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.
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.
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
This workshop is designed to equip biomanufacturers with the knowledge and tools needed to optimize continuous processing. It will cover:
- Core Principles:
- Automating and controlling continuous processes to reduce costs, improve quality, and enhance efficiency.
- Navigating regulatory hurdles and implementation challenges specific to continuous manufacturing.
- Leveraging process intensification to maximize efficiency and ROI.
- Practical Application:
- Real-world case studies and examples of successful continuous processing implementations.
- Interactive problem-solving sessions to address specific challenges.
- Detailed risk assessment and mitigation strategies.
- Advanced Topics:
- Sustainability considerations within continuous biomanufacturing.
- Future trends and emerging technologies in the field.
- Data management and digitalization strategies for continuous processes.
- Expert Interaction:
- Panel discussions and Q&A sessions with industry experts.
- Networking opportunities to foster connections and collaboration.
The workshop aims to provide a comprehensive understanding of continuous processing, from fundamental principles to advanced applications, enabling attendees to drive innovation and achieve bioprocess excellence.
Key Speakers:
- Shireen Goh, Associate Professor, Singapore University of Technology & Design
- Stefano Menegatti, Associate Professor, North Carolina State University
- Anticipating the needs on the work streams
- Process Understanding and Design
- Technology Transfer
- Dealing with changes and Comparability
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.
- 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
- 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
- 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
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.
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
• 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
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.
This workshop is designed to equip biomanufacturers with the knowledge and tools needed to optimize continuous processing. It will cover:
- Core Principles:
- Automating and controlling continuous processes to reduce costs, improve quality, and enhance efficiency.
- Navigating regulatory hurdles and implementation challenges specific to continuous manufacturing.
- Leveraging process intensification to maximize efficiency and ROI.
- Practical Application:
- Real-world case studies and examples of successful continuous processing implementations.
- Interactive problem-solving sessions to address specific challenges.
- Detailed risk assessment and mitigation strategies.
- Advanced Topics:
- Sustainability considerations within continuous biomanufacturing.
- Future trends and emerging technologies in the field.
- Data management and digitalization strategies for continuous processes.
- Expert Interaction:
- Panel discussions and Q&A sessions with industry experts.
- Networking opportunities to foster connections and collaboration.
The workshop aims to provide a comprehensive understanding of continuous processing, from fundamental principles to advanced applications, enabling attendees to drive innovation and achieve bioprocess excellence.
Key Speakers:
- Shireen Goh, Associate Professor, Singapore University of Technology & Design
- Stefano Menegatti, Associate Professor, North Carolina State University
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 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.
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
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 a Cell and Gene Therapy 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
• Addressing supply chain disruptions: strategies for procurement, digitalization, and raw material management
• Sustainability challenges and cost-saving innovations for large-scale media production
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.
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.
As biopharmaceutical production scales, the environmental footprint of downstream processing becomes a growing concern. This talk will explore sustainable practices in downstream processing, including waste reduction, energy-efficient processes, and minimizing the use of toxic solvents. Techniques like recyclable chromatography resins, green solvents, and waste stream recycling will be discussed to promote sustainability in the industry.
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
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.
- 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?
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!
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
The latest trends in biomanufacturing facility design, including the integration of single-use systems and automation to achieve greater flexibility, efficiency, and cost-effectiveness. This session will address how to bridge the gap between islands of automation and fully integrated plants.
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
- 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.
This session will provide insights into the new ICH guidelines on analytical profiles (Q14), including the ATP document, requirements for methods, and the shift to different validation approaches. The talk will cover long-term monitoring, case studies of switching methods or technologies, and how to meet acceptance criteria or update analytical technology.
- 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.
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.
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.
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
• Strategies to maintain minimum pressure to prevent viral particle penetration
• Practical considerations for implementing viral filtration in continuous processing
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.
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.
- 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
This session will cover the key steps in developing a scalable and efficient downstream process for biologics production. Topics will include translating small-scale laboratory processes to pilot and commercial scales, selecting the appropriate purification techniques, and ensuring robust, reproducible processes that meet regulatory requirements. Case studies will be presented to illustrate the challenges faced during scale-up and how they were overcome.
