Monday, September 21, 2020

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

This panel will explore the critical role of CDMOs in modern biomanufacturing, covering topics from partner selection and relationship management to navigating regulatory challenges and optimizing for success in a rapidly changing environment

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at Cell and Gene Therapy International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at Cell and Gene Therapy International.

• Developing formulations that enhance the stability of biologics

• Include the use of excipients, lyophilization, and novel delivery systems

The Biophorum ATMP Visible Particles workstream has proposed a holistic, lifecycle approach to reduce and de-risk visible particulates in cell therapy (CT) drug formulations. This involves characterizing and detecting particulates in the manufacturing process and formulation, then improving material quality and process controls to minimize them.

CTs face unique challenges in particulate control and detection compared to other injectables, including difficult-to-inspect formulations and containers. CTs, with inherent cell-related particulates, complicate the detection of other particles. Terminal sterilizing filtration isn't applicable due to cell size and formulation needs. Small batch volumes, especially in autologous therapies, make rejecting units with particle defects critical. Regulatory guidance and health authority expectations are not aligned with the unique characteristics of cell and gene therapies, posing challenges for sponsors in meeting particulate specifications.

A Representative from Single Use Support

• Establishing clear communication channels and quality agreements that foster a shared commitment to quality.
• Implementing effective oversight and monitoring systems for CDMO operations.
• Navigating differences in quality cultures and ensuring alignment.
• Leveraging technology and data to enhance quality and compliance in external manufacturing.
• Addressing challenges related to investigations, deviations, and CAPAs across organizations.
• Building trust and transparency as foundational elements of a strong quality partnership.

Patient convenience and the need for self-administration are fueling the demand for prefilled syringes and autoinjectors for biologics, requiring specialized formulation and fill finish capabilities.

• Anticipating the needs on the work streams
• Process Understanding and Design
• Technology Transfer
• Dealing with changes and Comparability

• Frameworks for evaluating make vs. buy decisions in biopharma.
• Identifying core competencies and areas ripe for outsourcing.
• Balancing control and flexibility in manufacturing choices.
• Case studies illustrating successful outsourcing strategies.

• Review of the main streams and initial status
• Broad requirements for Tox and for First in Human Studies
• Cell banking
• Other Raw materials
• Drug Substance Process and Manufacture
• Minimum requirements
• Understanding your process :
• Impurities : identity, clearance, control
• First steps towards a control strategy
• Adventitious contamination and Viral Clearance Studies
• Drug formulation and Drug Product Processing
• Analytical package
• Release methods definition and development
• From method performance to method validation
• In Process Controls (else cover under process?)
• Batch data in the submission
• Product Characterisation and Reference standard
• Stability ( DS and DP)
• Forced degradation studies : necessity and importance
• Why is stability important ?
• Different type of stability studies and typical package for PhI
• Shelf life assignment

This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.

1. 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.

2. 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.

3. Navigating Regulatory Compliance and Tech Transfer Challenges

   • Explore key regulatory considerations in tech transfer.

   • Understand compliance challenges and how to effectively navigate regulatory requirements.

4. 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.

5. 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.

6. Expert Insights and Practical Advice

   • Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.

   • Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.

• Advances in transposon technology for targeted integration and enhanced stability

• Stability improvements through transposon innovations: what works and what doesn’t

• What to start early in the process?
• How early is too early to consider strategy for scaling up?
• How to define the right commercial scale early in development
• Best practices for locking in scalable processes from the start
• Designing therapies with long-term success in mind—beyond Phase 1
• Key considerations for commercial scale up, reimbursement, raw material availability, and regulatory approvals
• Pathways to developing safe, reimbursable therapies with broad patient access

• Addressing supply chain disruptions: strategies for procurement, digitalization, and raw material management

• Sustainability challenges and cost-saving innovations for large-scale media production

• How to ensure safety and compliance while accelerating clinical and manufacturing timelines
• Common pitfalls and best practices
• Strategies to address changing regulatory requirements during rapid development
• Strategic approaches to transitioning from clinical development to manufacturing readiness
• Aligning speed with regulatory expectations for product quality and safety

• Key FDA regulations and guidelines relevant to CDMO partnerships (e.g., 21 CFR Parts 210 & 211).
• Understanding and implementing robust quality agreements.
• Navigating supplier qualification and audit processes.
• Strategies for maintaining compliance throughout the partnership lifecycle.

Round Table Session 1: Host Cell Proteins (HCPs): The Unseen Challenge

• Focus: Managing HCPs throughout the bioprocessing lifecycle.
• Key Discussion Points:
  • HCP Characterization and Detection: Advancements in analytical methods for identifying and quantifying HCPs, including challenges with low-level detection and diverse HCP populations. Discuss the use of orthogonal methods.
  • Impact of HCPs on Product Quality: Explore the mechanisms by which HCPs can affect product stability, efficacy, and immunogenicity. Focus on specific examples and case studies.
  • HCP Removal Strategies: Evaluate current HCP removal technologies (chromatography, filtration) and discuss emerging approaches (e.g., novel resins, targeted removal). Consider the challenges of removing specific "high-risk" HCPs (lipases, proteases).
  • HCP-Related Challenges in Different Product Modalities: Discuss specific HCP challenges associated with different types of biologics (e.g., antibodies, proteins, vaccines, cell therapies).
  • Setting HCP Acceptance Criteria: How do we determine appropriate levels of HCPs, and what factors influence these decisions? Discuss the role of regulatory guidance.
  • Future Directions in HCP Management: Explore innovative technologies and strategies for improved HCP control and monitoring. Discuss the role of AI/ML in predicting and managing HCP-related risks.

