projects from laboratory research to large-scale clinical manufacturing.
Identify common challenges and best practices to ensure a smooth and efficient transfer.
Optimizing Scale-Up and Clinical Trial Readiness
Learn strategies for scaling up manufacturing processes efficiently.
Discover methods for ensuring reproducibility and overcoming obstacles in clinical trial preparation.
Navigating Regulatory Compliance and Tech Transfer Challenges
Explore key regulatory considerations in tech transfer.
Understand compliance challenges and how to effectively navigate regulatory requirements.
Enhancing Efficiency and Speed in Tech Transfer
Modernize processes with innovative approaches and new technologies.
Implement strategies to reduce development-to-manufacturing time and accelerate the tech transfer timeline.
Leveraging Automation and Cloud-Based Solutions
Explore the integration of automated tech transfer systems across platforms.
Learn how cloud-based solutions can enhance data transfer and collaboration.
Expert Insights and Practical Advice
Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.
Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.
- Review of the main streams and initial status
- Broad requirements for Tox and for First in Human Studies
- Cell banking
- Other Raw materials
- Drug Substance Process and Manufacture
- Minimum requirements
- Understanding your process :
- Impurities : identity, clearance, control
- First steps towards a control strategy
- Adventitious contamination and Viral Clearance Studies
- Drug formulation and Drug Product Processing
- Analytical package
- Release methods definition and development
- From method performance to method validation
- In Process Controls (else cover under process?)
- Batch data in the submission
- Product Characterisation and Reference standard
- Stability ( DS and DP)
- Forced degradation studies : necessity and importance
- Why is stability important ?
- Different type of stability studies and typical package for PhI
- Shelf life assignment
Join industry leaders as we explore how AI, digitalization, and advanced analytics are transforming cell and gene therapy manufacturing. This dynamic panel will dive into:
- AI-Powered Manufacturing: How AI-driven models, predictive analytics, and digital twins are optimizing process control
- Process Analytical Technologies (PAT): The latest breakthroughs in real-time monitoring, automation, and data-driven decision-making
- Overcoming Data Challenges: Strategies to build robust AI models despite limited datasets
- Success Stories: Case studies showcasing AI and PAT integration for improved yield, efficiency, and product quality in cell and gene therapy manufacturing and process development
This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.
Understanding the Tech Transfer Process
Gain a clear roadmap for transitioning projects from laboratory research to large-scale clinical manufacturing.
Identify common challenges and best practices to ensure a smooth and efficient transfer.
Optimizing Scale-Up and Clinical Trial Readiness
Learn strategies for scaling up manufacturing processes efficiently.
Discover methods for ensuring reproducibility and overcoming obstacles in clinical trial preparation.
Navigating Regulatory Compliance and Tech Transfer Challenges
Explore key regulatory considerations in tech transfer.
Understand compliance challenges and how to effectively navigate regulatory requirements.
Enhancing Efficiency and Speed in Tech Transfer
Modernize processes with innovative approaches and new technologies.
Implement strategies to reduce development-to-manufacturing time and accelerate the tech transfer timeline.
Leveraging Automation and Cloud-Based Solutions
Explore the integration of automated tech transfer systems across platforms.
Learn how cloud-based solutions can enhance data transfer and collaboration.
Expert Insights and Practical Advice
Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.
Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
- CDMO Selection: Models and Process
- Contract and Quality Agreement Negotiation
- Tech Transfer: Best practices and risk management for external tech transfers
- Digital Transformation in Tech Transfer
- Quality Assurance and Quality Control
- Life Cycle & Relationship Management
- Improvements and innovations in cell line development & engineering to increase yields for gene therapies
- Application of producer cell lines to lower transfection vector costs: Data for large scale use of stable cell lines
- Scalability of producer cell lines? Case studies on experiences at larger scales
- Improvements and innovations in cell line development & engineering to increase yields for gene therapies
- Application of producer cell lines to lower transfection vector costs: Data for large scale use of stable cell lines
- Scalability of producer cell lines? Case studies on experiences at larger scales
- Case Studies: Success stories of regulatory approvals and commercialization
- Overcoming Hurdles: Key challenges from R&D to approval—and how to tackle them
- Scaling Up: Strategies for manufacturing, validation, and CMC data packages
- Regulatory Insights: Feedback from authorities on characterization, specifications, and approval pathways
- Post-Approval Management: Long-term data tracking, durability studies, and lifecycle strategies
- End-to-End Logistics: Mastering supply chain and global distribution
- Pathways to approval –Case study on the journey from discovery to commercial development and beyond
- Internalising or externalising manufacturing – What are the decision points?
- How to select CDMO partners? – Industry criteria for selection
- When / what stage of development should you bring on second CDMO?
- Understanding CDMO capacities, timelines, and capabilities
- Qualification, and quality agreements for commercial manufacturing
- Tech transfer: best practices and risk management
- Strategies for transferring product from development phase to CDMO for large scale production: How to scale up?
- Lessons learnt from transferring to CDMO and vice versa
- Increasing skills and experience in manufacturing of next generation CGT products
- Partnership and communication – how to partner most effectively with CDMOs?
Regulatory expectations for potency and release testing are evolving, particularly with the rise of advanced biologics such as CGTs, ADCs, and bispecific antibodies. Companies must navigate challenges in assay standardization, surrogate potency methods, and comparability assessments while ensuring compliance with global regulatory frameworks. This session, led by industry experts and regulatory specialists, will provide practical insights into meeting the latest standards, including ICH Q6B (Specifications for Biotechnological/Biological Products), USP General Chapter <1032> (Design and Development of Biological Assays), and FDA guidance on potency testing for gene therapy products. Attendees will gain actionable strategies for improving assay validation, reducing variability, and streamlining regulatory approval pathways.
During this session, leaders from the Biopharma community will discuss their approach to creating, promoting, investing in, and accessing innovation, and ultimately, coming together to bring cures to patients.
In an effort to access cutting edge research and stay ahead of the curve during this unprecedented time, investors and pharma companies are taking less risks. What long term trends have fizzled out and what is on the rise to increase innovation? Are new strategies sustainable as increasing investment rounds requiring more and more funds to obtain new treatments, therapies and technologies?
Leading biopharma experts and VCs will share their insights on how to engage investors, which trends to watch for, how to position technologies and discoveries to attract investors, and navigate this evolving landscape.
An acceleration in the speed at which therapies are breaking through from basic scientific discovery to development is placing a premium on innovative business development models. Biotech innovators, Pharma, and VCs alike are competing for successful win-win deal-making models in the face of novel personalized applications of gene therapies, gene editing, regenerative medicines, vaccines, therapies, and companion biomarkers. This session will cover best practices and strategies in finding the right partnerships to engage.
Whether they’re public-private partnerships, CROs and biotechs or incubators and pharma companies, novel agreements between stakeholders are leveraging the resources and unique expertise to deliver previously only theoretical contributions to science. This session will walk through review successful and impactful partnerships that are shaping the future of drug development.
This past decade has seen an explosion of technologies that are transforming healthcare. From AI to cell and gene therapies to immunotherapies, we’ve seen significant advances in life sciences and the impact on patients, but what can we expect in the next decade? What are investors looking for in the next wave of breakthroughs? What areas of development excite them most? What new business models enable riskier investments to support the development of these novel methods and technologies?
One-to-one meetings will be available to schedule online throughout the duration of the digital event.
Meetings on this day will start at 8:00am EST.
| 10:00 – 10:15 | Qatch Technologies |
| 10:15 – 10:25 | Bowhead Health |
| 10:30 – 10:45 | Dignify Therapeutics |
| 10:45 – 11:00 | Microvascular Therapeutics |
| 11:00 – 11:15 | XtalPi Inc |
| 10:45 - 11:00 | EydisBio Inc. |
| 11:00 - 11:15 | Mirimus, Inc. |
| 11:15 - 11:30 | TregTherapeutics |
| 11:30 - 11:45 | Aer Therapeutics, Inc. |
| 11:45 - 12:00 | Lumos Pharma Inc. |
| 12:00 - 12:15 | Shaperon Inc. |
1:00 - 1:10 Praetego Inc
1:20- 1:30 Peri-Nuc Labs
1:30 - 1:40 Adamas Nanotechnologies, Inc.
| 2:30 - 2:40 | ANYO Labs |
| 2:45 - 3:00 | Zucara Therapeutics Inc. |
| 3:00 - 3:15 | Lindy Biosciences, Inc. |
| 3:15 - 3:30 | HDT Bio |
| 3:30 - 3:45 | CellVax Therapeutics |
| 3:45 - 4:00 | Glycan Therapeutics |
| 4:00 - 4:15 | Caeregen Therapeutics |
The Startup Spotlight is a pitch competition featuring the most innovative startup biotech companies. This live pitch competition will give a group of hand selected startups the opportunity to pitch in front of the BioPharm America audience.
Startup Spotlight Presenters:
Atom Bioworks Inc.
Enrich Biosystems Inc.
GeneVentiv Therapeutics, Inc.
Opus Genetics
Qprotyn Inc.
SelSym Biotech
• What is considered a must for CLD?
• What about your process has proved indispensable?
• What might work in the future with more improvement?
A major challenge encountered during biotherapeutic process development is the control of product quality while maximizing cellular productivity. Despite many physical and biochemical parameters that are routinely measured in a bioreactor during manufacturing, a gap still remains in the inability to directly assess intracellular pathways which are linked to high fidelity protein expression. In this work, we developed and evaluated flow cytometry-based tools to measure cellular phenotypes to support process development in vaccine and biologics production cell lines. Utilization of pathway-specific markers of viability, redox, ER-stress and glycosylation at a single-cell resolution will enable a better mechanistic understanding of specific process changes that influence product quality attributes. We anticipate that the implementation of this workflow during early stage and commercial process development will facilitate more efficient refinement of processes which would ultimately yield higher quality biotherapeutics.
Plant-based cell lines bear the potential to provide a viable alternative to mammalian and eukaryotic expression for producing biological therapies in the pharmaceutical industry. The number of approved and commercially available plant-made pharmaceuticals (PMPs) has, however, remained limited. One notable example is taliglucerase alfa (Elelyso), a recombinant glucocerebrosidase enzyme produced using transgenic carrot cell cultures, approved by the FDA in May 2012 for the treatment of Type 1 Gaucher's disease. Moss-based expression systems have emerged as promising platforms for producing plant-made pharmaceuticals (PMPs) more broadly. These systems offer several advantages, including precise genetic modifications through homologous recombination, the ability to perform complex post-translational modifications such as N-glycosylation, and cultivation in controlled bioreactor environments that minimize contamination risks. Ongoing research has aimed to address remaining limitations, focusing on enhancing yield, improving morphology, and increasing the scalability and compatibility with standard fermentation infrastructure, thereby paving the way for broader application of moss-based manufacturing in the biopharmaceutical industry. This talk will provide novel data on an enhanced moss-based expression cell line with significantly improved growth rates and shortened doubling time as the basis to potentially match CHO-like expression titers for a range of biopharmaceutical developments. In addition, careful strain selection and genetic optimization has resulted in a photosynthesis-independent expression option, avoiding the need to customize existing fermenter equipment by adding artificial light sources and, as a consequence, unlocking the possibility to apply moss-based expression at any industrial scale.
Cell line development is crucial for biopharmaceutical production but traditionally time-consuming. The Beacon Optofluidic system accelerates this by screening thousands of clones in chips for antibody production. For bispecific antibodies production, we developed a beacon on-chip binding assay to measure heterodimers, validating its accuracy and reliability. This assay streamlined the process and improved efficiency in selecting high-quality clones.
Sequence confirmation is a key quality attribute in production cell line development. At Takeda, we established an internal next-generation sequencing (NGS) platform to support diverse projects, including GOI identification, retroviral vector sequencing, clone screening for gene knockouts, and adventitious pathogen detection, enhancing the accuracy and efficiency of our cell line development processes.
Cell line stability and long-term productivity are major concerns in biomanufacturing. This session will focus on strategies to optimize both, including the development of stable clones, innovative screening methods, and improvements in cell culture environments. Techniques like epigenetic modifications, the use of transposon-based vectors, and advancements in protein expression systems will be discussed.
Site-specific integration of genes that encode for therapeutic proteins gained traction in the field of cell line engineering in the last decade. Successful implementation of the technology depends on the integration site in the genome. Identifying optimal loci for targeted integration will benefit the site-specific integration platform and improve therapeutic protein production pipelines in internally derived CHO cell lines.
Cellular metabolism is a highly complex and dynamic process that is essential for maintaining cellular function and homeostasis. This complexity arises from the vast network of interconnected biochemical pathways, the diversity of metabolic intermediates, and intricate regulation mechanisms. Understanding cellular metabolism is therefore critical for optimizing bioprocesses and enhancing productivity. Metabolic Flux Analysis (MFA) is a powerful tool that provides metabolic flux rates and the ability to track the fate of metabolites. It can be used to uncover the metabolic signatures and metabolic bottlenecks that can influence protein production. MFA was performed on CHO cells cultured in different media and feed packages. The objectives were to elucidate metabolic bottlenecks, nutrient limitation or excess metabolic waste when using media #1 compared to media #2 and provide strategies to overcome them. The results showed impacts in glycolytic flux, amino acid catabolism, TCA cycle flux and production of toxic metabolic byproducts.
• Importance of selecting the right host (CHO cells, HEK293, etc.)