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
This workshop is designed to equip biomanufacturers with the knowledge and tools needed to optimize continuous processing. It will cover:
- Core Principles:
- Automating and controlling continuous processes to reduce costs, improve quality, and enhance efficiency.
- Navigating regulatory hurdles and implementation challenges specific to continuous manufacturing.
- Leveraging process intensification to maximize efficiency and ROI.
- Practical Application:
- Real-world case studies and examples of successful continuous processing implementations.
- Interactive problem-solving sessions to address specific challenges.
- Detailed risk assessment and mitigation strategies.
- Advanced Topics:
- Sustainability considerations within continuous biomanufacturing.
- Future trends and emerging technologies in the field.
- Data management and digitalization strategies for continuous processes.
- Expert Interaction:
- Panel discussions and Q&A sessions with industry experts.
- Networking opportunities to foster connections and collaboration.
The workshop aims to provide a comprehensive understanding of continuous processing, from fundamental principles to advanced applications, enabling attendees to drive innovation and achieve bioprocess excellence.
Key Speakers:
- Shireen Goh, Associate Professor, Singapore University of Technology & Design
- Stefano Menegatti, Associate Professor, North Carolina State University
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.
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.
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
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
- 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
- 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
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.
- 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
• 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?
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)
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)
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 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 talk could address the increasing demand for biologics and discuss innovative solutions for capacity expansion, including modular facilities, process intensification, and improved resource utilization.
- 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
• Integrating in silico models for process development
• Using digital twins and ML to generate predictive results without experiments
• High-throughput screening and model refinement for improved accuracy
- 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
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
- 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 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 is designed to equip biomanufacturers with the knowledge and tools needed to optimize continuous processing. It will cover:
- Core Principles:
- Automating and controlling continuous processes to reduce costs, improve quality, and enhance efficiency.
- Navigating regulatory hurdles and implementation challenges specific to continuous manufacturing.
- Leveraging process intensification to maximize efficiency and ROI.
- Practical Application:
- Real-world case studies and examples of successful continuous processing implementations.
- Interactive problem-solving sessions to address specific challenges.
- Detailed risk assessment and mitigation strategies.
- Advanced Topics:
- Sustainability considerations within continuous biomanufacturing.
- Future trends and emerging technologies in the field.
- Data management and digitalization strategies for continuous processes.
- Expert Interaction:
- Panel discussions and Q&A sessions with industry experts.
- Networking opportunities to foster connections and collaboration.
The workshop aims to provide a comprehensive understanding of continuous processing, from fundamental principles to advanced applications, enabling attendees to drive innovation and achieve bioprocess excellence.
Key Speakers:
- Shireen Goh, Associate Professor, Singapore University of Technology & Design
- Stefano Menegatti, Associate Professor, North Carolina State University
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
Round Table Session 1: Winning in the Biosimilar Market: Strategies for Cost-Effective Manufacturing and Competitive Advantage
- Cost-Effective Manufacturing Strategies: Learn practical strategies for reducing manufacturing costs, including process optimization, efficient raw material sourcing, and streamlined operations.
- Efficient Intensification Techniques: Discover how to implement process intensification to increase productivity and reduce costs without compromising product quality.
- Real-World Examples of Cost Reduction
- Gaining a competitive edge
Round Table Session 2: Maximizing mAb Profitability: Optimization Strategies for a Competitive Landscape
- mAb Process Optimization: Explore techniques for optimizing mAb manufacturing processes to improve efficiency, yield, and product quality.
- Cost Reduction Strategies: Identify and implement effective cost reduction measures across the mAb manufacturing lifecycle, from raw materials to final product.
- Process Improvements for Enhanced Productivity: Discover process improvements that can lead to increased productivity and shorter timelines.
- Staying Ahead of the Curve: Gain insights into emerging technologies and best practices that can help you stay ahead of the competition in mAb manufacturing.
- 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
- 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
This workshop is designed to equip biomanufacturers with the knowledge and tools needed to optimize continuous processing. It will cover:
- Core Principles:
- Automating and controlling continuous processes to reduce costs, improve quality, and enhance efficiency.