Round Table Session 2: Extractables and Leachables (E&L): Ensuring Patient Safety

• Focus: Understanding, controlling, and mitigating the risks associated with E&L.
• Key Discussion Points:
  • E&L Sources and Identification: Discuss common sources of E&L in bioprocessing and packaging, including materials of construction, processing equipment, and packaging components. Explore advanced analytical techniques for identifying and characterizing E&L.
  • Risk Assessment and Toxicological Evaluation: How do we assess the potential toxicity of identified E&L? Discuss the use of toxicological studies and risk-based approaches to determine acceptable levels.
  • E&L Control Strategies: Explore strategies for minimizing E&L, including material selection, process optimization, and container closure system design. Discuss the importance of supplier qualification and change control.
  • Analytical Method Development and Validation: Discuss the challenges of developing and validating robust analytical methods for E&L testing, especially for trace-level contaminants.
  • Regulatory Requirements for E&L: Review current regulatory guidelines and expectations for E&L characterization and control. Discuss differences in global regulatory requirements.
  • Emerging Trends in E&L Management: Explore new technologies and approaches for E&L analysis and control, including the use of predictive modeling and simulation.

Roundtable Session 3: Polysorbate Degradation: Protecting Product Stability

• Focus: Understanding the mechanisms of polysorbate degradation and developing strategies to mitigate it.
• Key Discussion Points:
  • Mechanisms of Polysorbate Degradation: Discuss the various pathways of polysorbate degradation, including hydrolysis, oxidation, and enzymatic degradation (with a focus on lipase contamination).
  • Factors Influencing Degradation: Explore the factors that can influence polysorbate degradation, such as pH, temperature, oxygen levels, and the presence of specific HCPs (especially lipases).
  • Analytical Methods for Monitoring Degradation: Discuss analytical techniques for detecting and quantifying polysorbate degradation products.
  • Strategies for Mitigating Degradation: Explore strategies for preventing or minimizing polysorbate degradation, including process optimization, excipient selection, and the use of inhibitors.
  • Impact of Polysorbate Degradation on Product Quality: Discuss the potential consequences of polysorbate degradation on product stability, efficacy, and safety.
  • Case Studies of Polysorbate Degradation: Share real-world examples of polysorbate degradation challenges and the solutions implemented.
  • Future Directions in Polysorbate Management: Explore new approaches for stabilizing polysorbates and preventing degradation, including the use of novel excipients and formulation strategies. Discuss the potential of more sensitive analytical methods.

Round Table Session 1: Host Cell Proteins (HCPs): The Unseen Challenge

• Focus: Managing HCPs throughout the bioprocessing lifecycle.
• Key Discussion Points:
  • HCP Characterization and Detection: Advancements in analytical methods for identifying and quantifying HCPs, including challenges with low-level detection and diverse HCP populations. Discuss the use of orthogonal methods.
  • Impact of HCPs on Product Quality: Explore the mechanisms by which HCPs can affect product stability, efficacy, and immunogenicity. Focus on specific examples and case studies.
  • HCP Removal Strategies: Evaluate current HCP removal technologies (chromatography, filtration) and discuss emerging approaches (e.g., novel resins, targeted removal). Consider the challenges of removing specific "high-risk" HCPs (lipases, proteases).
  • HCP-Related Challenges in Different Product Modalities: Discuss specific HCP challenges associated with different types of biologics (e.g., antibodies, proteins, vaccines, cell therapies).
  • Setting HCP Acceptance Criteria: How do we determine appropriate levels of HCPs, and what factors influence these decisions? Discuss the role of regulatory guidance.
  • Future Directions in HCP Management: Explore innovative technologies and strategies for improved HCP control and monitoring. Discuss the role of AI/ML in predicting and managing HCP-related risks.

Round Table Session 2: Extractables and Leachables (E&L): Ensuring Patient Safety

• Focus: Understanding, controlling, and mitigating the risks associated with E&L.
• Key Discussion Points:
  • E&L Sources and Identification: Discuss common sources of E&L in bioprocessing and packaging, including materials of construction, processing equipment, and packaging components. Explore advanced analytical techniques for identifying and characterizing E&L.
  • Risk Assessment and Toxicological Evaluation: How do we assess the potential toxicity of identified E&L? Discuss the use of toxicological studies and risk-based approaches to determine acceptable levels.
  • E&L Control Strategies: Explore strategies for minimizing E&L, including material selection, process optimization, and container closure system design. Discuss the importance of supplier qualification and change control.
  • Analytical Method Development and Validation: Discuss the challenges of developing and validating robust analytical methods for E&L testing, especially for trace-level contaminants.
  • Regulatory Requirements for E&L: Review current regulatory guidelines and expectations for E&L characterization and control. Discuss differences in global regulatory requirements.
  • Emerging Trends in E&L Management: Explore new technologies and approaches for E&L analysis and control, including the use of predictive modeling and simulation.

Roundtable Session 3: Polysorbate Degradation: Protecting Product Stability

• Focus: Understanding the mechanisms of polysorbate degradation and developing strategies to mitigate it.
• Key Discussion Points:
  • Mechanisms of Polysorbate Degradation: Discuss the various pathways of polysorbate degradation, including hydrolysis, oxidation, and enzymatic degradation (with a focus on lipase contamination).
  • Factors Influencing Degradation: Explore the factors that can influence polysorbate degradation, such as pH, temperature, oxygen levels, and the presence of specific HCPs (especially lipases).
  • Analytical Methods for Monitoring Degradation: Discuss analytical techniques for detecting and quantifying polysorbate degradation products.
  • Strategies for Mitigating Degradation: Explore strategies for preventing or minimizing polysorbate degradation, including process optimization, excipient selection, and the use of inhibitors.
  • Impact of Polysorbate Degradation on Product Quality: Discuss the potential consequences of polysorbate degradation on product stability, efficacy, and safety.
  • Case Studies of Polysorbate Degradation: Share real-world examples of polysorbate degradation challenges and the solutions implemented.
  • Future Directions in Polysorbate Management: Explore new approaches for stabilizing polysorbates and preventing degradation, including the use of novel excipients and formulation strategies. Discuss the potential of more sensitive analytical methods.

The biopharmaceutical industry is moving towards greater flexibility and efficiency in manufacturing. This session will explore the concept of integrated, product-agnostic biomanufacturing facilities – those designed to produce a range of biologics using common equipment and platforms. Attendees will learn how this approach can reduce capital costs, accelerate development timelines, and improve responsiveness to market demands.