.• Advantages of CHO cells for monoclonal antibodies and limitations for more complex modalities
• Potential for new host cell lines for specific biotherapeutics
• Random integration vs. site-specific integration: Impact on product consistency
• Engineering approaches to enhance proper folding and reduce aggregation
• Screening strategies for high-producing, stable clones
• Use of synthetic promoters, pathways, and regulatory elements
Engineering strategies for bispecific antibodies have been developed to enhance the formation of heterodimer molecules; however, challenges persist in their expression due to polypeptide chain mispairing. To optimize the assembly of high-purity heterodimers, achieving the optimal expression ratios of each polypeptide chain is crucial. In this presentation, we will share case studies on vector design strategies for bispecific antibodies, demonstrating how these approaches can improve productivity and quality of multi-specific therapeutic proteins in biopharmaceutical applications.
• Advances in transposon technology for targeted integration and enhanced stability
• Stability improvements through transposon innovations: what works and what doesn’t
Label-free optical microscopy enables non-perturbative profiling of biological samples based on their intrinsic molecular properties. We developed a multimodal label-free optical imaging system to identify biopharmaceutical CHO cell lines with favorable process performance at early stages. Combined with a machine learning-assisted single-cell analysis pipeline, the system achieved over 95% classification accuracy in early passages (0–5). This approach offers a promising solution to accelerate cell line selection, reducing time and resource demands in biopharmaceutical development.
This will be a fireside chat discussing:
• The readiness of synthetic biology and cell-free systems for commercialization
• Challenges surrounding cost and scalability.
• How synthetic biology can complement traditional cell-based approaches
.• Real-world examples of success and where the field is headed next.
Amgen has developed an improved upstream process, integrating a robust cellular host and innovative cell culture bioprocess to enhance manufacturing productivity and reduce raw material costs. Compared with the legacy fed batch baseline, the improved process increased the productivity by 3-fold in fed-batch and an additional 2-fold in intensified perfusion process. To further increase the process robustness, an in-silico cell culture metabolic model was employed and optimize the feeding schedule, leading to increased cell density and titers across various modalities. The development of this optimized end-to-end process is a significant milestone in Amgen's mission to provide innovative therapeutic solutions. By combining a robust new CHO host with an optimized cell culture process, Amgen has enhanced manufacturing productivity, reduced costs, and increased the flexibility of its multi-modality pipeline. These advancements ensure that Amgen can continue to meet clinical mass demands and deliver high-quality, cost-effective treatments to patients worldwide.
Process intensification is a key strategy to increase the productivity of cell culture systems while reducing footprint and cost. This session will discuss methods such as increased cell density, optimized feeding strategies, and higher perfusion rates. The speaker will highlight examples of successful process intensification in mammalian cell cultures and how this approach can lead to higher product titers and more efficient use of resources.
Optimizing feeding strategies and amino acid concentrations is crucial for enhancing CHO cell metabolism and overall bioprocess performance. In this presentation, we will introduce an amino acid uptake rate calculator that leverages historical experiment data to effortlessly calculate amino acid uptake rates. Building on this, we will also present an innovative amino acid simulator tool that uses these uptake rates to simulate adjustments in process feeding and amino acid concentrations. By simplifying media complexity through enhanced predictability and boosting cost-efficiency by optimizing feeding strategies and resource allocation, these tools have been instrumental in achieving high-yield results. We will share a case study demonstrating how they have informed critical decisions in media formulation within laboratory settings, showcasing their impact on improving bioprocess performance and economic viability.
Increasing global demand for Alexion biologics necessitates optimization of upstream processes. This study extends our fed-batch platform capabilities through development of an intensified fed-batch (iFB) process. Optimizing media, implementing dynamic feeding, and integrating N-1 perfusion technology for high-density N-production inoculation achieved a >2-fold increase in volumetric productivity while maintaining product quality. Here we demonstrate a high-efficiency solution for biologics manufacturing.
This work discusses the in-house development of a smart APC platform in our Upstream Process Development group, leveraging MLOps for flexible and scalable AI deployment. In addition to case studies of various bioreactor advanced process control (APC) experiments (PAT & model-based), we will share our iterative journey of APC solutions, addressing social and technical challenges to ensure usability for non-software focused teams.
• Preparing media for high-volume systems (e.g., 50,000L tanks): daily, on-demand, or batch strategies?
• Reducing media reconstitution times to support continuous processing
• Challenges and opportunities in scaling up media production workflows
• Examining the strengths and limitations of ambr15 as a screening vs. modeling tool
• Sharing data on process performance across ambr15 and ambr250 scales
• Strategies for maintaining process consistency and scaling robustly from small-scale to production-scale
Scalability is critical to ensure that bioprocesses developed at small scales translate to larger scales without compromising product quality, yield, or consistency in cell growth kinetics. Maintaining key culture conditions, such as nutrient supply, mass transfer, and mixing, is essential for achieving consistent cell growth, metabolic behavior, and viral vector production. This study explores how intensified cell perfusion systems can work hand in hand with cell culture media development to address these challenges. Our focus for this work is on scale-up strategies that ensure reproducible cell growth and viral vector production across different scales in HEK293 cell cultures.
Join us for a session to discover how to:
- Enable optimization of nutrient balance, execute efficient waste management, and achieve consistent and scalable HEK293 cell growth via a specialized HEK293 media perfusion process
- Intensify viral vector production by transiently transfecting cells across various perfusion processes and bioreactors
- Achieve consistent HEK293 cell metabolic profiles in different cell densities, demonstrating a robust and versatile bioprocess with precise control of bioprocess parameters
- Reduce operational costs while enhancing titer yield with the combination of automated control systems and advanced perfusion techniques
• Recent advancements in Raman spectroscopy, near-infrared (NIR) spectroscopy, and IR spectroscopy
• Case studies on implementing high-throughput PAT for upstream process development
• Challenges and opportunities in scaling PAT for GMP bioreactors and downstream processes.
- How to ensure safety and compliance while accelerating clinical and manufacturing timelines
- Common pitfalls and best practices
- Strategies to address changing regulatory requirements during rapid development
- Strategic approaches to transitioning from clinical development to manufacturing readiness
- Aligning speed with regulatory expectations for product quality and safety
Protein hydrolysates are commonly used to enhance process performance and cell productivity. However, they often introduce challenges to process consistency. To address this, we have developed a transition strategy to a new platform using chemically defined raw materials suitable for all stages of development and manufacturing. This strategy maintains our agile development strategy to efficiently deliver our pipeline. To accelerate delivery, the team has adopted a multi-faceted approach to tackle various challenges associated with a global strategy transition. Starting with Cell Line Development, new media feeds were incorporated into our early clone selection processes, enabling seamless development cycles for upstream processes. Feeding into our pipeline projects, we introduced new raw materials with minimal difference to productivity or product quality. We have experimented with new process solutions across multiple pipeline projects, consistently delivering high-performance processes in fed-batch, intensified fed-batch, and perfusion operations. Through successful upstream optimization, our chemically defined media feeds have approached cGMP Implementation, ensuring supply chain and process consistency through stability testing, feed preparation characterization, and scalability of new formulations. In parallel, the team continues to adapt and refine our development media and feed solutions, enhancing productivity by optimizing chemically defined media through compound screening. We observed increases in antibody titers across multiple projects by implementing specific supplements and optimizing them using classical design of experiments (DoE). As we collect more data across modes of operation and scales, our approach evolves to include techniques such as metabolic flux analysis and systems biology to analyze metabolic differences between low- and high-productivity media feeds. In conclusion, our comprehensive strategy addresses raw material variability challenges while enabling product-specific modulations and productivity enhancements, all while accelerating our pipeline as we follow the science.
This study evaluates the use of capacitance probes in the Sartorius Generation2 ambr250 bioreactors
• How to optimize cell culture conditions (temperature, pH, agitation) early on to increase monomer purity, reduce aggregation, and improve product yield
• The role of media optimization, hydrolysates, and supplements in ensuring consistent product quality and speeding up scaling
• The value of process analytical technologies (PAT) in providing real-time data to improve upstream processes and reduce development timelines
• Strategies for cell line development, including overcoming the challenges associated with Bispecifics, gene therapy (e.g., iPSCs), and cell-based therapies
• The importance of automation and digital tools in streamlining upstream processes and reducing time spent on manual intervention
• The role of outsource partnerships (CDMOs) in accelerating biologics development, especially for small companies
• Case studies of how companies have successfully implemented upstream optimizations for faster time to market
High throughput (HT) technologies, such as the Ambr® 250 HT mini bioreactor system, have been adopted across the industry for early and late-stage development to address the increasing needs to further accelerate the development timelines for cell culture processes for recombinant therapeutic proteins. These HT technologies have the potential to streamline experimental turnaround and enhance throughput. However, much of the focus in late-stage development has been on process optimization and scale-down model (SDM) qualification. To demonstrate a complete end-to-end implementation of the system, a complete process characterization (PC) data package needs to be evaluated and benchmarked against an existing qualified SDM. Sartorius has developed a second-generation Ambr® 250 HT system with improvements intended to facilitate PC studies. This presentation focuses on the qualification of the second-generation Ambr® 250 system as a SDM and its application in PC for a Chinese Hamster Ovary (CHO) fed-batch process. This detailed case study, will showcase the consistency and robustness of the Ambr®250 system, validating its qualification as a SDM. Subsequently, we will compare the process data derived from the qualified SDM with that obtained from the Ambr® 250 system to highlight data comparability and reliability. This study underscores the potential of the Ambr® 250 system in late-stage process development.
Examples of working with biopharma customers to optimize raw material formulations for specific applications.
• Techniques for maintaining uniform cell suspension and minimizing damage
• Innovative bioreactor designs to enhance consistency in mixing and mechanical forces
• Case studies showcasing successful implementation in large-scale biomanufacturing
• Addressing supply chain disruptions: strategies for procurement, digitalization, and raw material management
• Sustainability challenges and cost-saving innovations for large-scale media production
• Case study: Overcoming challenges with poor monomer purity and antibody aggregation
• Strategies for optimizing cell culture conditions to enhance product quality
A next-generation, internally developed CHO cell culture media was implemented in Upstream Process development and subsequently transferred to a manufacturing facility. Upon scaling up the process with the new media, impaired cell culture growth characterized by low viability, high lactate levels, and low titer was observed. Analysis of the media loaded into the bioreactor revealed lower-than-expected copper concentrations. As a mitigation, copper was supplemented into the bioreactor via sterile solution, restoring expected growth. An internal investigation identified that copper was lost during filtration, suggesting it existed in a non-soluble form during preparation. A detailed study was conducted to pinpoint the specific time points during large-scale media preparation where copper converted to an insoluble form. Additionally, a bench-scale model mimicking largescale conditions was established to test mitigations. Factors such as pH, dissolved oxygen, and redox potential were identified as contributors to the issue. Long-term solutions tested included removing pH adjustments from the media preparation protocol and completely removing copper from the media powder. In summary, the investigation revealed that copper loss during large-scale media preparation was due to its conversion to a nonsoluble form, influenced by pH, dissolved oxygen, and redox potential. Supplementation restored cell growth, and long-term solutions involved protocol adjustments to address the copper solubility issue.
• Building the Cell Culture Toolbox for Modern Bioprocessing
• Discuss what hasn’t been done before?
• What needs to be done for that next big leap in innovation?
• What’s the dream?
• AI and automation beyond academia – real-world industrial applications
• How molecular modeling, AI, and ML integrate into bioprocess workflows
• Case study: AI-driven digital tools for process acceleration
- The role of AI in data structuring, report authoring, and regulatory submissions.
Automation is rapidly transforming downstream processing by improving reproducibility, reducing labor costs, and accelerating timelines. This talk will focus on the integration of automation technologies in the downstream workflow, from automated chromatography systems to robotic liquid handling and filtration. The speaker will discuss how automated systems enhance productivity, improve process control, and allow for more consistent and scalable processes.
Tangential flow filtration (TFF) is an essential cell retention device for the intensified dynamic perfusion cell culture in integrated dynamic bioprocessing (IDB). The demands of the IDB process are high: the TFF must support high cell concentration (>100 million CHO cells/mL) inlarge-scale bioreactors (~2000L) for an extended continuous bioprocess while maintaining relatively high sieving performance (low product retention) and relatively low residence time and mechanical stress on cells. Of these demands, those related to membrane fouling, and the concomitant large facility footprint and increasing operational complexity, remain the most persistent. Through bench scale experimentation and mechanistic modelling, we have found several operational regimes that increase filter capacity. Counterintuitively, our studies suggest that high flux TFF can increase apparent filter capacity by reducing the extent of Starling flow relative to permeate flow, resulting in decreased filter area demand. Utilizing high flux at large scales with sequentially run filters minimizes facility footprint and reduces operational demand during processing, compared to the low flux condition. In this work, we will review high flux, highcapacity data across varied membrane chemistry, vendors, and projects to explore the differences in fouling mechanisms and failure modes at different scales. Additionally, we will review how operational methods can be utilized to swap filters and remove foulants mid processing via TFF regeneration.