- Navigating regulatory hurdles and implementation challenges specific to continuous manufacturing.
- Leveraging process intensification to maximize efficiency and ROI.
- Practical Application:
- Real-world case studies and examples of successful continuous processing implementations.
- Interactive problem-solving sessions to address specific challenges.
- Detailed risk assessment and mitigation strategies.
- Advanced Topics:
- Sustainability considerations within continuous biomanufacturing.
- Future trends and emerging technologies in the field.
- Data management and digitalization strategies for continuous processes.
- Expert Interaction:
- Panel discussions and Q&A sessions with industry experts.
- Networking opportunities to foster connections and collaboration.
The workshop aims to provide a comprehensive understanding of continuous processing, from fundamental principles to advanced applications, enabling attendees to drive innovation and achieve bioprocess excellence.
Key Speakers:
- Shireen Goh, Associate Professor, Singapore University of Technology & Design
- Stefano Menegatti, Associate Professor, North Carolina State University
This workshop is designed to equip biomanufacturers with the knowledge and tools needed to optimize continuous processing. It will cover:
- Core Principles:
- Automating and controlling continuous processes to reduce costs, improve quality, and enhance efficiency.
- Navigating regulatory hurdles and implementation challenges specific to continuous manufacturing.
- Leveraging process intensification to maximize efficiency and ROI.
- Practical Application:
- Real-world case studies and examples of successful continuous processing implementations.
- Interactive problem-solving sessions to address specific challenges.
- Detailed risk assessment and mitigation strategies.
- Advanced Topics:
- Sustainability considerations within continuous biomanufacturing.
- Future trends and emerging technologies in the field.
- Data management and digitalization strategies for continuous processes.
- Expert Interaction:
- Panel discussions and Q&A sessions with industry experts.
- Networking opportunities to foster connections and collaboration.
The workshop aims to provide a comprehensive understanding of continuous processing, from fundamental principles to advanced applications, enabling attendees to drive innovation and achieve bioprocess excellence.
Key Speakers:
- Shireen Goh, Associate Professor, Singapore University of Technology & Design
- Stefano Menegatti, Associate Professor, North Carolina State University
This workshop is designed to equip biomanufacturers with the knowledge and tools needed to optimize continuous processing. It will cover:
- Core Principles:
- Automating and controlling continuous processes to reduce costs, improve quality, and enhance efficiency.
- Navigating regulatory hurdles and implementation challenges specific to continuous manufacturing.
- Leveraging process intensification to maximize efficiency and ROI.
- Practical Application:
- Real-world case studies and examples of successful continuous processing implementations.
- Interactive problem-solving sessions to address specific challenges.
- Detailed risk assessment and mitigation strategies.
- Advanced Topics:
- Sustainability considerations within continuous biomanufacturing.
- Future trends and emerging technologies in the field.
- Data management and digitalization strategies for continuous processes.
- Expert Interaction:
- Panel discussions and Q&A sessions with industry experts.
- Networking opportunities to foster connections and collaboration.
The workshop aims to provide a comprehensive understanding of continuous processing, from fundamental principles to advanced applications, enabling attendees to drive innovation and achieve bioprocess excellence.
Key Speakers:
- Shireen Goh, Associate Professor, Singapore University of Technology & Design
- Stefano Menegatti, Associate Professor, North Carolina State University
This workshop is designed to equip biomanufacturers with the knowledge and tools needed to optimize continuous processing. It will cover:
- Core Principles:
- Automating and controlling continuous processes to reduce costs, improve quality, and enhance efficiency.
- Navigating regulatory hurdles and implementation challenges specific to continuous manufacturing.
- Leveraging process intensification to maximize efficiency and ROI.
- Practical Application:
- Real-world case studies and examples of successful continuous processing implementations.
- Interactive problem-solving sessions to address specific challenges.
- Detailed risk assessment and mitigation strategies.
- Advanced Topics:
- Sustainability considerations within continuous biomanufacturing.
- Future trends and emerging technologies in the field.
- Data management and digitalization strategies for continuous processes.
- Expert Interaction:
- Panel discussions and Q&A sessions with industry experts.