This session will take a grounded approach to the application of AI in biomanufacturing, exploring practical use cases, addressing common misconceptions, and discussing the limitations of current AI technologies.

• Approaching the challenges and considerations when scaling-up ADC production
  • Process Optimization
  • Quality Control
  • Regulatory compliance
• Strategies to incorporate when scaling up conjugation reactions, purification processing, formulation development
• Implementation of robust quality control measures to ensure purity, potency & stability
• Case study on overcoming scale-up challenges

• Introduction
• Objectives and outline of course
• Main development stages and regulatory cadre
• The main streams of activity from cell line to drug product
• The CTD for Regulatory filings. Quality Module – overview of main sections
• The importance of CMC : Quality (and Safety and Efficacy)
• Supply
• The pillars of pharmaceutical development
• Criticality Assessment
• Control strategy & Validation
• QBD Principles
• Most relevant regulatory Guidance and where to find it MH

Quiz

• The evolution of biopharmaceutical modality
• Analytical methods and their purpose in biopharmaceutical development and manufacturing
• Analytical method development and validation
• Product physicochemical characterization - high-performance liquid chromatography (HPLC), capillary electrophoresis (CE), spectroscopy, imaging, and post-translational modification (PTM)
• Product biological assays - cell-based assays (CBA), enzyme-linked immunosorbent assays (ELISA), and potency assays
• Microbiological contaminants - sterility testing, endotoxin testing, and microbial limits testing
• Process impurity testing - host cell DNA, host cell proteins, chromatography ligand
• Role of quality control (QC) and quality assurance (QA) in biopharma
• Case studies and industry examples
• Latest and future advancements in analytical methods and quality assurance

In order to achieve development timelines, rapid process development can result in the sacrifice of capsid recovery in order to meet specifications. By establishing a strategy focused on critical process parameters, leveraging design of experiments strategies, and using statistically sound analytics, genome-containing capsid recovery can be optimized in recombinant AAV downstream unit operations.

In order to achieve development timelines, rapid process development can result in the sacrifice of capsid recovery in order to meet specifications. By establishing a strategy focused on critical process parameters, leveraging design of experiments strategies, and using statistically sound analytics, genome-containing capsid recovery can be optimized in recombinant AAV downstream unit operations.

The CGT Supply Chain forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences between supply chain executives of 20-30 CGT therapeutic development companies.

An exclusive invite-only session, the forum will include 2-3 case studies and panel discussions focussed on brainstorming solutions to navigate the complexities of supply chain management for CGTs. Offering comprehensive insights into innovations, tools and trends to enhance efficiency, visibility, and responsiveness across the entire network and ultimately increasing CGT access to patients.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Discover cutting-edge analytical strategies for characterizing complex next-generation biologics, including LNPs, hypoimmune cell therapies, and CRISPR-based treatments. As cell and gene therapies rapidly evolve, innovative analytical techniques are needed to ensure product quality, accelerate development, and navigate the evolving regulatory landscape. This session explores new tools for characterizing engineered immune cells and gene-edited therapies, the growing role of Next-Generation Sequencing (NGS) and Mass Spectrometry (MS), and the integration of emerging bioanalysis techniques. Attendees will gain actionable insights to tackle the analytical challenges posed by complex biologics and learn about innovations in analytical methodologies for biopharmaceutical characterization, process analytics, and advanced automated peptide synthesis and purification methods

• Optimizing bioconjugation processes for dual-payload ADCs
  • E.g. Cysteine conjugation, amino acid incorporation, for payload attachment
  • Varying payload incorporation
• Manufacturing considerations for site-specific conjugation
• Analytical method development for complex ADCs
• CMC aspects of regulatory submissions for novel ADCs

Process intensification (PI) is a strategy to maximize throughput and reduce operational costs by improving the efficiency of each step in the downstream process. This session will cover the latest techniques in PI, such as multi-column chromatography, hybrid filtration systems, and integrating various purification steps. Attendees will learn how these innovations are driving faster and more cost-effective biologics manufacturing.

• How to effectively adopt AI tools in the CGT space
• Opportunities for AI and machine learning in process development & manufacturing
• Real-world applications of AI in CGT pipeline development
• Case study insights on AI-driven innovation

• How to effectively adopt AI tools in the CGT space
• Opportunities for AI and machine learning in process development & manufacturing
• Real-world applications of AI in CGT pipeline development
• Case study insights on AI-driven innovation

Automation is rapidly transforming downstream processing by improving reproducibility, reducing labor costs, and accelerating timelines. This talk will focus on the integration of automation technologies in the downstream workflow, from automated chromatography systems to robotic liquid handling and filtration. The speaker will discuss how automated systems enhance productivity, improve process control, and allow for more consistent and scalable processes.

BioPhorum members, through an industry-wide collaboration, have devised a proof-of-concept for controlling product collection during cation exchange (CIEX) bind-and-elute chromatography. Our approach to this task, using real-time multi-angle light scattering (RT-MALS) technology, involves directly monitoring aggregate content - a critical quality attribute (CQA) - and applying a defined breakthrough or cumulative threshold to stop pooling.

The intent of this partnership is to show that direct monitoring of a suitable CQA enables recovery of good material, currently lost due to conservative pooling practices. In addition to yield improvements for this polishing unit operation, the resulting pools should be more consistent batch-to-batch with respect to aggregate content. In this presentation, we share results from Phase I of the project.

MALS technology measures the intensity of scattered light produced by a sample at different angles. The scattered intensity relates to the molar mass and size. Averaged over a sample, the reported value is the weight-average solution molar mass (Mw) which will increase with increasing aggregate content. In our work, the in-line MALS detector reports the change in Mw in real-time allowing us to “see” when the aggregate population starts to break through. This is already an improvement over traditional UV which does not differentiate between protein populations. We could have set a reasonable threshold limit to the Mw signal and used it to trigger stop pooling and moved on with our work; however, the team took a more ambitious approach to use the Mw measurement in a more accurate way to calculate the cumulative aggregate content in the pool and use a cumulative aggregate threshold to trigger stop pooling. The more ambitious approach ensures tighter consistency of aggregate content in pools batch-to-batch, irrespective of peak shape. This approach also reduces the need for conservative pooling since stop pooling is triggered by the actual CQA (pool % HMW).