Modern biologics process development spans multiple stages and scales—ranging from early cell line screening and small-scale process optimization to toxicology campaigns, pilot studies, and large-scale production runs. At each step, substantial volumes of data are generated, yet often reside in disparate systems and formats, making integration a significant challenge. Traditional, manual data collection and offline processing can lead to errors, impede timely decision-making, and hamper overall process understanding. Here, we present a comprehensive, automated strategy that unifies upstream and downstream data capture within a single digital ecosystem. Leveraging a cloud-based data science and engineering platform, our approach seamlessly ingests raw instrument files, electronic lab notebook (ELN) data, and other operational records into a centralized repository. Automated transformation pipelines harmonize these diverse data streams, enforcing consistent formats and validating entries in near real-time. The resulting integrated database—accessed via interactive dashboards—provides immediate visibility into growth profiles, metabolite analytics, chromatographic performance, and product-quality attributes across various process scales. By eliminating offline merges and ensuring robust connections between upstream and downstream datasets, this paradigm accelerates timelines, reduces human error, and provides a single source of truth for all stakeholders. Over time, these curated data sets enable advanced statistical assessments and machine learning models to further enhance process development efficiency and robustness. This end-to-end solution demonstrates how automating data pipelines can break down traditional silos in biologics development, driving improved process understanding, faster decision cycles, and stronger alignment across multiple functional teams.
Solventum Harvest RC and RCC clarification filters are designed for harvesting cell cultures. These filters were compared with a platform monoclonal clarification process. The comparison included assessments of yield, product quality and clarity. The evaluation continued downstream to the subsequent Protein A affinity chromatography step. Additionally, the downstream Solventum Polisher ST, utilizing AEX membrane chromatography, was evaluated in comparison to a platform AEX process, similar to the upstream clarification and Protein A steps. The assessment included the use of Polisher ST without a pre-filter.
Technological advances in cell culture processing have not only led to increased cell densities and productivity, but also have resulted in increased levels of solids and sub-micron particles from cell debris, which considerably decrease the efficiency of the particle separation through centrifugation during harvest. An innovative harvest method using a cationic polymer, namely pDADMAC, was investigated for its potential to improve particle separation efficiency through centrifuge by forming larger colloidal particles. This presentation will focus on the implementation of pDADMAC flocculation-based harvest method at large-scale harvest and the identification of key process parameters affecting pDADMAC flocculation performance using small-scale studies.
Process intensification (PI) is a strategy to maximize throughput and reduce operational costs by improving the efficiency of each step in the downstream process. This session will cover the latest techniques in PI, such as multi-column chromatography, hybrid filtration systems, and integrating various purification steps. Attendees will learn how these innovations are driving faster and more cost-effective biologics manufacturing.
Subcutaneous administration of monoclonal antibody therapies offers ease of use, improved patient experience, and the potential for self-administration at home. However, subcutaneous administration often requires high-concentration (≥ 150 mg/mL) drug product (DP). To achieve this, ultrafiltration/diafiltration (UFDF) is implemented as the final step in the purification process for biologics in order to concentrate and buffer exchange the product pool prior to further formulation to DP. Achieving high protein concentrations while maximizing UFDF process efficiency and yield presents significant challenges such as increased processing time, reduced process yield, and potential impact on product quality. This talk will elaborate on key considerations associated with developing and scaling-up UFDF processes including high product pool viscosity, UFDF system pressure constraints, incorporation of additional excipients into the UFDF processes, and formulation strategies.
Surface polysaccharides (Lipopolysaccharides; LPS) of E.coli and Capsular Polysaccharides of Streptococcus pneumoniae located in the outer surface of cell wall of the bacteria are both a virulence factor and a target for protective antibodies. Antibodies against the O-antigen & capsular polysaccharides may confer protection against infections and thus vaccines have been developed by conjugation of O-Antigen & capsular polysaccharides to carrier proteins to enhance immunogenicity. Purification of O-Antigen & Capsular polysaccharides are a critical precursor for production polysaccharide-protein conjugate vaccines. This talk will discuss the purification unit operations employed to obtain pure polysaccharide drug substance and illustrate through examples the successful scale-up and addressing of challenges faced while optimizing the process.
Some ADC products have very low UFDF sieving coefficients necessitating a high, and often prohibitive, number of diavolumes. A novel purification step has been developed to augment a formerly UFDF-based purification process to significantly reduce the diavolumes required. This case study will demonstrate an approach to characterization and validation of this carbon filter based purification of an ADC product.
This case study highlights key challenges and insights drawn from the accelerated development of harvest and downstream purification processes for an early-phase monoclonal antibody. Clinical considerations for this asset motivated both the implementation of an intensified upstream process and the introduction of high-concentration drug substance. This, as well as timeline acceleration, led to key challenges in harvest, the mitigation of PS80 degradation, and ultrafiltration/diafiltration (UF/DF) process development. Harvest engineering addressed both the evolution of upstream conditions and changing equipment capabilities by strategically using development campaigns to sequentially specify process parameters and assess operational robustness. The PS80 degradation challenge was addressed using a broad toolbox of process changes to improve impurity clearance that included capture wash tuning, post-viral inactivation depth filter screening, and polishing step optimization. These modifications were shown to provide sufficiently robust impurity clearance to maintain PS80 stability despite differences in upstream performance among demonstration batches. In parallel, a UF/DF step was developed to supply high-concentration drug substance while multiple formulations were under consideration. Mapping Gibbs-Donnan effects and exploring various operational procedures enabled the project team to maintain flexibility and readily adapt UF/DF process parameters as needed. By addressing these common challenges through technical advancement and cross-functional collaboration, this case study offers insights that may inform the framework for biopharmaceutical process development.
BioPhorum members, through an industry-wide collaboration, have devised a proof-of-concept for controlling product collection during cation exchange (CIEX) bind-and-elute chromatography. Our approach to this task, using real-time multi-angle light scattering (RT-MALS) technology, involves directly monitoring aggregate content - a critical quality attribute (CQA) - and applying a defined breakthrough or cumulative threshold to stop pooling.
The intent of this partnership is to show that direct monitoring of a suitable CQA enables recovery of good material, currently lost due to conservative pooling practices. In addition to yield improvements for this polishing unit operation, the resulting pools should be more consistent batch-to-batch with respect to aggregate content. In this presentation, we share results from Phase I of the project.
MALS technology measures the intensity of scattered light produced by a sample at different angles. The scattered intensity relates to the molar mass and size. Averaged over a sample, the reported value is the weight-average solution molar mass (Mw) which will increase with increasing aggregate content. In our work, the in-line MALS detector reports the change in Mw in real-time allowing us to “see” when the aggregate population starts to break through. This is already an improvement over traditional UV which does not differentiate between protein populations. We could have set a reasonable threshold limit to the Mw signal and used it to trigger stop pooling and moved on with our work; however, the team took a more ambitious approach to use the Mw measurement in a more accurate way to calculate the cumulative aggregate content in the pool and use a cumulative aggregate threshold to trigger stop pooling. The more ambitious approach ensures tighter consistency of aggregate content in pools batch-to-batch, irrespective of peak shape. This approach also reduces the need for conservative pooling since stop pooling is triggered by the actual CQA (pool % HMW).
This talk will focus on specific strategies for removing high-risk HCPs, such as lipases, chromatin, and other species that bind to proteins or cause other processing issues. It will discuss the use of functionalized resins and membranes.
- Initiatives to reduce their carbon footprint and implement recycling programs
Round Table Session 1: Host Cell Proteins (HCPs): The Unseen Challenge
- Focus: Managing HCPs throughout the bioprocessing lifecycle.
- Key Discussion Points:
- HCP Characterization and Detection: Advancements in analytical methods for identifying and quantifying HCPs, including challenges with low-level detection and diverse HCP populations. Discuss the use of orthogonal methods.
- Impact of HCPs on Product Quality: Explore the mechanisms by which HCPs can affect product stability, efficacy, and immunogenicity. Focus on specific examples and case studies.
- HCP Removal Strategies: Evaluate current HCP removal technologies (chromatography, filtration) and discuss emerging approaches (e.g., novel resins, targeted removal). Consider the challenges of removing specific "high-risk" HCPs (lipases, proteases).
- HCP-Related Challenges in Different Product Modalities: Discuss specific HCP challenges associated with different types of biologics (e.g., antibodies, proteins, vaccines, cell therapies).
- Setting HCP Acceptance Criteria: How do we determine appropriate levels of HCPs, and what factors influence these decisions? Discuss the role of regulatory guidance.
- Future Directions in HCP Management: Explore innovative technologies and strategies for improved HCP control and monitoring. Discuss the role of AI/ML in predicting and managing HCP-related risks.
Round Table Session 2: Extractables and Leachables (E&L): Ensuring Patient Safety
- Focus: Understanding, controlling, and mitigating the risks associated with E&L.
- Key Discussion Points:
- E&L Sources and Identification: Discuss common sources of E&L in bioprocessing and packaging, including materials of construction, processing equipment, and packaging components. Explore advanced analytical techniques for identifying and characterizing E&L.
- Risk Assessment and Toxicological Evaluation: How do we assess the potential toxicity of identified E&L? Discuss the use of toxicological studies and risk-based approaches to determine acceptable levels.
- E&L Control Strategies: Explore strategies for minimizing E&L, including material selection, process optimization, and container closure system design. Discuss the importance of supplier qualification and change control.
- Analytical Method Development and Validation: Discuss the challenges of developing and validating robust analytical methods for E&L testing, especially for trace-level contaminants.
- Regulatory Requirements for E&L: Review current regulatory guidelines and expectations for E&L characterization and control. Discuss differences in global regulatory requirements.
- Emerging Trends in E&L Management: Explore new technologies and approaches for E&L analysis and control, including the use of predictive modeling and simulation.
Roundtable Session 3: Polysorbate Degradation: Protecting Product Stability
- Focus: Understanding the mechanisms of polysorbate degradation and developing strategies to mitigate it.
- Key Discussion Points:
- Mechanisms of Polysorbate Degradation: Discuss the various pathways of polysorbate degradation, including hydrolysis, oxidation, and enzymatic degradation (with a focus on lipase contamination).
- Factors Influencing Degradation: Explore the factors that can influence polysorbate degradation, such as pH, temperature, oxygen levels, and the presence of specific HCPs (especially lipases).
- Analytical Methods for Monitoring Degradation: Discuss analytical techniques for detecting and quantifying polysorbate degradation products.
- Strategies for Mitigating Degradation: Explore strategies for preventing or minimizing polysorbate degradation, including process optimization, excipient selection, and the use of inhibitors.
- Impact of Polysorbate Degradation on Product Quality: Discuss the potential consequences of polysorbate degradation on product stability, efficacy, and safety.
- Case Studies of Polysorbate Degradation: Share real-world examples of polysorbate degradation challenges and the solutions implemented.
- Future Directions in Polysorbate Management: Explore new approaches for stabilizing polysorbates and preventing degradation, including the use of novel excipients and formulation strategies. Discuss the potential of more sensitive analytical methods.
This panel will look at emerging technologies in bioprocess chromatography and discuss approaches that have the potential to enhance downstream purification. This would include discussions on continuous chromatography, in-silico modeling technologies and novel chromatography media such as monoliths and membranes.
Advancements in biologics manufacturing and quality control have driven the development of novel technologies aimed at improving efficiency, consistency, and safety in the production of therapeutic biologics. The speaker will present some of the recent development on continuous bioprocessing and real-time analytics. The integration of artificial intelligence (AI) into process optimization and predictive analytics to streamline manufacture operation excellence. The impact of these technologies on scalability, cost-effectiveness, and regulatory compliance is also examined, highlighting their role in meeting the growing demand for biologics.
This session will provide practical advice for bioprocess programs navigating post-approval submissions and late-stage commercialization. A key focus will be post-approval comparability, including strategies for managing situations where materials fall outside of established acceptance criteria. The talk will explore the use of risk assessment.
The biopharmaceutical industry is under constant pressure to expedite early-stage process development to deliver more medicines to patients faster. Despite significant advancements in development and manufacturing workflows, the imperative for speed and efficiency remains paramount. Robotic automation, employing robotic arms and liquid handlers for sample preparation, plate transfers, and assay execution with minimal human intervention, emerges as a potent solution. This technology ensures high throughput and consistent assay outcomes. Over the past decade, the focus has been on automating individual assays with relatively straightforward data management and digitalization requirements. While automation of single assays may not substantially reduce assay time, it allows analysts to multitask, engaging in more creative and productive activities while robots operate autonomously. However, the overall turnaround time often remains unchanged or insignificantly decreased due to inter-assay waiting periods. We have developed an end-to-end, fit-for-purpose integrated robotic automation platform. This platform comprises plug-and-play modular units equipped with various analytical instruments, including fully integrated liquid handling, chromatographic, electrophoretic, and spectroscopic devices. A centralized SCARA (Selective Compliance Assembly Robot Arm) orchestrates sample handling across these instruments. The platform facilitates the parallel execution of multiple analytical assays, enabling users to load samples and walk away. This novel automation platform improves the speed and productivity of in-process analytical testing during early-stage process development of biologics in our pipeline.
We have developed a maturity model for defining tangible stages, requirements and benefits associated with understanding the implementation of the fundamental building blocks to transition from paper-based manual operations to a fully autonomous lights out facility. The aim of the roadmap is to provide guidance on the ecosystem needed to transition proof of concept projects into business as usual processes by synchronizing activities, enable stakeholder management and define the prerequisites needed at each level to realize the benefits on the journey to autonomous lights out facilities. Consequently, the model enables gap identification, prioritization and stage gating of activities to ensure successful integration of new technologies and tools into existing and future factilities.
We discuss how the maturity matrix builds standardization and complements existing assessment models, e.g. Digital Plant Maturity Model, by building on them to provide a holistic overview through considering process, plant, people and planet.