- Networking opportunities to foster connections and collaboration.
The workshop aims to provide a comprehensive understanding of continuous processing, from fundamental principles to advanced applications, enabling attendees to drive innovation and achieve bioprocess excellence.
Key Speakers:
- Shireen Goh, Associate Professor, Singapore University of Technology & Design
- Stefano Menegatti, Associate Professor, North Carolina State University
- 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
- 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.
- 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.
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 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
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
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
• What is considered a must for CLD?
• What about your process has proved indispensable?
• What might work in the future with more improvement?
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.
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.
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.
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.
• 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
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
• 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.
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.
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.
• Strategies to maintain minimum pressure to prevent viral particle penetration
• Practical considerations for implementing viral filtration in continuous processing
• Integrating in silico models for process development
• Using digital twins and ML to generate predictive results without experiments
• High-throughput screening and model refinement for improved accuracy
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.
This session will cover the key steps in developing a scalable and efficient downstream process for biologics production. Topics will include translating small-scale laboratory processes to pilot and commercial scales, selecting the appropriate purification techniques, and ensuring robust, reproducible processes that meet regulatory requirements. Case studies will be presented to illustrate the challenges faced during scale-up and how they were overcome.
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.
As biopharmaceutical production scales, the environmental footprint of downstream processing becomes a growing concern. This talk will explore sustainable practices in downstream processing, including waste reduction, energy-efficient processes, and minimizing the use of toxic solvents. Techniques like recyclable chromatography resins, green solvents, and waste stream recycling will be discussed to promote sustainability in the industry.
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 insights into the new ICH guidelines on analytical profiles (Q14), including the ATP document, requirements for methods, and the shift to different validation approaches. The talk will cover long-term monitoring, case studies of switching methods or technologies, and how to meet acceptance criteria or update analytical technology.
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 latest trends in biomanufacturing facility design, including the integration of single-use systems and automation to achieve greater flexibility, efficiency, and cost-effectiveness. This session will address how to bridge the gap between islands of automation and fully integrated plants.
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.
Learn how to effectively implement digitalization strategies in biomanufacturing, addressing the critical need for skill upgrades and a common understanding of innovation data. This session will provide practical guidance on leveraging data analytics and other digital tools to drive innovation.
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.
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.
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.
Round Table Session 1: Winning in the Biosimilar Market: Strategies for Cost-Effective Manufacturing and Competitive Advantage
- Cost-Effective Manufacturing Strategies: Learn practical strategies for reducing manufacturing costs, including process optimization, efficient raw material sourcing, and streamlined operations.
- Efficient Intensification Techniques: Discover how to implement process intensification to increase productivity and reduce costs without compromising product quality.
- Real-World Examples of Cost Reduction
- Gaining a competitive edge
Round Table Session 2: Maximizing mAb Profitability: Optimization Strategies for a Competitive Landscape
- mAb Process Optimization: Explore techniques for optimizing mAb manufacturing processes to improve efficiency, yield, and product quality.
- Cost Reduction Strategies: Identify and implement effective cost reduction measures across the mAb manufacturing lifecycle, from raw materials to final product.
- Process Improvements for Enhanced Productivity: Discover process improvements that can lead to increased productivity and shorter timelines.
- Staying Ahead of the Curve: Gain insights into emerging technologies and best practices that can help you stay ahead of the competition in mAb manufacturing.
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.
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.
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.
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 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.
Traditional column-based chromatography may not be sufficient for cell and gene therapies, ADCs, and mRNA-based therapeutics. As the industry shifts toward novel modalities, companies must adopt innovative purification strategies and real-time analytical tools for enhanced bioprocess monitoring. This session will explore case studies on adopting membrane-based purification, real-world applications of Raman spectroscopy, and strategies for overcoming scale-up and manufacturing bottlenecks in emerging modalities. Attendees will evaluate next-generation technologies for their manufacturing pipelines, learning how to implement them successfully and overcome regulatory challenges, while future-proofing purification workflows for next-gen biologics
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.
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.
- 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
- 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.
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)
- 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
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
- 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
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
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.
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
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!
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
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
- 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.
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
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 a 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.
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
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