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

An acceleration in the speed at which therapies are breaking through from basic scientific discovery to development is placing a premium on innovative business development models. Biotech innovators, Pharma, and VCs alike are competing for successful win-win deal-making models in the face of novel personalized applications of gene therapies, gene editing, regenerative medicines, vaccines, therapies, and companion biomarkers. This session will cover best practices and strategies in finding the right partnerships to engage.

Spotlight Presentation – Calling all Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Spotlight Presentation – Calling all Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Examples of working with biopharma customers to optimize raw material formulations for specific applications.

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

The CGT C-Level Forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences between executives of ~20 CGT therapeutic development companies. The forum is designed to foster new ideas and create new relationships between attendees so that they can leave with tangible outcomes and that will drive their businesses and the sector forward.

An exclusive invite-only session, in a closed room setting, the forum will include 2-3 case studies discussing the right approach to engage and partner with pharma and industry. It will also include general discussion focussed on brainstorming solutions to the most pressing and current challenges of CEO/CSO/CBO/CMOs in the CGT sector.

Process intensification is a key strategy to increase the productivity of cell culture systems while reducing footprint and cost. This session will discuss methods such as increased cell density, optimized feeding strategies, and higher perfusion rates. The speaker will highlight examples of successful process intensification in mammalian cell cultures and how this approach can lead to higher product titers and more efficient use of resources.

Delivery of plasmid DNA enables systemic expression of therapeutic molecules, including but not limited to monoclonal antibodies, proteins and peptides. However conventional delivery techniques are limited by constraints of redosability and toxicity in their ability to delivery DNA effectively. Polymeric systems can overcome these constraints but have a very large design space. This presentation will describe how machine learning can leverage large design spaces for the design of polymeric delivery vehicles for a broad range of therapeutically relevant molecules in vivo.

Delivery of plasmid DNA enables systemic expression of therapeutic molecules, including but not limited to monoclonal antibodies, proteins and peptides. However conventional delivery techniques are limited by constraints of redosability and toxicity in their ability to delivery DNA effectively. Polymeric systems can overcome these constraints but have a very large design space. This presentation will describe how machine learning can leverage large design spaces for the design of polymeric delivery vehicles for a broad range of therapeutically relevant molecules in vivo.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

• The evolution of biopharmaceutical modality
• Analytical methods and their purpose in biopharmaceutical development and manufacturing
• Analytical method development and validation
• Product physicochemical characterization - high-performance liquid chromatography (HPLC), capillary electrophoresis (CE), spectroscopy, imaging, and post-translational modification (PTM)
• Product biological assays - cell-based assays (CBA), enzyme-linked immunosorbent assays (ELISA), and potency assays
• Microbiological contaminants - sterility testing, endotoxin testing, and microbial limits testing
• Process impurity testing - host cell DNA, host cell proteins, chromatography ligand
• Role of quality control (QC) and quality assurance (QA) in biopharma
• Case studies and industry examples
• Latest and future advancements in analytical methods and quality assurance

• Recent advancements in Raman spectroscopy, near-infrared (NIR) spectroscopy, and IR spectroscopy

• Case studies on implementing high-throughput PAT for upstream process development

• Challenges and opportunities in scaling PAT for GMP bioreactors and downstream processes.

• Gap analyses of the studies available to prepare a Phase 1 submission.
• 4 teams (Analytics, DS, DP, Materials) to cover different parts of case
• 30 – 45 min work in groups : Brief :
• do the existing studies support a Phase 1 file and what are the risks involved?
• Recommendation on eventual additional or superfluous studies
• Map studies / data onto CTD modules (if time)
• 60 min debrief (15 min / group)
• General Conclusions and End of Session

This session will delve into the complex regulatory landscape surrounding the use of Artificial Intelligence (AI) and Machine Learning (ML) in the development, manufacturing, and quality control of biologics. Experts will discuss evolving guidelines from regulatory agencies, including best practices for validating AI/ML models, ensuring data integrity, and demonstrating the reliability and transparency of AI/ML-driven decisions, panelists will deliver strategies, improvements in processes, and lower costs of operations through the proper application of the data. Key focus areas include strategies for generating trust and confidence in AI/ML-based analytics for regulatory submissions and maintaining compliance throughout the product lifecycle with specific actionable strategies

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Through a series of presentations, case studies, interactive discussion and exercises this workshop will delve deeply into the underlying science of developing potency assays.

Some of the topics to be discussed include:

• Overview of what potency is, and what it is not
• Considerations when qualifying and validating bioassays
• From initial ideas, through characterisation to the final potency method
• Clinical correlation
• Regulator objections and why
• Examples of potency assays for approved products
• Examples of a cell and gene therapy potency assays for various product types

• Targeting ligand selection and impact on isotope selection
• Advancements in radiochemistry for improving the safety & efficacy of radiopharmaceuticals
  • E.g. use of chelators to attach radioligands to targeting vehicle
• Impacts on manufacturing and quality
  • Dealing with variability in radioisotope production
  • Implementing robust quality control measures for radioactive materials
  • Optimizing manufacturing processes to account for isotope decay
  • Strategies for consistent batch-to-batch production
• Considerations for new radioisotopes and indications
  • Biodistribution
  • Metabolism, Excretions and Decay
  • CMC considerations
    
    

• Growing importance of digitalization, AI, and machine learning in the biopharma industry.
• Key pillars of digital transformation in biopharma.

Key Areas of Digitalization

• Data Management and Integration (from Development to Manufacturing).
• Automation and Robotics in bioprocess workflows.
• Real-time Monitoring and Advanced Analytics for process optimization.