Efficient bioprocess development increasingly relies on smart, model-driven workflows to reduce timelines and experimental burden. By combining AI methods with structured experimental design and iterative learning cycles, it is possible to cut experimental effort by up to 70% while deepening process insight. This talk will explore how such workflows enable faster, data-informed decisions across key development stages - from lab-scale studies to scale-up and fully continuous manufacturing. Industrially relevant use cases will illustrate how integrating predictive models with real-time learning supports robust process definition, reduces uncertainty, and accelerates technology transfer -ultimately enabling more agile and efficient biomanufacturing strategies.
This session will take a grounded approach to the application of AI in biomanufacturing, exploring practical use cases, addressing common misconceptions, and discussing the limitations of current AI technologies.
This talk could address the increasing demand for biologics and discuss innovative solutions for capacity expansion, including modular facilities, process intensification, and improved resource utilization.
Biopharmaceutical landscape is getting increasing complex with a broad spectrum of modalities to meet unmet medical needs, while external geo-political, regulatory, generic competition and sustainability challenges demand acceleration of drug development pipelines. Despite significant advancements in the development and manufacturing workflows for faster and more efficient processes such as intensified and continuous operations, in-process analytical testing is still mainly centric around manual testing. Harnessing the power of automation enables fit-for-purpose and plug-and-play modular units with end-to-end parallel testing of multiple analytical assays with significant improvement in speed and productivity. The concept of robotic process automation (RPA) tools resembles as “Digital Workers” to automatically process, analyze and transcribe analytical results in real time which greatly improves the rapid availability of data to the stakeholders. Complex modalities in the pipeline often require non-platform analytical methods, unique approaches, and upfront investment on method development. Such efforts can be greatly simplified using data interrogation techniques such as Machine Learning and Deep Learning approaches. Herein a vision, roadmap and case studies of automation and digital transformation efforts measured against main KPI’s such as speed and productivity for in-process analytics for biologics are presented.
This talk will address the critical challenges of technology transfer, particularly the data-related bottlenecks that often hinder the scaling of projects from lab to clinical trial and manufacturing. It will explore the role of software solutions in streamlining this process and discuss how modernizing tech transfer practices can improve efficiency, data handling, QA, and overall speed. The presentation will offer practical strategies for overcoming these hurdles and provide insights into what an optimized, modernized tech transfer workflow could entail.
This talk could address the CMC challenges associated with personalized medicines, which often involve small batch sizes, complex manufacturing processes, and stringent quality requirements. It could explore innovative approaches to process development, analytical testing, and supply chain management for these therapies.
This panel will explore the critical role of CDMOs in modern biomanufacturing, covering topics from partner selection and relationship management to navigating regulatory challenges and optimizing for success in a rapidly changing environment
In addition to the development challenges that all clinical stage companies face, CMC teams are also expected to support production of the regulatory filing (IND in US and IMPD for EU/UK). This presentation addresses:
(1) Governing guidance;
(2) Structure/logistics of building these filings;
(3) Logic of content inclusion/exclusion;
(4) Converting an IND into an IMPD. The focus will be on NCE’s, but most practices are portable to Biologics.
The biopharmaceutical industry is moving towards greater flexibility and efficiency in manufacturing. This session will explore the concept of integrated, product-agnostic biomanufacturing facilities – those designed to produce a range of biologics using common equipment and platforms. Attendees will learn how this approach can reduce capital costs, accelerate development timelines, and improve responsiveness to market demands.
- Process development focusses on quality-by-design principles and is often focused on CQA to CPP relationships.
- However, in many bioprocesses, the effect of raw material attributes is the main source for variation.
- The attributes of this variation may be know or also unknown, so how can we produce robustly, by including those effects in the control strategy?
- This contribution proposes digital tools to make the process more robust despite raw material variability
- We propose a plethora of tools, such as using PAT, random effect modelling using linear mixed models, and (Multiple Input Multiple Output) MIMO feedback control tools and End to End Digital twins..
Gain the knowledge and tools to develop robust Target Product Profiles that guide development efforts, ensure alignment with commercial and marketing objectives and maximize the likelihood of a successful product launch and beyond.
With AI amd Machine Learning gainig traction in analytics and process control, many companies are still unclear on the actual benefits of implementation. This session critically examines the real-world applications, separating genuine advancements from overhyped claims. Bringing together industry leaders, AI pioneers, and regulatory experts, the session will discuss successes, failures, and where AI is truly delivering value. It will explore successful AI/ML implementations for process optimization, predictive quality control, streamlined data analysis, and the automation of flow cytometry, how AI-driven computational modeling is reshaping biomanufacturing, and the next five years of AI in analytical sciences and QC. Attendees will gain insights into AI’s current applications and future potential, how it redefines quality control, data analysis, and process optimization, and hurdles that need to be overcome.
Digital transformation is revolutionizing analytical workflows and bioprocess data management. This session explores innovative software solutions, cloud-based platforms, and AI-powered tools that enhance data management, streamline method validation, improve data quality, and enhance analytical lab efficiency. It will showcase solutions to improve data quality, reduce the burden on analytical staff, and improve the data lifecycle. The session will also examine how leading pharma companies are using inline monitoring to reduce manual sample handling and improve data quality and present case studies of real-time sensor applications in bioprocessing. Attendees will discover strategies for improving data quality, streamlining processes, and accelerating digital maturity by integrating real-time data across platforms, emphasizing a systematic data management framework, including the use of machine learning for in-depth process understanding.
This session will delve into the complex regulatory landscape surrounding the use of Artificial Intelligence (AI) and Machine Learning (ML) in the development, manufacturing, and quality control of biologics. Experts will discuss evolving guidelines from regulatory agencies, including best practices for validating AI/ML models, ensuring data integrity, and demonstrating the reliability and transparency of AI/ML-driven decisions, panelists will deliver strategies, improvements in processes, and lower costs of operations through the proper application of the data. Key focus areas include strategies for generating trust and confidence in AI/ML-based analytics for regulatory submissions and maintaining compliance throughout the product lifecycle with specific actionable strategies
High-throughput screening (HTS) and bioassays are transforming biologics characterization, vaccine development, and drug discovery by enabling rapid testing of numerous compounds. This session explores automated platforms, robotics integration, and AI-assisted assay development, demonstrating how companies reduce assay variability, increase throughput, and accelerate time-to-market. It will cover the design of robust in vitro assays, the use of AI and machine learning in flow cytometry, and case studies on successful adoption of membrane-based purification. Attendees will discover how HTS and automated liquid handling speed up drug discovery with improved accuracy and reduced costs, along with AI's ability to predict and optimize AAV vectors for gene therapy. The session will showcase the latest AI-driven innovations in flow cytometry and their impact on drug development and quality control.
Maintaining product comparability throughout biopharmaceutical development is critical for consistent quality and regulatory compliance. Companies often face challenges aligning analytical methods and managing process changes as their understanding deepens. To overcome these hurdles, proactive strategies for early-stage analytical readiness and predictive modeling are key for robust analytical method development. This session will explore strategies for ensuring product comparability across clinical and commercial manufacturing, developing data-driven comparability strategies that integrate machine learning and real-time analytics, bridging offline and online analytical methods, and employing predictive stability modeling to preemptively identify analytical risks. The session will also showcase innovations in method development that aim to reduce validation timelines.
Antibody-drug conjugates (ADCs) represent a significant advancement in targeted cancer therapy, offering the potential to selectively deliver cytotoxic drugs to tumor cells while minimizing systemic toxicity. However, the structural complexity of ADCs, particularly those conjugated through cysteine residues, poses significant analytical challenges. Due to the hydrophobicity of ADCs, Hydrophobic interaction chromatography (HIC) is often the method of choice to analyze the drug-to-antibody ratio (DAR). However, it requires high-concentration salts, which are often incompatible with mass spectrometry (MS) analysis. By employing ammonium acetate as an MS-compatible salt and integrating a 4-way liquid junction cross configuration for simultaneous introduction of the makeup flow and splitting the flow right before coupling to a mass spectrometer, we achieve high-quality separation and sensitive mass spectrometric analysis. This innovative setup allows for simultaneous DAR measurement and positional isomer characterization by switching the makeup flow solvent from water to a denaturation solution. Our method offers a streamlined and effective approach to ADC characterization, facilitating the identification of positional isomers without the need for fractionation or multiple chromatographic steps. The versatility and robustness of this HIC-MS method are demonstrated through the analysis of two ADCs, highlighting its potential for broad application in ADC development and quality control.
The talk will highlight how to approach automation with a tailored mindset, ensuring that solutions are fit-for-purpose and aligned with team-specific needs. A core focus will be on building robust, user-friendly automation scripts that enable broader team adoption without requiring advanced technical skills. Additionally, we share the department’s collective view on automation, detailing the collaborative efforts that are driving this initiative forward.
Gene and cell therapies face quality control, batch release, and regulatory compliance challenges due to the early stage of characterizing their critical quality attributes (CQAs), unlike monoclonal antibodies. As regulators focus on potency, purity, and safety, companies need strong analytical frameworks for CQA monitoring. This session provides a strategy for CQA identification, monitoring, and validation to reduce regulatory obstacles and accelerate approvals. It will cover advancements in defining CQAs for gene therapies, applying learnings from mAb analytical frameworks to cell and gene therapies, and innovations in analytical tools and manufacturing controls to meet evolving regulatory expectations.
As gene therapy evolves beyond AAV-based delivery systems, new analytical challenges arise in ensuring genome integrity, optimizing therapeutic dosing, and mitigating immunogenicity risks. The industry is shifting toward alternative delivery methods such as CRISPR-based therapies, lipid nanoparticles (LNPs), plasmids, and synthetic vectors, requiring advanced analytical approaches to maintain product quality, efficacy, and regulatory compliance. This session will explore: Genome integrity analysis beyond traditional capsid characterization, leveraging long-read sequencing and multiplex digital PCR to assess payload stability and completeness, optimizing viral vectors for complete genomes, reducing dosing requirements, improving patient outcomes, and lowering production costs, second-dose strategies for non-AAV gene therapies, addressing durability, re-dosing challenges, and immunogenicity considerations, emerging analytical approaches for non-AAV delivery systems, including mRNA-based CRISPR therapies, LNP formulations, and synthetic gene vectors, key regulatory considerations for characterizing non-viral gene therapy platforms, ensuring alignment with evolving global standards.
Traditional QC methods often cause bottlenecks due to their slowness, labor intensity, and variability. This session explores cutting-edge automation solutions, high-throughput analytics, and real-time release testing (RTRT) designed to dramatically enhance efficiency and data integrity in biopharmaceutical QC, streamline workflows, and accelerate testing. As gene and cell therapies move to commercial manufacturing, you'll learn how to scale QC processes, ensure assay transferability, and maintain data integrity across production sites. The session will cover implementing RTRT and high-throughput analytics, strategies for harmonizing QC across multiple manufacturing locations, and the rise of plug-and-play analytical tools. Attendees will gain actionable insights for optimizing QC operations, improving process control, ensuring compliance, and reducing risk during commercial expansion. Learn about next-gen QC strategies that increase efficiency, improve consistency, and ensure regulatory compliance while reducing product release bottlenecks.
Traditional analytical methods offer incomplete insights into biologic products. Multi-omics approaches, integrating proteomics, genomics, and metabolomics data, provide a holistic view of product quality and batch consistency. As the industry seeks deeper root cause analysis and process optimization, companies must leverage advanced analytical frameworks for thorough characterization. This session will explore high-resolution mass spectrometry for integrating multi-omics data, next-generation sequencing (NGS) for transforming process characterization, and innovative omics-based analytics for enhanced biologics development. Attendees will gain a clear strategy for leveraging multi-omics for deeper process insights, ensuring data integrity and regulatory compliance, and streamlining process development, with insights from Sartorius on applying omics-based technologies for root cause analytics and overall process understanding.
Discover cutting-edge analytical strategies for characterizing complex next-generation biologics, including LNPs, hypoimmune cell therapies, and CRISPR-based treatments. As cell and gene therapies rapidly evolve, innovative analytical techniques are needed to ensure product quality, accelerate development, and navigate the evolving regulatory landscape. This session explores new tools for characterizing engineered immune cells and gene-edited therapies, the growing role of Next-Generation Sequencing (NGS) and Mass Spectrometry (MS), and the integration of emerging bioanalysis techniques. Attendees will gain actionable insights to tackle the analytical challenges posed by complex biologics and learn about innovations in analytical methodologies for biopharmaceutical characterization, process analytics, and advanced automated peptide synthesis and purification methods
As the biopharma industry rapidly expands beyond monoclonal antibodies, the analytical strategies required for gene therapies, ADCs, and RNA-based biologics must evolve to keep pace. This presentation will explore how industry leaders and regulators are adapting analytical methods to address the unique complexities of next-gen biologics. Attendees will gain insights into the biggest challenges in characterizing these novel modalities, strategies for integrating therapies into traditional biologic systems, regulatory expectations for CQAs, and how to ensure robust analytical development from discovery to commercialization.
As biologics become increasingly complex, ensuring product stability and maintaining potency throughout development presents major challenges. This session will explore novel analytical and formulation strategies for overcoming these hurdles, with a focus on bispecific antibodies, T-cell engagers, and other emerging modalities. A case study will highlight the development of a highly potent bispecific antibody targeting Claudin 6, including the innovative dual-drug lyophilized formulation designed for a Phase I clinical trial. Experts will discuss key analytical and stability challenges encountered, how they were resolved, and best practices for optimizing drug-delivery strategies to enhance clinical safety and product performance.