Applications in Bioprocessing

• Use of digital twins and AI to optimize upstream and downstream unit operations.
• Role of ML/AI-driven tools for Advanced Therapy Medicinal Products (ATMP) manufacturing.
• AI-driven real-time monitoring, predictive maintenance, and anomaly detection in production lines.
• Simulation-based process development for rapid scale-up.

Challenges and Considerations

• Overcoming data silos and ensuring system interoperability.
• Addressing regulatory requirements for AI and digital tools in GMP environments.
• Ensuring data quality, integrity, and security in digitalized workflows.
• Bridging talent gaps and fostering a digitally skilled workforce.

Case Studies

• Real-world examples of digital transformation in bioprocessing.
• Lessons learned from integrating AI-driven tools in ATMP production.

Future Trends and Directions

• Adoption of Industry 4.0 principles in biopharma manufacturing.
• Emerging technologies such as edge computing and IoT for bioprocessing.
• Sustainability and digitalization: How to?

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

A Representative from Solvias

A Representative from Solvias

• Explain what downstream bioprocessing involves: the purification and formulation of the biological product after cell culture and fermentation.
• Outline the key objectives: ensuring product purity, quality, and stability.
• Describe the process of harvesting cells or extracellular products from the bioreactor.
• Explain the methods used for cell separation (e.g., centrifugation, filtration).
• Introduce the main purification methods: chromatography, filtration, and precipitation.
• Describe different types of chromatography (e.g., affinity, ion-exchange, size-exclusion) and their applications.
• Explain the principles and applications of ultrafiltration and diafiltration.

• Introduction to Biopharmaceutical Life Cycle.
• Explain what upstream bioprocessing involves: the early stages of production, including cell culture and fermentation.
• Outline the key objectives: generating the desired biological product through cell growth and expression.
• Discuss the selection of cell lines (e.g., CHO cells, microbial cells).
• Introduce bioreactors and their role in providing a controlled environment for cell growth.
• Discuss different types of bioreactors (e.g., stirred-tank, wave, single-use) and their applications.
• Explain the fermentation process and its parameters (e.g., pH, temperature, oxygen levels).
• Explain the importance of culture media in supporting cell growth and productivity.
• Describe the process of scaling up from lab-scale to commercial-scale production.
• Highlight current trends in upstream bioprocessing (e.g., single-use technologies, continuous processing).
• Discuss future directions and innovations in the field.

Some of the topics to be discussed include:

• Overview of what potency is, and what it is not
• Considerations when qualifying and validating bioassays
• From initial ideas, through characterisation to the final potency method
• Clinical correlation
• Regulator objections and why
• Examples of potency assays for approved products
• Examples of a cell and gene therapy potency assays for various product types

Traditional QC methods often cause bottlenecks due to their slowness, labor intensity, and variability. This session explores cutting-edge automation solutions, high-throughput analytics, and real-time release testing (RTRT) designed to dramatically enhance efficiency and data integrity in biopharmaceutical QC, streamline workflows, and accelerate testing. As gene and cell therapies move to commercial manufacturing, you'll learn how to scale QC processes, ensure assay transferability, and maintain data integrity across production sites. The session will cover implementing RTRT and high-throughput analytics, strategies for harmonizing QC across multiple manufacturing locations, and the rise of plug-and-play analytical tools. Attendees will gain actionable insights for optimizing QC operations, improving process control, ensuring compliance, and reducing risk during commercial expansion. Learn about next-gen QC strategies that increase efficiency, improve consistency, and ensure regulatory compliance while reducing product release bottlenecks.

With AI amd Machine Learning gainig traction in analytics and process control, many companies are still unclear on the actual benefits of implementation. This session critically examines the real-world applications, separating genuine advancements from overhyped claims. Bringing together industry leaders, AI pioneers, and regulatory experts, the session will discuss successes, failures, and where AI is truly delivering value. It will explore successful AI/ML implementations for process optimization, predictive quality control, streamlined data analysis, and the automation of flow cytometry, how AI-driven computational modeling is reshaping biomanufacturing, and the next five years of AI in analytical sciences and QC. Attendees will gain insights into AI’s current applications and future potential, how it redefines quality control, data analysis, and process optimization, and hurdles that need to be overcome.

Round Table 1: Scaling CP: From Bench to Plant

• What breaks between lab-scale CP and full-scale implementation?
• What scale counts as “commercially viable” for CP?
• What's missing in current tech to make this easier

Round Table 2: The Business Case for Hybrid and CP Models

• How do you model ROI on continuous or hybrid investments?
• What incentives drive adoption (speed, cost of goods, flexibility)?
• When is it worth revisiting old filings to modernize an existing process?

Round Table 3: Continuous But Flexible: Connected vs Fully Continuous Systems

• Is connected processing a stepping stone or an endpoint?
• When is hybrid actually better than true CP?

This session will provide practical advice for bioprocess programs navigating post-approval submissions and late-stage commercialization. A key focus will be post-approval comparability, including strategies for managing situations where materials fall outside of established acceptance criteria. The talk will explore the use of risk assessment.

• Case study on challenges with formulating high concentration biologics – particularly emerging biologics

• Improve viscosity to avoid issues with syringe ability and injectability - development of specialized delivery devices

• Optimising buffer systems and excipient usage

• Maintaining homogeneity

• Storage and temperature challenges

• What is required for product characterization by regulatory authorities?
• Use of risk-based assessment to identify product CQAs
• In-process controls, stability, and release testing expectations from authorities
• How analytics can be used to support the move to “the product is the product”
• Development of rapid process characterization assays

• What is required for product characterization by regulatory authorities?
• Use of risk-based assessment to identify product CQAs
• In-process controls, stability, and release testing expectations from authorities
• How analytics can be used to support the move to “the product is the product”
• Development of rapid process characterization assays

Scalability is critical to ensure that bioprocesses developed at small scales translate to larger scales without compromising product quality, yield, or consistency in cell growth kinetics. Maintaining key culture conditions, such as nutrient supply, mass transfer, and mixing, is essential for achieving consistent cell growth, metabolic behavior, and viral vector production. This study explores how intensified cell perfusion systems can work hand in hand with cell culture media development to address these challenges. Our focus for this work is on scale-up strategies that ensure reproducible cell growth and viral vector production across different scales in HEK293 cell cultures.

Join us for a session to discover how to:

• Enable optimization of nutrient balance, execute efficient waste management, and achieve consistent and scalable HEK293 cell growth via a specialized HEK293 media perfusion process
• Intensify viral vector production by transiently transfecting cells across various perfusion processes and bioreactors
• Achieve consistent HEK293 cell metabolic profiles in different cell densities, demonstrating a robust and versatile bioprocess with precise control of bioprocess parameters
• Reduce operational costs while enhancing titer yield with the combination of automated control systems and advanced perfusion techniques
  
  

Small group discussions focused on specific challenges in CDMO selection, such as:

• Phase-appropriate CDMO selection & finding CDMOs with niche capabilities (e.g., specific cell lines, advanced therapies).
• Balancing cost, quality, and timelines in the selection process.
• Building trust and transparency in the initial stages of partnership.

30min table discussion

10 minute findings feedback to the group for each table

This talk will address the critical challenges of technology transfer, particularly the data-related bottlenecks that often hinder the scaling of projects from lab to clinical trial and manufacturing. It will explore the role of software solutions in streamlining this process and discuss how modernizing tech transfer practices can improve efficiency, data handling, QA, and overall speed. The presentation will offer practical strategies for overcoming these hurdles and provide insights into what an optimized, modernized tech transfer workflow could entail.

• Defining point-of-care manufacturing and decentralized manufacturing
• Scaling with consistency: key elements for global standardization
• Tech & innovation: cutting-edge solutions for safe, efficient, and cost-effective decentralized manufacturing
• Overcoming obstacles to decentralised manufacturing to enable access to approved CGTs
• Differences in systems across decentralized cell therapy manufacturing models - how can we harmonize?
• Standardization vs a modular approach
• Equipment, data, and technologies
• Dialogue and collaboration between developer/practitioner to streamline eventual implementation
• Improving worldwide access: how can local manufacturers and supplier help?
• Real world experience with a decentralized cell therapy manufacturing platforms supporting Phase I/II clinical trials

UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene for the treatment of Wilson Disease. UX701 leverages Ultragenyx’s proprietary producer cell line platform, Pinnacle PCL™, to produce rAAV at 2000L scale.

Process characterization of UX701 is an essential aspect of the process validation lifecycle, aimed at defining manufacturing process ranges to maintain the target product profile (TPP). A risk-based approach was applied to the late-stage development activities for UX701 upstream and downstream process including process parameter and raw materials risk assessment, scale-down model (SDM) qualification, and process characterization (PC) studies, to establish process controls and inform parameter criticality for the upstream and downstream unit operations in the 2000L manufacturing process. We assessed over 400 upstream and downstream process parameters for their impacts to process performance indicators (PIs) and critical quality attributes (CQAs) and classified them into high, medium or low risk parameters. Of these we studied 11 high and medium risk upstream process parameters and 2 raw materials, and 14 high and medium risk downstream process parameters in PC studies.

We identified 8 process parameters in upstream and 3 downstream process parameters to be critical (CPP) or key (KPP). In the upstream process, temperature and pH, seeding density, and helper virus concentration were all found to be CPPs during production. In the downstream process, viral heat inactivation step, temperature and time affect the inactivation kinetics of the helper virus. Characterization of our anion-exchange (AEX) polishing chromatography step identified an upper threshold which the load conductivity should stay below to ensure binding onto the AEX column.

In this work, we have characterized the cell culture and purification manufacturing process and demonstrate a robust and high yielding process for the manufacture of UX701. This is the first PC campaign for our Pinnacle PCL™ rAAV gene therapy manufacturing platform.

In an effort to access cutting edge research and stay ahead of the curve during this unprecedented time, investors and pharma companies are taking less risks. What long term trends have fizzled out and what is on the rise to increase innovation? Are new strategies sustainable as increasing investment rounds requiring more and more funds to obtain new treatments, therapies and technologies?

Leading biopharma experts and VCs will share their insights on how to engage investors, which trends to watch for, how to position technologies and discoveries to attract investors, and navigate this evolving landscape.

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Round off your day at BPI School with an interactive and insightful discussion:

• Learnings of the day and a chance to recap:
  • Key trends and challenges in biopharmaceutical manufacturing, upstream and downstream.
  • The impact of digitalization on the future of biopharma.
  • Emerging therapies: Opportunities and obstacles.
  • How to utilize and implement analytics tools
• Q&A and interactive discussion with industry experts.
• Bridging the gap between theory and practice in biopharma.

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)

• Understanding emerging therapies: distinctions between cell therapy, gene therapy, etc.
  • Therapeutic potential and current clinical landscape of different emerging therapies, unique challenges and opportunities presented.
  • Differences and similarities from ‘traditional’ biologics:
  • What learnings can we take from traditional modalities to approach novel modalities?
• Understanding the Cell Therapy and Gene Therapy manufacturing processes.
  • Best practices when entering/transitioning into the advanced therapy industry.
  • Leveraging experiences from your background into industry.
• Strategies and approaches to best utilise available technologies in the development & production of emerging therapies.
  • Moving and translating research from academia, to start up, industry, and beyond.
  • Understanding the difference between these, how to transition, pros and cons.
• Lessons and experiences from our panellists.

• Defining clear requirements and objectives in your RFP.
• Key criteria for evaluating potential CDMO partners (e.g., technical capabilities, quality systems, financial stability, cultural fit).
• Best practices for conducting thorough due diligence (technical, quality, financial).
• Utilizing site visits and audits effectively.