This interactive session brings together experts from biopharmaceutical companies, technology providers, and regulatory agencies to address the practical challenges of implementing next-generation analytical technologies. The discussion will center on strategies for robust method validation, effective data management, and ensuring compliance with evolving regulatory expectations when adopting techniques like advanced mass spectrometry, Next-Generation Sequencing (NGS), Process Analytical Technology (PAT), and AI/ML-driven analytical tools.
Key discussion points:
- Overcoming the hurdles in validating novel analytical methods for complex biologics.
- Strategies for managing and integrating the large datasets generated by NGS, multi-omics, and other high-throughput technologies.
- Addressing regulatory considerations and demonstrating comparability when transitioning from traditional methods to next-generation approaches.
- Ensuring data integrity and traceability in digitalized analytical workflows.
Bioassays remain one of the most complex and variable analytical methods, making their development, validation, and regulatory approval highly challenging. As the industry moves toward advanced biologics, gene therapies, and ADCs, ensuring potency assay reproducibility and alignment with clinical outcomes is more critical than ever. Emerging technologies—including cell-based assay automation, surrogate potency assays, and advanced statistical modeling—are revolutionizing bioassay design, reducing variability, and enhancing regulatory compliance. This session will provide: Breakthroughs in bioassay development, including automation, surrogate potency assays, and real-time analytics to improve reproducibility and regulatory confidence, new analytical tools enabling the measurement of previously unquantifiable attributes, bridging the gap between characterization and potency outcomes, case studies on linking analytical data to real-world clinical efficacy, ensuring potency assays capture the most relevant product attributes, regulatory considerations for next-generation bioassays, reducing compliance risks and scrutiny during regulatory filings, how AI-driven statistical modeling enhances bioassay accuracy, minimizing variability and streamlining batch release.
The effectiveness of emerging analytical technologies often depends on the specific biotherapeutic modality. This roundtable will provide practical guidance on selecting and applying the most appropriate next-generation analytical tools for different types of biologics, including monoclonal antibodies, ADCs, bispecifics, cell and gene therapies, and mRNA-based therapeutics. Experts will share case studies and best practices for leveraging technologies like mass photometry, digital PCR, advanced chromatography techniques (e.g., SEC-MS, CE-MS), and AI/ML-powered data analysis to address specific analytical challenges in biopharma development and manufacturing.
Key discussion points:
- Matching the right analytical tools to the unique characterization needs of different biotherapeutic modalities (mAbs, ADCs, CGTs, mRNA, etc.).
- Leveraging mass spectrometry-based methods for in-depth characterization of protein structure, post-translational modifications, and impurities.
- Applying NGS for genomic integrity analysis, cell line characterization, and viral safety testing in cell and gene therapy products.
- Utilizing AI/ML for predictive modeling, process optimization, and automated data analysis in bioprocessing and QC.
- Discussing how analytical requirements and the location of analytical physical sites challenging with cell therapies.
Regulatory expectations for potency and release testing are evolving, particularly with the rise of advanced biologics such as CGTs, ADCs, and bispecific antibodies. Companies must navigate challenges in assay standardization, surrogate potency methods, and comparability assessments while ensuring compliance with global regulatory frameworks. This session, led by industry experts and regulatory specialists, will provide practical insights into meeting the latest standards, including ICH Q6B (Specifications for Biotechnological/Biological Products), USP General Chapter <1032> (Design and Development of Biological Assays), and FDA guidance on potency testing for gene therapy products. Attendees will gain actionable strategies for improving assay validation, reducing variability, and streamlining regulatory approval pathways.
Large molecule biotherapeutics are primarily expressed and produced in mammalian host cells. At every step of downstream bioprocess and prior to fill-finish, monitoring residual host cell impurities is critical due to their immunogenic and oncogenic risks. Regulatory agencies mandate levels of residual host cell impurities to be under acceptable limits. A workflow solution to detect low levels of residual impurities with high specificity and reliability is important to meet the critical quality attributes (CQAs) of biotherapeutic products. Here, we introduce high-performance qPCR based residual CHO or HEK293 DNA quantitation and host cell protein monitoring solutions to enable accurate and reliable monitoring and reporting of residual levels of host cell DNA and proteins in large molecule products.
Multi-Attribute Methods (MAM) streamline testing and improve biologics characterization, yet practical case studies showcasing their impact in reducing testing burdens are limited. This session will explore real-world case studies and challenges of implementing MAM in Quality Control (QC) labs, an overview of MAM’s potential to streamline biologics testing and reduce assay burden, and regulatory perspectives on adopting MAM for IND/BLA submissions and batch release testing. Attendees will learn how to ensure a true correlation instead of a coincidence, reduce testing burdens, and navigate the opportunities and hurdles for MAM's wider integration within regulatory frameworks.
Process Analytical Technology (PAT) and AI-driven modeling tools such as AlphaFold are transforming bioprocessing through real-time monitoring, predictive analytics, and automation. Adoption is inconsistent due to concerns about data reliability, regulatory validation, and integration with existing systems. This session explores how companies use PAT to improve batch consistency and reduce deviations, the role of AI in modeling bioprocess outcomes to enhance process control and optimize yields, and the challenges and successes of implementing AI-driven tools like AlphaFold in industrial settings. Attendees will gain a deeper understanding of the potential of PAT and AI and learn how to overcome adoption hurdles to achieve greater efficiency and compliance.
- What to start early in the process?
- How early is too early to consider strategy for scaling up?
- How to define the right commercial scale early in development
- Best practices for locking in scalable processes from the start
- Designing therapies with long-term success in mind—beyond Phase 1
- Key considerations for commercial scale up, reimbursement, raw material availability, and regulatory approvals
- Pathways to developing safe, reimbursable therapies with broad patient access
- How to effectively adopt AI tools in the CGT space
- Opportunities for AI and machine learning in process development & manufacturing
- Real-world applications of AI in CGT pipeline development
- Case study insights on AI-driven innovation
Delivery of plasmid DNA enables systemic expression of therapeutic molecules, including but not limited to monoclonal antibodies, proteins and peptides. However conventional delivery techniques are limited by constraints of redosability and toxicity in their ability to delivery DNA effectively. Polymeric systems can overcome these constraints but have a very large design space. This presentation will describe how machine learning can leverage large design spaces for the design of polymeric delivery vehicles for a broad range of therapeutically relevant molecules in vivo.
Onechain Immunotherapeutics developed a stromal-free bioprocess for the novo generation of γδ T cells from cord blood (CB) CD34+ HSPC or iPSC-derived CD34+, using recombinant Notch ligands, eliminating the need for expanding these cells from peripheral blood or other sources. The process can yield functional γδ T cells that can be armed with CARs with cytotoxic activity against multiple cancer cells. This strategy is highly scalable, consistent with GMP guidelines, and represents a step forward in the field of allogeneic, off-the-shelf CAR-T cell therapies.
Through a series of case studies this session will focus on the latest innovations and advances in next-generation therapies.
- Explore cutting-edge preclinical and clinical case studies driving the future of cell and gene therapy.
- Discover how data-driven innovation is transforming therapy development, from control and targeting to tackling undruggable diseases.
- Unpack the challenges vs. traditional approaches, advancements in new modalities, and the manufacturing needs shaping commercialization of future therapies.
Key Case Studies Areas Include:
- Gamma Delta T Cells & T Cell Engagers
- mRNA, RNA, & NK Therapies
- Multiplex & Combination Cell Therapies
- 3D Bioprinting & Scaffold Development
- Solid Tumours & Expanding Disease Areas (Autoimmune, Neurology, Cardiometabolic)
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
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Join industry experts as we explore the cutting edge of non-viral gene delivery. This dynamic discussion will cover:
- The Non-Viral Advantage – Where does it shine over viral vectors?
- Beyond the Liver – Innovative delivery routes (brain, tissue-specific targeting).
- Next-Gen Delivery Vehicles – How to utilize LNP alternatives (EVs, VLPs etc) and machine learning-powered targeting
- Optimizing Payloads – Single vs. combination payload strategies
- Analytical & Regulatory Insights – Ensuring safety, efficacy, and consistency
- Future Trends – Will in vivo LNP therapies overtake ex vivo approaches?
- Collaboration & Acceleration – How can companies drive non-viral innovation?
Where is non-viral delivery headed? Let’s discuss the breakthroughs shaping the future!
- Internalising or externalising manufacturing – What are the decision points?
- How to select CDMO partners? – Industry criteria for selection
- When / what stage of development should you bring on second CDMO?
- Understanding CDMO capacities, timelines, and capabilities
- Qualification, and quality agreements for commercial manufacturing
- Tech transfer: best practices and risk management
- Strategies for transferring product from development phase to CDMO for large scale production: How to scale up?
- Lessons learnt from transferring to CDMO and vice versa
- Increasing skills and experience in manufacturing of next generation CGT products
- Partnership and communication – how to partner most effectively with CDMOs?
- Overcoming challenges at manufacturing scale
- Applying automation to meet manufacturing end points
- Achieving maximum doses per lot of drug product to drive patient cost down
- Defining point-of-care manufacturing and decentralized manufacturing
- Scaling with consistency: key elements for global standardization
- Tech & innovation: cutting-edge solutions for safe, efficient, and cost-effective decentralized manufacturing
- Overcoming obstacles to decentralised manufacturing to enable access to approved CGTs
- Differences in systems across decentralized cell therapy manufacturing models - how can we harmonize?
- Standardization vs a modular approach
- Equipment, data, and technologies
- Dialogue and collaboration between developer/practitioner to streamline eventual implementation
- Improving worldwide access: how can local manufacturers and supplier help?
- Real world experience with a decentralized cell therapy manufacturing platforms supporting Phase I/II clinical trials
- Cryopreservation scientific principles and its applications in process development
- Development guidance in minimizing variation within and across batches from freezing to shipping
- Scale up challenges in fill-finish/cryopreservation and solutions
Scalable and cost-effective manufacturing of pluripotent stem cells is essential for advancing regenerative medicine and cell therapy. We have developed a commercial-scale, automated, and closed-system expansion platform capable of producing billions of cells from a single vial with a 10-fold cost reduction. Our system integrates real-time, in-line process analytical tools to continuously monitor cell growth and health, ensuring optimal expansion conditions and consistent product quality. Additionally, we have implemented and validated analytical metrics to meet regulatory and commercialization standards. By addressing scalability, automation, and regulatory compliance, our platform significantly reduces manufacturing costs and batch-to-batch inconsistencies and accelerates the path to clinical and commercial applications. This poster presentation will cover the system’s design, analytical validation, and its impact on the future of stem cell manufacturing.
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
The Biophorum ATMP Visible Particles workstream has proposed a holistic, lifecycle approach to reduce and de-risk visible particulates in cell therapy (CT) drug formulations. This involves characterizing and detecting particulates in the manufacturing process and formulation, then improving material quality and process controls to minimize them.
CTs face unique challenges in particulate control and detection compared to other injectables, including difficult-to-inspect formulations and containers. CTs, with inherent cell-related particulates, complicate the detection of other particles. Terminal sterilizing filtration isn't applicable due to cell size and formulation needs. Small batch volumes, especially in autologous therapies, make rejecting units with particle defects critical. Regulatory guidance and health authority expectations are not aligned with the unique characteristics of cell and gene therapies, posing challenges for sponsors in meeting particulate specifications.
- What is required for product characterization by regulatory authorities?
- Use of risk-based assessment to identify product CQAs
- In-process controls, stability, and release testing expectations from authorities
- How analytics can be used to support the move to “the product is the product”
- Development of rapid process characterization assays
You have exciting clinical data, but can the CMC aspects keep up? This presentation will discuss advice and lessons learned from preparing a BLA and supporting the inspection & review process on an accelerated timeline.
- Building with the End in Mind: Key takeaways from embedding market and patient needs from day one.
- Fast-Tracking Development: What a seamless integration of R&D and process development speeds up timelines
- Cost Reduction Innovations: Key drivers in reducing CGT production costs
- Choosing the Best Manufacturing Model: External, internal, or shared capacity—what’s the winning strategy?
- Driving Down CoGs: Game-changing improvements in process, automation, and scaling without compromising on quality.
- Scaling Smart: Navigating the balance between speed, cost, and quality in manufacturing.
- Harnessing Modular & Advanced Tech: How next-gen technologies are supercharging production.
- Analytics & Automation in Action: Tackling batch variability and scaling hurdles head-on.
- Securing the Supply Chain: Mitigating risks in raw materials and critical components.
- Batch Consistency at Scale: Unlocking the secret to flawless, consistent production.
- Scaling for Rare & Common Diseases: Addressing challenges and opportunities
- Cost-Effectiveness for Larger Populations: Balancing cost-cutting strategies with uncompromising quality.
- Best Practices: Reducing CoGs and improving manufacturing efficiency in gene therapies
UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene for the treatment of Wilson Disease. UX701 leverages Ultragenyx’s proprietary producer cell line platform, Pinnacle PCL™, to produce rAAV at 2000L scale.
Process characterization of UX701 is an essential aspect of the process validation lifecycle, aimed at defining manufacturing process ranges to maintain the target product profile (TPP). A risk-based approach was applied to the late-stage development activities for UX701 upstream and downstream process including process parameter and raw materials risk assessment, scale-down model (SDM) qualification, and process characterization (PC) studies, to establish process controls and inform parameter criticality for the upstream and downstream unit operations in the 2000L manufacturing process. We assessed over 400 upstream and downstream process parameters for their impacts to process performance indicators (PIs) and critical quality attributes (CQAs) and classified them into high, medium or low risk parameters. Of these we studied 11 high and medium risk upstream process parameters and 2 raw materials, and 14 high and medium risk downstream process parameters in PC studies.