• Novel radiolabelling techniques and their impact on manufacturing
• Microfluidics in radiopharmaceutical production
• Scaling up production for solid tumor therapies
• Site-specific conjugation methods to enhance stability and targeting of radioligand-based therapies
  • Enzymatic conjugation
  • Click chemistry

Label-free optical microscopy enables non-perturbative profiling of biological samples based on their intrinsic molecular properties. We developed a multimodal label-free optical imaging system to identify biopharmaceutical CHO cell lines with favorable process performance at early stages. Combined with a machine learning-assisted single-cell analysis pipeline, the system achieved over 95% classification accuracy in early passages (0–5). This approach offers a promising solution to accelerate cell line selection, reducing time and resource demands in biopharmaceutical development.

• Improving the precision and control of drug attachment in ADC conjugation with site-specific conjugation techniques
  • The use of engineered antibodies (e.g. THIOMABs)
  • Implementing selenocysteine-containing antibodies

Development of novel conjugation techniques to enhance efficiency and specificity for improved patient outcomes

• Early-Stage Considerations: How can formulation scientists and process development teams collaborate early in the development lifecycle to ensure seamless transition to fill finish? What are the key formulation attributes that need to be considered from an aseptic processing perspective?
• Handling Challenging Formulations: What are the best practices for aseptically processing complex formulations (e.g., high concentration, viscous, sensitive biologics)? What innovative technologies or approaches can be employed?
• Impact of Excipients on Aseptic Processing: How do different excipients influence the aseptic process? Are there any excipients that pose particular challenges for sterilization or filtration?
• Single-Use Systems and Formulation Compatibility: How does the increasing adoption of single-use systems in fill finish interact with different biologic formulations? Are there any compatibility considerations?
• Real-World Case Studies: Panelists could share anonymized examples of challenges encountered at the intersection of formulation and aseptic processing and the solutions implemented.
• Future Trends and Innovations: What emerging formulation strategies or fill finish technologies hold promise for improving the integration of these two critical areas?

Traditional analytical methods offer incomplete insights into biologic products. Multi-omics approaches, integrating proteomics, genomics, and metabolomics data, provide a holistic view of product quality and batch consistency. As the industry seeks deeper root cause analysis and process optimization, companies must leverage advanced analytical frameworks for thorough characterization. This session will explore high-resolution mass spectrometry for integrating multi-omics data, next-generation sequencing (NGS) for transforming process characterization, and innovative omics-based analytics for enhanced biologics development. Attendees will gain a clear strategy for leveraging multi-omics for deeper process insights, ensuring data integrity and regulatory compliance, and streamlining process development, with insights from Sartorius on applying omics-based technologies for root cause analytics and overall process understanding.

• Case Studies: Success stories of regulatory approvals and commercialization
• Overcoming Hurdles: Key challenges from R&D to approval—and how to tackle them
• Scaling Up: Strategies for manufacturing, validation, and CMC data packages
• Regulatory Insights: Feedback from authorities on characterization, specifications, and approval pathways
• Post-Approval Management: Long-term data tracking, durability studies, and lifecycle strategies
• End-to-End Logistics: Mastering supply chain and global distribution
• Pathways to approval –Case study on the journey from discovery to commercial development and beyond

This talk will focus on specific strategies for removing high-risk HCPs, such as lipases, chromatin, and other species that bind to proteins or cause other processing issues. It will discuss the use of functionalized resins and membranes.

• Techniques for maintaining uniform cell suspension and minimizing damage

• Innovative bioreactor designs to enhance consistency in mixing and mechanical forces

• Case studies showcasing successful implementation in large-scale biomanufacturing

As gene therapy evolves beyond AAV-based delivery systems, new analytical challenges arise in ensuring genome integrity, optimizing therapeutic dosing, and mitigating immunogenicity risks. The industry is shifting toward alternative delivery methods such as CRISPR-based therapies, lipid nanoparticles (LNPs), plasmids, and synthetic vectors, requiring advanced analytical approaches to maintain product quality, efficacy, and regulatory compliance. This session will explore: Genome integrity analysis beyond traditional capsid characterization, leveraging long-read sequencing and multiplex digital PCR to assess payload stability and completeness, optimizing viral vectors for complete genomes, reducing dosing requirements, improving patient outcomes, and lowering production costs, second-dose strategies for non-AAV gene therapies, addressing durability, re-dosing challenges, and immunogenicity considerations, emerging analytical approaches for non-AAV delivery systems, including mRNA-based CRISPR therapies, LNP formulations, and synthetic gene vectors, key regulatory considerations for characterizing non-viral gene therapy platforms, ensuring alignment with evolving global standards.

This talk could address the increasing demand for biologics and discuss innovative solutions for capacity expansion, including modular facilities, process intensification, and improved resource utilization.

This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.

1. 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.

2. 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.

3. Navigating Regulatory Compliance and Tech Transfer Challenges

   • Explore key regulatory considerations in tech transfer.

   • Understand compliance challenges and how to effectively navigate regulatory requirements.

4. 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.

5. 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.

6. Expert Insights and Practical Advice

   • Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.

   • Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.

• Snapshot into the investment landscape for CGT products and enabling technologies for 2025/ 2026 and beyond
• How to drive investment in CGT?
• Positioning for success in a competitive funding environment: What do investors need to see?
• “Ask the Investor” interactive Q&A open discussion with our investor panel to get tips and tricks when pitching and their views on where innovation lies in the CGT field

• Initiatives to reduce their carbon footprint and implement recycling programs

• Defining point-of-care manufacturing and decentralized manufacturing
• Scaling with consistency: key elements for global standardization
• Tech & innovation: cutting-edge solutions for safe, efficient, and cost-effective decentralized manufacturing
• Overcoming obstacles to decentralised manufacturing to enable access to approved CGTs
• Differences in systems across decentralized cell therapy manufacturing models - how can we harmonize?
• Standardization vs a modular approach
• Equipment, data, and technologies
• Dialogue and collaboration between developer/practitioner to streamline eventual implementation
• Improving worldwide access: how can local manufacturers and supplier help?
• Real world experience with a decentralized cell therapy manufacturing platforms supporting Phase I/II clinical trials

Spotlight Presentation – Calling all Technology Thought Leaders!