We identified 8 process parameters in upstream and 3 downstream process parameters to be critical (CPP) or key (KPP). In the upstream process, temperature and pH, seeding density, and helper virus concentration were all found to be CPPs during production. In the downstream process, viral heat inactivation step, temperature and time affect the inactivation kinetics of the helper virus. Characterization of our anion-exchange (AEX) polishing chromatography step identified an upper threshold which the load conductivity should stay below to ensure binding onto the AEX column.
In this work, we have characterized the cell culture and purification manufacturing process and demonstrate a robust and high yielding process for the manufacture of UX701. This is the first PC campaign for our Pinnacle PCL™ rAAV gene therapy manufacturing platform.
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
- Case study examples on potency assay development and validation strategies – What factors/ studies to choose for potency?
- How far do you have to go with regards to potency?
- Functional assays for late-stage programmes
- Requirements for development vs BLA – How to account for BLA requirements from the start?
- Specification requirements to meet regulatory expectations
Feedback received from regulatory authorities on expectations for potency assays
In order to achieve development timelines, rapid process development can result in the sacrifice of capsid recovery in order to meet specifications. By establishing a strategy focused on critical process parameters, leveraging design of experiments strategies, and using statistically sound analytics, genome-containing capsid recovery can be optimized in recombinant AAV downstream unit operations.
Join industry leaders as we explore how AI, digitalization, and advanced analytics are transforming cell and gene therapy manufacturing. This dynamic panel will dive into:
- AI-Powered Manufacturing: How AI-driven models, predictive analytics, and digital twins are optimizing process control
- Process Analytical Technologies (PAT): The latest breakthroughs in real-time monitoring, automation, and data-driven decision-making
- Overcoming Data Challenges: Strategies to build robust AI models despite limited datasets
- Success Stories: Case studies showcasing AI and PAT integration for improved yield, efficiency, and product quality in cell and gene therapy manufacturing and process development
AAV and Lentiviral vectors are widely utilized tools in cell and gene therapy due to their unique characteristics. For different applications, it's crucial to ensure the product's purity and demonstrate the elimination of impurities. CIM® monoliths play a vital role in achieving high purity standards of the final product. When paired with PATfix® on the analytical side, they allow for the assessment of recovery, monitoring of impurity removal, and act as complementary analytics to traditional methods, offering a more comprehensive understanding of the sample's composition.
• Developing formulations that enhance the stability of biologics
• Include the use of excipients, lyophilization, and novel delivery systems
• Case study on challenges with formulating high concentration biologics – particularly emerging biologics
• Improve viscosity to avoid issues with syringe ability and injectability - development of specialized delivery devices
• Optimising buffer systems and excipient usage
• Maintaining homogeneity
• Storage and temperature challenges
Patient convenience and the need for self-administration are fueling the demand for prefilled syringes and autoinjectors for biologics, requiring specialized formulation and fill finish capabilities.
- Early-Stage Considerations: How can formulation scientists and process development teams collaborate early in the development lifecycle to ensure seamless transition to fill finish? What are the key formulation attributes that need to be considered from an aseptic processing perspective?
- Handling Challenging Formulations: What are the best practices for aseptically processing complex formulations (e.g., high concentration, viscous, sensitive biologics)? What innovative technologies or approaches can be employed?
- Impact of Excipients on Aseptic Processing: How do different excipients influence the aseptic process? Are there any excipients that pose particular challenges for sterilization or filtration?
- Single-Use Systems and Formulation Compatibility: How does the increasing adoption of single-use systems in fill finish interact with different biologic formulations? Are there any compatibility considerations?
- Real-World Case Studies: Panelists could share anonymized examples of challenges encountered at the intersection of formulation and aseptic processing and the solutions implemented.
- Future Trends and Innovations: What emerging formulation strategies or fill finish technologies hold promise for improving the integration of these two critical areas?
- Frameworks for evaluating make vs. buy decisions in biopharma.
- Identifying core competencies and areas ripe for outsourcing.
- Balancing control and flexibility in manufacturing choices.
- Case studies illustrating successful outsourcing strategies.
- Leveraging CDMO expertise for specialized technologies and capabilities.
- Mitigating risks and enhancing supply chain resilience through external partners.
- Accelerating speed to market and managing capacity constraints.
- The role of external manufacturing in supporting innovation pipelines.
- Key FDA regulations and guidelines relevant to CDMO partnerships (e.g., 21 CFR Parts 210 & 211).
- Understanding and implementing robust quality agreements.
- Navigating supplier qualification and audit processes.
- Strategies for maintaining compliance throughout the partnership lifecycle.
- Establishing clear communication channels and quality agreements that foster a shared commitment to quality.
- Implementing effective oversight and monitoring systems for CDMO operations.
- Navigating differences in quality cultures and ensuring alignment.
- Leveraging technology and data to enhance quality and compliance in external manufacturing.
- Addressing challenges related to investigations, deviations, and CAPAs across organizations.
- Building trust and transparency as foundational elements of a strong quality partnership.
- Defining clear requirements and objectives in your RFP.
- Key criteria for evaluating potential CDMO partners (e.g., technical capabilities, quality systems, financial stability, cultural fit).
- Best practices for conducting thorough due diligence (technical, quality, financial).
- Utilizing site visits and audits effectively.
Small group discussions focused on specific challenges in CDMO selection, such as:
- Phase-appropriate CDMO selection & finding CDMOs with niche capabilities (e.g., specific cell lines, advanced therapies).
- Balancing cost, quality, and timelines in the selection process.
- Building trust and transparency in the initial stages of partnership.
30min table discussion
10 minute findings feedback to the group for each table
When Tracy Ryan’s daughter Sophie was diagnosed with a relentless brain tumor, it sparked a determined search for a cure. Their journey led to breakthrough NK cell science and the founding of NKore BioTherapeutics. In this keynote, Tracy and Sophie share how their fight inspired a pioneering, non-toxic immunotherapy platform now offering hope to patients worldwide.
- Introduction
- Objectives and outline of course
- Main development stages and regulatory cadre
- The main streams of activity from cell line to drug product
- The CTD for Regulatory filings. Quality Module – overview of main sections
- The importance of CMC : Quality (and Safety and Efficacy)
- Supply
- The pillars of pharmaceutical development
- Criticality Assessment
- Control strategy & Validation
- QBD Principles
- Most relevant regulatory Guidance and where to find it MH
Quiz
- Review of the main streams and initial status
- Broad requirements for Tox and for First in Human Studies
- Cell banking
- Other Raw materials
- Drug Substance Process and Manufacture
- Minimum requirements
- Understanding your process :
- Impurities : identity, clearance, control
- First steps towards a control strategy
- Adventitious contamination and Viral Clearance Studies
- Drug formulation and Drug Product Processing
- Analytical package
- Release methods definition and development
- From method performance to method validation
- In Process Controls (else cover under process?)
- Batch data in the submission
- Product Characterisation and Reference standard
- Stability ( DS and DP)
- Forced degradation studies : necessity and importance
- Why is stability important ?
- Different type of stability studies and typical package for PhI
- Shelf life assignment
- Review of the main streams and initial status
- Broad requirements for Tox and for First in Human Studies
- Cell banking
- Other Raw materials
- Drug Substance Process and Manufacture
- Minimum requirements
- Understanding your process :
- Impurities : identity, clearance, control
- First steps towards a control strategy
- Adventitious contamination and Viral Clearance Studies
- Drug formulation and Drug Product Processing
- Analytical package
- Release methods definition and development
- From method performance to method validation
- In Process Controls (else cover under process?)
- Batch data in the submission
- Product Characterisation and Reference standard
- Stability ( DS and DP)
- Forced degradation studies : necessity and importance
- Why is stability important ?
- Different type of stability studies and typical package for PhI
- Shelf life assignment
- Anticipating the needs on the work streams
- Process Understanding and Design
- Technology Transfer
- Dealing with changes and Comparability
- Gap analyses of the studies available to prepare a Phase 1 submission.
- 4 teams (Analytics, DS, DP, Materials) to cover different parts of case
- 30 – 45 min work in groups : Brief :
- do the existing studies support a Phase 1 file and what are the risks involved?
- Recommendation on eventual additional or superfluous studies
- Map studies / data onto CTD modules (if time)
- 60 min debrief (15 min / group)
- General Conclusions and End of Session
Recent publications have shown that HCPs shorten shelf-life of monoclonal antibodies (mAbs), aggregate or proteolytically cleave mAbs inside the bioreactor and interfere with the binding of mAbs during purification leading to lower purification yield. However, it is a major challenge to remove these HCPs as they are cell-derived impurities. We have developed a novel perfusion filter that can remove dead cells from the bioreactor leading to lower HCP impurities in the cell culture. Our perfusion filter uses the principle of high density inertial microfluidics and is based on a new scientific discovery in our group. We have also developed a numerical model to understand our discovery so that we can optimize the design for higher throughput. This novel perfusion filter is clog-free, can work up to a cell concentration of 10^8 cells/ml and has the ability to remove dead cells from the bioreactor. This technology has been described as disruptive by big biopharmaceutical companies and we will be sharing more about this technology in this presentation
Round Table 1: Scaling CP: From Bench to Plant
- What breaks between lab-scale CP and full-scale implementation?
- What scale counts as “commercially viable” for CP?
- What's missing in current tech to make this easier
Round Table 2: The Business Case for Hybrid and CP Models
- How do you model ROI on continuous or hybrid investments?
- What incentives drive adoption (speed, cost of goods, flexibility)?
- When is it worth revisiting old filings to modernize an existing process?
Round Table 3: Continuous But Flexible: Connected vs Fully Continuous Systems
- Is connected processing a stepping stone or an endpoint?
- When is hybrid actually better than true CP?
Tangential flow filtration (TFF) is an essential cell retention device for the intensified dynamic perfusion cell culture in integrated dynamic bioprocessing (IDB). The demands of the IDB process are high: the TFF must support high cell concentration (>100 million CHO cells/mL) inlarge-scale bioreactors (~2000L) for an extended continuous bioprocess while maintaining relatively high sieving performance (low product retention) and relatively low residence time and mechanical stress on cells. Of these demands, those related to membrane fouling, and the concomitant large facility footprint and increasing operational complexity, remain the most persistent. Through bench scale experimentation and mechanistic modelling, we have found several operational regimes that increase filter capacity. Counterintuitively, our studies suggest that high flux TFF can increase apparent filter capacity by reducing the extent of Starling flow relative to permeate flow, resulting in decreased filter area demand. Utilizing high flux at large scales with sequentially run filters minimizes facility footprint and reduces operational demand during processing, compared to the low flux condition. In this work, we will review high flux, highcapacity data across varied membrane chemistry, vendors, and projects to explore the differences in fouling mechanisms and failure modes at different scales. Additionally, we will review how operational methods can be utilized to swap filters and remove foulants mid processing via TFF regeneration.
- Continuous Biomanufacturing is a clear business case
- However just arranging hardware in a continuous set up will not suffice, because process variability needs to be identified and compensated in real time
- PAT is one solution to measure outputs of each unit operation, however precise measurements are still scarce
- Digital Twins have the power to capture knowledge and to relate inputs and outputs between individual unit operations but also in an End -to End approach. They allow to partly replace PAT.
- This contribution shows different applications to deploy digital twins at single units operations in upstream and downstream processing as well as proposes a real time release approach using end-to end predictions.
This presentation focuses on CMC perspective for monoclonal antibody (mAb) continuous bioprocess (CBP) from design, implementation to manufacturing.
- It is based on the scientific understanding of mAb physicochemical properties, proven bioprocessing principles, available technologies, current industrial practices, regulatory guidelines, challenges, potential solutions, and future perspectives.
- It addresses the conventional, intensified, integral and/or fully automated end-to-end mAb CBP manufacturing process from cell line development (CLD), cell culture process development (e.g., upstream process), protein purification process development (e.g., downstream process), analytical method development, qualification, and validation for process performance and product quality monitoring and control perspectives.
- It also briefly discusses the mAb market demand, demonstrated process consistency and product quality, and potential cost of goods (COGs) reduction.
- Bispecific antibody related proteins are more difficult to produce than monoclonal IgG’s
- A plethora of molecule engineering strategies have been tested over decades with the dual aims of creating a molecule which fits with the desired therapeutic goal and also enabling efficient / practical protein production
- Divergent expression strategies are currently being used: ‘in vitro exchange’ and ‘in cell’ approaches
- Purification (DSP) and analytics pose significant challenges
- Industry wide and UCB examples will be given to illustrate the challenge and current state of the art
Bi- and tri-specific therapeutic antibodies are rapidly emerging, yet achieving optimal safety, efficacy, and manufacturability remains challenging. Using computer-aided design, we’ve developed several multi-specific candidates with favorable preclinical profiles. Notably, one bi-specific and one tri-specific antibody have advanced to Phase I/II trials. We will highlight how AI/ML and CAAD tools can drive next-generation multi-specific antibody design.
- Targeting ligand selection and impact on isotope selection
- Advancements in radiochemistry for improving the safety & efficacy of radiopharmaceuticals
- E.g. use of chelators to attach radioligands to targeting vehicle
- Impacts on manufacturing and quality
- Dealing with variability in radioisotope production
- Implementing robust quality control measures for radioactive materials
- Optimizing manufacturing processes to account for isotope decay
- Strategies for consistent batch-to-batch production
- Considerations for new radioisotopes and indications
- Biodistribution
- Metabolism, Excretions and Decay
- CMC considerations
- Novel radiolabelling techniques and their impact on manufacturing
- Microfluidics in radiopharmaceutical production
- Scaling up production for solid tumor therapies
- Site-specific conjugation methods to enhance stability and targeting of radioligand-based therapies
- Enzymatic conjugation
- Click chemistry
- Optimizing bioconjugation processes for dual-payload ADCs
- E.g. Cysteine conjugation, amino acid incorporation, for payload attachment
- Varying payload incorporation
- Manufacturing considerations for site-specific conjugation
- Analytical method development for complex ADCs
- CMC aspects of regulatory submissions for novel ADCs
- Improving the precision and control of drug attachment in ADC conjugation with site-specific conjugation techniques
- The use of engineered antibodies (e.g. THIOMABs)
- Implementing selenocysteine-containing antibodies
Development of novel conjugation techniques to enhance efficiency and specificity for improved patient outcomes
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- Evaluating challenges specific to early-stage ADC manufacturing
- Highlighting challenges across drug-linker, mAb, DS, and DP manufacturing
- Ensuring effective clinical supply for early-stage manufacturing
- Approaching the challenges and considerations when scaling-up ADC production
- Process Optimization
- Quality Control
- Regulatory compliance
- Strategies to incorporate when scaling up conjugation reactions, purification processing, formulation development
- Implementation of robust quality control measures to ensure purity, potency & stability
- Case study on overcoming scale-up challenges
This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.
Understanding the Tech Transfer Process
Gain a clear roadmap for transitioning projects from laboratory research to large-scale clinical manufacturing.
Identify common challenges and best practices to ensure a smooth and efficient transfer.
Optimizing Scale-Up and Clinical Trial Readiness
Learn strategies for scaling up manufacturing processes efficiently.
Discover methods for ensuring reproducibility and overcoming obstacles in clinical trial preparation.
Navigating Regulatory Compliance and Tech Transfer Challenges
Explore key regulatory considerations in tech transfer.
Understand compliance challenges and how to effectively navigate regulatory requirements.
Enhancing Efficiency and Speed in Tech Transfer
Modernize processes with innovative approaches and new technologies.
Implement strategies to reduce development-to-manufacturing time and accelerate the tech transfer timeline.
Leveraging Automation and Cloud-Based Solutions
Explore the integration of automated tech transfer systems across platforms.
Learn how cloud-based solutions can enhance data transfer and collaboration.
Expert Insights and Practical Advice
Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.
Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.
This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.
Understanding the Tech Transfer Process
Gain a clear roadmap for transitioning projects from laboratory research to large-scale clinical manufacturing.
Identify common challenges and best practices to ensure a smooth and efficient transfer.
Optimizing Scale-Up and Clinical Trial Readiness
Learn strategies for scaling up manufacturing processes efficiently.
Discover methods for ensuring reproducibility and overcoming obstacles in clinical trial preparation.
Navigating Regulatory Compliance and Tech Transfer Challenges
Explore key regulatory considerations in tech transfer.
Understand compliance challenges and how to effectively navigate regulatory requirements.
Enhancing Efficiency and Speed in Tech Transfer
Modernize processes with innovative approaches and new technologies.
Implement strategies to reduce development-to-manufacturing time and accelerate the tech transfer timeline.
Leveraging Automation and Cloud-Based Solutions
Explore the integration of automated tech transfer systems across platforms.
Learn how cloud-based solutions can enhance data transfer and collaboration.
Expert Insights and Practical Advice
Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.
Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.
This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.
Understanding the Tech Transfer Process
Gain a clear roadmap for transitioning projects from laboratory research to large-scale clinical manufacturing.
Identify common challenges and best practices to ensure a smooth and efficient transfer.
Optimizing Scale-Up and Clinical Trial Readiness
Learn strategies for scaling up manufacturing processes efficiently.
Discover methods for ensuring reproducibility and overcoming obstacles in clinical trial preparation.
Navigating Regulatory Compliance and Tech Transfer Challenges
Explore key regulatory considerations in tech transfer.
Understand compliance challenges and how to effectively navigate regulatory requirements.
Enhancing Efficiency and Speed in Tech Transfer
Modernize processes with innovative approaches and new technologies.
Implement strategies to reduce development-to-manufacturing time and accelerate the tech transfer timeline.
Leveraging Automation and Cloud-Based Solutions
Explore the integration of automated tech transfer systems across platforms.
Learn how cloud-based solutions can enhance data transfer and collaboration.
Expert Insights and Practical Advice
Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.
Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.
This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.
Understanding the Tech Transfer Process
Gain a clear roadmap for transitioning projects from laboratory research to large-scale clinical manufacturing.
Identify common challenges and best practices to ensure a smooth and efficient transfer.
Optimizing Scale-Up and Clinical Trial Readiness
Learn strategies for scaling up manufacturing processes efficiently.
Discover methods for ensuring reproducibility and overcoming obstacles in clinical trial preparation.
Navigating Regulatory Compliance and Tech Transfer Challenges
Explore key regulatory considerations in tech transfer.
Understand compliance challenges and how to effectively navigate regulatory requirements.
Enhancing Efficiency and Speed in Tech Transfer
Modernize processes with innovative approaches and new technologies.
Implement strategies to reduce development-to-manufacturing time and accelerate the tech transfer timeline.
Leveraging Automation and Cloud-Based Solutions
Explore the integration of automated tech transfer systems across platforms.
Learn how cloud-based solutions can enhance data transfer and collaboration.
Expert Insights and Practical Advice
Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.
Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.
- Introduction to Biopharmaceutical Life Cycle.
- Explain what upstream bioprocessing involves: the early stages of production, including cell culture and fermentation.
- Outline the key objectives: generating the desired biological product through cell growth and expression.
- Discuss the selection of cell lines (e.g., CHO cells, microbial cells).
- Introduce bioreactors and their role in providing a controlled environment for cell growth.
- Discuss different types of bioreactors (e.g., stirred-tank, wave, single-use) and their applications.
- Explain the fermentation process and its parameters (e.g., pH, temperature, oxygen levels).
- Explain the importance of culture media in supporting cell growth and productivity.
- Describe the process of scaling up from lab-scale to commercial-scale production.
- Highlight current trends in upstream bioprocessing (e.g., single-use technologies, continuous processing).
- Discuss future directions and innovations in the field.
- Explain what downstream bioprocessing involves: the purification and formulation of the biological product after cell culture and fermentation.
- Outline the key objectives: ensuring product purity, quality, and stability.
- Describe the process of harvesting cells or extracellular products from the bioreactor.
- Explain the methods used for cell separation (e.g., centrifugation, filtration).
- Introduce the main purification methods: chromatography, filtration, and precipitation.
- Describe different types of chromatography (e.g., affinity, ion-exchange, size-exclusion) and their applications.
- Explain the principles and applications of ultrafiltration and diafiltration.
- Growing importance of digitalization, AI, and machine learning in the biopharma industry.
- Key pillars of digital transformation in biopharma.
Key Areas of Digitalization
- Data Management and Integration (from Development to Manufacturing).
- Automation and Robotics in bioprocess workflows.
- Real-time Monitoring and Advanced Analytics for process optimization.
Applications in Bioprocessing
- Use of digital twins and AI to optimize upstream and downstream unit operations.
- Role of ML/AI-driven tools for Advanced Therapy Medicinal Products (ATMP) manufacturing.
- AI-driven real-time monitoring, predictive maintenance, and anomaly detection in production lines.
- Simulation-based process development for rapid scale-up.
Challenges and Considerations
- Overcoming data silos and ensuring system interoperability.
- Addressing regulatory requirements for AI and digital tools in GMP environments.
- Ensuring data quality, integrity, and security in digitalized workflows.
- Bridging talent gaps and fostering a digitally skilled workforce.
Case Studies
- Real-world examples of digital transformation in bioprocessing.
- Lessons learned from integrating AI-driven tools in ATMP production.
Future Trends and Directions
- Adoption of Industry 4.0 principles in biopharma manufacturing.
- Emerging technologies such as edge computing and IoT for bioprocessing.
- Sustainability and digitalization: How to?
- Understanding emerging therapies: distinctions between cell therapy, gene therapy, etc.
- Therapeutic potential and current clinical landscape of different emerging therapies, unique challenges and opportunities presented.
- Differences and similarities from ‘traditional’ biologics:
- What learnings can we take from traditional modalities to approach novel modalities?
- Understanding the Cell Therapy and Gene Therapy manufacturing processes.
- Best practices when entering/transitioning into the advanced therapy industry.
- Leveraging experiences from your background into industry.
- Strategies and approaches to best utilise available technologies in the development & production of emerging therapies.
- Moving and translating research from academia, to start up, industry, and beyond.
- Understanding the difference between these, how to transition, pros and cons.
- Lessons and experiences from our panellists.
- The evolution of biopharmaceutical modality
- Analytical methods and their purpose in biopharmaceutical development and manufacturing
- Analytical method development and validation
- Product physicochemical characterization - high-performance liquid chromatography (HPLC), capillary electrophoresis (CE), spectroscopy, imaging, and post-translational modification (PTM)
- Product biological assays - cell-based assays (CBA), enzyme-linked immunosorbent assays (ELISA), and potency assays
- Microbiological contaminants - sterility testing, endotoxin testing, and microbial limits testing
- Process impurity testing - host cell DNA, host cell proteins, chromatography ligand
- Role of quality control (QC) and quality assurance (QA) in biopharma
- Case studies and industry examples
- Latest and future advancements in analytical methods and quality assurance
- The evolution of biopharmaceutical modality
- Analytical methods and their purpose in biopharmaceutical development and manufacturing
- Analytical method development and validation
- Product physicochemical characterization - high-performance liquid chromatography (HPLC), capillary electrophoresis (CE), spectroscopy, imaging, and post-translational modification (PTM)
- Product biological assays - cell-based assays (CBA), enzyme-linked immunosorbent assays (ELISA), and potency assays
- Microbiological contaminants - sterility testing, endotoxin testing, and microbial limits testing
- Process impurity testing - host cell DNA, host cell proteins, chromatography ligand
- Role of quality control (QC) and quality assurance (QA) in biopharma
- Case studies and industry examples
- Latest and future advancements in analytical methods and quality assurance
Round off your day at BPI School with an interactive and insightful discussion:
- Learnings of the day and a chance to recap:
- Key trends and challenges in biopharmaceutical manufacturing, upstream and downstream.
- The impact of digitalization on the future of biopharma.
- Emerging therapies: Opportunities and obstacles.
- How to utilize and implement analytics tools
- Q&A and interactive discussion with industry experts.
- Bridging the gap between theory and practice in biopharma.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
- Which CGT modalities, disease areas, and technologies are top priorities for big pharma investment?
- How major players are integrating CGT into wider R&D pipelines
- Key breakthroughs shaping pharma’s next moves in CGT
- Allocating R&D investment: Autologous vs. Allogeneic programmes
- Snapshot into the investment landscape for CGT products and enabling technologies for 2025/ 2026 and beyond
- How to drive investment in CGT?
- Positioning for success in a competitive funding environment: What do investors need to see?
- “Ask the Investor” interactive Q&A open discussion with our investor panel to get tips and tricks when pitching and their views on where innovation lies in the CGT field
- Case Studies: Success stories of regulatory approvals and commercialization
- Overcoming Hurdles: Key challenges from R&D to approval—and how to tackle them
- Scaling Up: Strategies for manufacturing, validation, and CMC data packages
- Regulatory Insights: Feedback from authorities on characterization, specifications, and approval pathways
- Post-Approval Management: Long-term data tracking, durability studies, and lifecycle strategies
- End-to-End Logistics: Mastering supply chain and global distribution
- Pathways to approval –Case study on the journey from discovery to commercial development and beyond
At Fujifilm Diosynth Biotechnologies, our teams understand that along with cutting edge science there is trepidation in choosing a partner. When your technology and science is advancing quicker than CMDOs can build experience, it can be hard to feel confident that a contract manufacture can get it right. That is why we have created an early access program which builds a strategy alongside our customers, establishing a roadmap to manufacturing through quicker data generation and collaborative consulting mentality. By showing our customers that we can reproduce their results, optimize their process, or simply grow their cell type, our teams build trust and provide a data set that customers can leverage to raise capital, show their leadership, and design the tech transfer that meets all of their program goals.
Through a series of presentations, case studies, interactive discussion and exercises this workshop will delve deeply into the underlying science of developing potency assays.
Some of the topics to be discussed include:
- Overview of what potency is, and what it is not
- Considerations when qualifying and validating bioassays
- From initial ideas, through characterisation to the final potency method
- Clinical correlation
- Regulator objections and why
- Examples of potency assays for approved products
- Examples of a cell and gene therapy potency assays for various product types
Some of the topics to be discussed include:
- Overview of what potency is, and what it is not
- Considerations when qualifying and validating bioassays
- From initial ideas, through characterisation to the final potency method
- Clinical correlation
- Regulator objections and why
- Examples of potency assays for approved products
- Examples of a cell and gene therapy potency assays for various product types
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
- CDMO Selection: Models and Process
- Contract and Quality Agreement Negotiation
- Tech Transfer: Best practices and risk management for external tech transfers
- Digital Transformation in Tech Transfer
- Quality Assurance and Quality Control
- Life Cycle & Relationship Management
Some of the topics to be discussed include:
- CDMO Selection: Models and Process
- Contract and Quality Agreement Negotiation
- Tech Transfer: Best practices and risk management for external tech transfers
- Digital Transformation in Tech Transfer
- Quality Assurance and Quality Control
- Life Cycle & Relationship Management
- What to start early in the process?