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

Some of the topics to be discussed include:

• CDMO Selection: Models and Process
• Contract and Quality Agreement Negotiation
• Tech Transfer: Best practices and risk management for external tech transfers
• Digital Transformation in Tech Transfer
• Quality Assurance and Quality Control
• Life Cycle & Relationship Management

A Representative from Intertek

Onechain Immunotherapeutics developed a stromal-free bioprocess for the novo generation of γδ T cells from cord blood (CB) CD34+ HSPC or iPSC-derived CD34+, using recombinant Notch ligands, eliminating the need for expanding these cells from peripheral blood or other sources. The process can yield functional γδ T cells that can be armed with CARs with cytotoxic activity against multiple cancer cells. This strategy is highly scalable, consistent with GMP guidelines, and represents a step forward in the field of allogeneic, off-the-shelf CAR-T cell therapies.

• Leveraging CDMO expertise for specialized technologies and capabilities.
• Mitigating risks and enhancing supply chain resilience through external partners.
• Accelerating speed to market and managing capacity constraints.
• The role of external manufacturing in supporting innovation pipelines.

This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.

1. 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.

2. 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.

3. Navigating Regulatory Compliance and Tech Transfer Challenges

   • Explore key regulatory considerations in tech transfer.

   • Understand compliance challenges and how to effectively navigate regulatory requirements.

4. 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.

5. 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.

6. Expert Insights and Practical Advice

   • Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.

   • Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.

• Review of the main streams and initial status
• Broad requirements for Tox and for First in Human Studies
• Cell banking
• Other Raw materials
• Drug Substance Process and Manufacture
• Minimum requirements
• Understanding your process :
• Impurities : identity, clearance, control
• First steps towards a control strategy
• Adventitious contamination and Viral Clearance Studies
• Drug formulation and Drug Product Processing
• Analytical package
• Release methods definition and development
• From method performance to method validation
• In Process Controls (else cover under process?)
• Batch data in the submission
• Product Characterisation and Reference standard
• Stability ( DS and DP)
• Forced degradation studies : necessity and importance
• Why is stability important ?
• Different type of stability studies and typical package for PhI
• Shelf life assignment

Join industry leaders as we explore how AI, digitalization, and advanced analytics are transforming cell and gene therapy manufacturing. This dynamic panel will dive into:

• AI-Powered Manufacturing: How AI-driven models, predictive analytics, and digital twins are optimizing process control
• Process Analytical Technologies (PAT): The latest breakthroughs in real-time monitoring, automation, and data-driven decision-making
• Overcoming Data Challenges: Strategies to build robust AI models despite limited datasets
• Success Stories: Case studies showcasing AI and PAT integration for improved yield, efficiency, and product quality in cell and gene therapy manufacturing and process development

This workshop provides attendees with actionable strategies and expert guidance to navigate the complexities of tech transfer, ensuring a streamlined and successful transition from development to clinical manufacturing.

1. 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.

2. 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.

3. Navigating Regulatory Compliance and Tech Transfer Challenges

   • Explore key regulatory considerations in tech transfer.

   • Understand compliance challenges and how to effectively navigate regulatory requirements.

4. 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.

5. 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.

6. Expert Insights and Practical Advice

   • Gain real-world insights from experienced professionals on avoiding common tech transfer pitfalls.

   • Participate in interactive discussions, including a regulatory-focused AMA and a panel discussion on overcoming challenges in tech transfer.

Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.

Some of the topics to be discussed include:

• CDMO Selection: Models and Process
• Contract and Quality Agreement Negotiation
• Tech Transfer: Best practices and risk management for external tech transfers
• Digital Transformation in Tech Transfer
• Quality Assurance and Quality Control
• Life Cycle & Relationship Management

• Improvements and innovations in cell line development & engineering to increase yields for gene therapies
• Application of producer cell lines to lower transfection vector costs: Data for large scale use of stable cell lines
• Scalability of producer cell lines? Case studies on experiences at larger scales

• Improvements and innovations in cell line development & engineering to increase yields for gene therapies
• Application of producer cell lines to lower transfection vector costs: Data for large scale use of stable cell lines
• Scalability of producer cell lines? Case studies on experiences at larger scales

• Case Studies: Success stories of regulatory approvals and commercialization
• Overcoming Hurdles: Key challenges from R&D to approval—and how to tackle them
• Scaling Up: Strategies for manufacturing, validation, and CMC data packages
• Regulatory Insights: Feedback from authorities on characterization, specifications, and approval pathways
• Post-Approval Management: Long-term data tracking, durability studies, and lifecycle strategies
• End-to-End Logistics: Mastering supply chain and global distribution
• Pathways to approval –Case study on the journey from discovery to commercial development and beyond

• Internalising or externalising manufacturing – What are the decision points?
• How to select CDMO partners? – Industry criteria for selection
• When / what stage of development should you bring on second CDMO?
• Understanding CDMO capacities, timelines, and capabilities
• Qualification, and quality agreements for commercial manufacturing
• Tech transfer: best practices and risk management
• Strategies for transferring product from development phase to CDMO for large scale production: How to scale up?
• Lessons learnt from transferring to CDMO and vice versa
• Increasing skills and experience in manufacturing of next generation CGT products
• Partnership and communication – how to partner most effectively with CDMOs?

Regulatory expectations for potency and release testing are evolving, particularly with the rise of advanced biologics such as CGTs, ADCs, and bispecific antibodies. Companies must navigate challenges in assay standardization, surrogate potency methods, and comparability assessments while ensuring compliance with global regulatory frameworks. This session, led by industry experts and regulatory specialists, will provide practical insights into meeting the latest standards, including ICH Q6B (Specifications for Biotechnological/Biological Products), USP General Chapter <1032> (Design and Development of Biological Assays), and FDA guidance on potency testing for gene therapy products. Attendees will gain actionable strategies for improving assay validation, reducing variability, and streamlining regulatory approval pathways.