- How early is too early to consider strategy for scaling up?
- How to define the right commercial scale early in development
- Best practices for locking in scalable processes from the start
- Designing therapies with long-term success in mind—beyond Phase 1
- Key considerations for commercial scale up, reimbursement, raw material availability, and regulatory approvals
- Pathways to developing safe, reimbursable therapies with broad patient access
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
- Cutting-edge technologies for smarter, more precise therapeutic delivery
- Strategies to minimize off-target effects and boost efficacy
- Cost-effective enabling innovations for scalable implementation
- Optimized engineering approaches for enhanced targeting
- Real-world case study showcasing successful applications
Onechain Immunotherapeutics developed a stromal-free bioprocess for the novo generation of γδ T cells from cord blood (CB) CD34+ HSPC or iPSC-derived CD34+, using recombinant Notch ligands, eliminating the need for expanding these cells from peripheral blood or other sources. The process can yield functional γδ T cells that can be armed with CARs with cytotoxic activity against multiple cancer cells. This strategy is highly scalable, consistent with GMP guidelines, and represents a step forward in the field of allogeneic, off-the-shelf CAR-T cell therapies.
- Integrating novel editing approaches – base, primer editing etc.
- In vivo gene editing
- Beyond current limits – emerging solutions for larger, more complex payload
- Addressing the gaps of established editing technologies– precision editing, single-strand cutting & beyond
- Therapeutic advancements – correcting genetic defects & enhancing therapies
- Case study– transitioning to scalable in vivo applications
Satellite Bio is aiming to treat liver disease with a hepatocyte cell therapy. In this presentation, we will discuss the development of a robust scalable process for expanding primary human hepatocytes while preserving their identity and function. By leveraging heterocellular aggregation with stromal cells, we also developed cryopreserved “seed” constructs that maintain viability and are reproducibly functional post-thaw.
Through a series of case studies this session will focus on the latest innovations and advances in next-generation therapies.
Explore cutting-edge preclinical and clinical case studies driving the future of cell and gene therapy.
Discover how data-driven innovation is transforming therapy development, from control and targeting to tackling undruggable diseases.
Unpack the challenges vs. traditional approaches, advancements in new modalities, and the manufacturing needs shaping commercialization of future therapies.
Key Case Studies Areas Include:
- Gamma Delta T Cells & T Cell Engagers
- mRNA, RNA, & NK Therapies
- Multiplex & Combination Cell Therapies
- 3D Bioprinting & Scaffold Development
- Solid Tumours & Expanding Disease Areas (Autoimmune, Neurology, Cardiometabolic)
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
- Internalising or externalising manufacturing – What are the decision points?
- How to select CDMO partners? – Industry criteria for selection
- When / what stage of development should you bring on second CDMO?
- Understanding CDMO capacities, timelines, and capabilities
- Qualification, and quality agreements for commercial manufacturing
- Tech transfer: best practices and risk management
- Strategies for transferring product from development phase to CDMO for large scale production: How to scale up?
- Lessons learnt from transferring to CDMO and vice versa
- Increasing skills and experience in manufacturing of next generation CGT products
- Partnership and communication – how to partner most effectively with CDMOs?
- Overcoming challenges at manufacturing scale
- Applying automation to meet manufacturing end points
- Achieving maximum doses per lot of drug product to drive patient cost down
- Precision genome engineering with chRDNA technology
- Strategies for armoring allogeneic cells to enhance potency and durability
- Scaling CAR-T production to broaden patient access
Spotlight Presentation – Calling all Technology Thought Leaders!
Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.
Contact us today to learn more: Partners@informaconnectls.com
- Defining point-of-care manufacturing and decentralized manufacturing
- Scaling with consistency: key elements for global standardization
- Tech & innovation: cutting-edge solutions for safe, efficient, and cost-effective decentralized manufacturing
- Overcoming obstacles to decentralised manufacturing to enable access to approved CGTs
- Differences in systems across decentralized cell therapy manufacturing models - how can we harmonize?
- Standardization vs a modular approach
- Equipment, data, and technologies
- Dialogue and collaboration between developer/practitioner to streamline eventual implementation
- Improving worldwide access: how can local manufacturers and supplier help?
- Real world experience with a decentralized cell therapy manufacturing platforms supporting Phase I/II clinical trials
- Cryopreservation scientific principles and its applications in process development
- Development guidance in minimizing variation within and across batches from freezing to shipping
- Scale up challenges in fill-finish/cryopreservation and solutions
A Representative from Single Use Support
The Biophorum ATMP Visible Particles workstream has proposed a holistic, lifecycle approach to reduce and de-risk visible particulates in cell therapy (CT) drug formulations. This involves characterizing and detecting particulates in the manufacturing process and formulation, then improving material quality and process controls to minimize them.
CTs face unique challenges in particulate control and detection compared to other injectables, including difficult-to-inspect formulations and containers. CTs, with inherent cell-related particulates, complicate the detection of other particles. Terminal sterilizing filtration isn't applicable due to cell size and formulation needs. Small batch volumes, especially in autologous therapies, make rejecting units with particle defects critical. Regulatory guidance and health authority expectations are not aligned with the unique characteristics of cell and gene therapies, posing challenges for sponsors in meeting particulate specifications.
A Representative from Solvias
You have exciting clinical data, but can the CMC aspects keep up? This presentation will discuss advice and lessons learned from preparing a BLA and supporting the inspection & review process on an accelerated timeline.
- Building with the End in Mind: Key takeaways from embedding market and patient needs from day one.
- Fast-Tracking Development: What a seamless integration of R&D and process development speeds up timelines
- Cost Reduction Innovations: Key drivers in reducing CGT production costs
- Choosing the Best Manufacturing Model: External, internal, or shared capacity—what’s the winning strategy?
- Driving Down CoGs: Game-changing improvements in process, automation, and scaling without compromising on quality.
- Scaling Smart: Navigating the balance between speed, cost, and quality in manufacturing.
- Harnessing Modular & Advanced Tech: How next-gen technologies are supercharging production.
- Analytics & Automation in Action: Tackling batch variability and scaling hurdles head-on.
- Securing the Supply Chain: Mitigating risks in raw materials and critical components.
- Batch Consistency at Scale: Unlocking the secret to flawless, consistent production.
- Scaling for Rare & Common Diseases: Addressing challenges and opportunities
- Cost-Effectiveness for Larger Populations: Balancing cost-cutting strategies with uncompromising quality.
- Best Practices: Reducing CoGs and improving manufacturing efficiency in gene therapies.
Spotlight Presentation – Calling all Thought Leaders!
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In the rapidly evolving field of gene therapy, technology transfer for a production process from research or development to cGMP manufacturing is a crucial activity that requires effective communication from planning stages throughout execution. This presentation will explore the general approach for technology transfer to Contract Manufacturers, with a specific focus on best practices for the gene therapy sector.
- Improvements and innovations in cell line development & engineering to increase yields for gene therapies
- Application of producer cell lines to lower transfection vector costs: Data for large scale use of stable cell lines
- Scalability of producer cell lines? Case studies on experiences at larger scales
UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene for the treatment of Wilson Disease. UX701 leverages Ultragenyx’s proprietary producer cell line platform, Pinnacle PCL™, to produce rAAV at 2000L scale.
Process characterization of UX701 is an essential aspect of the process validation lifecycle, aimed at defining manufacturing process ranges to maintain the target product profile (TPP). A risk-based approach was applied to the late-stage development activities for UX701 upstream and downstream process including process parameter and raw materials risk assessment, scale-down model (SDM) qualification, and process characterization (PC) studies, to establish process controls and inform parameter criticality for the upstream and downstream unit operations in the 2000L manufacturing process. We assessed over 400 upstream and downstream process parameters for their impacts to process performance indicators (PIs) and critical quality attributes (CQAs) and classified them into high, medium or low risk parameters. Of these we studied 11 high and medium risk upstream process parameters and 2 raw materials, and 14 high and medium risk downstream process parameters in PC studies.
We identified 8 process parameters in upstream and 3 downstream process parameters to be critical (CPP) or key (KPP). In the upstream process, temperature and pH, seeding density, and helper virus concentration were all found to be CPPs during production. In the downstream process, viral heat inactivation step, temperature and time affect the inactivation kinetics of the helper virus. Characterization of our anion-exchange (AEX) polishing chromatography step identified an upper threshold which the load conductivity should stay below to ensure binding onto the AEX column.
In this work, we have characterized the cell culture and purification manufacturing process and demonstrate a robust and high yielding process for the manufacture of UX701. This is the first PC campaign for our Pinnacle PCL™ rAAV gene therapy manufacturing platform.
A Representative from Intertek
- Case study examples on potency assay development and validation strategies – What factors/ studies to choose for potency?
- How far do you have to go with regards to potency?
- Functional assays for late-stage programmes
- Requirements for development vs BLA – How to account for BLA requirements from the start?
- Specification requirements to meet regulatory expectations
- Feedback received from regulatory authorities on expectations for potency assays
This talk will cover application of novel technologies to cell lysis, clarification and enrichment of full capsids by removing empty and partial capsids in AAV purification processes.
What does MVM infectivity in HEK cells mean for your viral safety profile? Here we discuss detectability of MVM using adherent HEK cells and how to integrate MVM detection into your AAV Viral Safety Strategy.
Host cell DNA (hcDNA) levels in AAV-based in vivo gene therapies have substantial implications for a product’s safety profile, however the current industry baseline is very broad due to order-of-magnitude differences in dose level from product to product. To obtain a more valuable baseline, members of a BioPhorum industry collaboration were surveyed to assess normalized hcDNA levels to establish a range for current manufacturing practices. Based on the BioPhorum industry survey, health authority guidelines, and literature, this presentation will cover the challenges with hcDNA and summarize the survey results, including current practices from industry on hcDNA analysis, control and reporting. An approach for harmonized reporting of hcDNA for AAV, based on normalization, will be proposed, with the collective benefits outlined.
Join us for an unforgettable evening of networking, innovation, and discovery at the iconic Museum of Science, Boston! Mingle with fellow cell and gene therapy professionals over delicious food and drinks while exploring fascinating museum exhibits. Engage in lively conversations, make valuable connections, and immerse yourself in an inspiring atmosphere where science and industry meet. This exclusive event offers the perfect blend of socializing and discovery—don’t miss this chance to connect, unwind, and experience the wonders of science in a truly unique setting!
Wrap up an insightful first day of the conference with a relaxed networking session over drinks and nibbles. Join fellow cell and gene therapy professionals, industry leaders, and innovators for an evening of meaningful conversations, idea-sharing, and collaboration opportunities. Whether you're looking to strengthen existing connections or forge new ones, this informal gathering provides the perfect setting to engage with peers in a friendly and welcoming atmosphere. Don't miss this chance to unwind, expand your network, and set the stage for productive discussions on Day Two!
The CGT C-Level Forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences between executives of ~20 CGT therapeutic development companies. The forum is designed to foster new ideas and create new relationships between attendees so that they can leave with tangible outcomes and that will drive their businesses and the sector forward.
An exclusive invite-only session, in a closed room setting, the forum will include 2-3 case studies discussing the right approach to engage and partner with pharma and industry. It will also include general discussion focussed on brainstorming solutions to the most pressing and current challenges of CEO/CSO/CBO/CMOs in the CGT sector.
The CGT Supply Chain forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences between supply chain executives of 20-30 CGT therapeutic development companies.
An exclusive invite-only session, the forum will include 2-3 case studies and panel discussions focussed on brainstorming solutions to navigate the complexities of supply chain management for CGTs. Offering comprehensive insights into innovations, tools and trends to enhance efficiency, visibility, and responsiveness across the entire network and ultimately increasing CGT access to patients.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at Cell and Gene Therapy International.
Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.
This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.
Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.
- Which CGT modalities, disease areas, and technologies are top priorities for big pharma investment?
- How major players are integrating CGT into wider R&D pipelines
- Key breakthroughs shaping pharma’s next moves in CGT
- Allocating R&D investment: Autologous vs. Allogeneic programmes
- Snapshot into the investment landscape for CGT products and enabling technologies for 2025/ 2026 and beyond
- How to drive investment in CGT?
- Positioning for success in a competitive funding environment: What do investors need to see?
- “Ask the Investor” interactive Q&A open discussion with our investor panel to get tips and tricks when pitching and their views on where innovation lies in the CGT field
Join industry leaders as we explore how AI, digitalization, and advanced analytics are transforming cell and gene therapy manufacturing. This dynamic panel will dive into:
- AI-Powered Manufacturing: How AI-driven models, predictive analytics, and digital twins are optimizing process control
- Process Analytical Technologies (PAT): The latest breakthroughs in real-time monitoring, automation, and data-driven decision-making
- Overcoming Data Challenges: Strategies to build robust AI models despite limited datasets
Success Stories: Case studies showcasing AI and PAT integration for improved yield, efficiency, and product quality in cell and gene therapy manufacturing and process development
- Case Studies: Success stories of regulatory approvals and commercialization
- Overcoming Hurdles: Key challenges from R&D to approval—and how to tackle them
- Scaling Up: Strategies for manufacturing, validation, and CMC data packages
- Regulatory Insights: Feedback from authorities on characterization, specifications, and approval pathways
- Post-Approval Management: Long-term data tracking, durability studies, and lifecycle strategies
- End-to-End Logistics: Mastering supply chain and global distribution
- Pathways to approval –Case study on the journey from discovery to commercial development and beyond