Day One Agenda
8:00 am Check-In & Coffee
9:00 am Chair’s Opening Remarks
9:10 am Industry Leader’s Fireside Chat: Cell Therapy Potency Assays
Synopsis
• What progress have we made since the potency assays produced for the first commercialized cell therapy products?
• What challenges are we still experiencing?
• What do we think about the current regulatory guidance, and how do we interpret it?
10:00 am Analytics Supporting CMC Potency Activities in Cell Therapies – Current Challenges
Synopsis
• Discussing the rapidly changing expectations in analytical development activities supporting cell therapies – emphasis on potency
• Exploring viral vs non-viral delivery – potency for both drug substance (intermediate) and drug product
• Outlining key considerations for the development of potency assays for cell therapies
10:30 am Mechanism of Action, Potency & Efficacy: Considerations for Cell-Based Assays
Synopsis
• A framework for MOA, potency and efficacy is presented that is consistent with regulatory guidelines while accommodating what has been learned from the approved cell therapies
• Potency ≠ efficacy
• Can a therapy be “potent but not efficacious”?
11:00 am Morning Break & Structured Networking
Synopsis
As this community reunites, this session will provide valuable networking time with your peers, enabling you to forge new and lasting connections.
CAR-T & TCR-T
Establishing a Potency Assay Matrix for T Cell Products
12:00 pm Matrix-based Approach to Potency Assay Development for Adoptive T Cell Therapies
Synopsis
• Developing phase-appropriate T cell potency assays, balancing critical quality attributes and available technologies
• Leveraging compliant characterization technologies to gain a well-rounded understanding of T cell functionality
• Establishing a potency assay matrix, maximizing correlation to clinical efficacy while minimizing late-phase risk
12:30 pm Evolution of a Cell Based Potency Assay from Early to Pivotal Clinical Stage
Synopsis
• Early assay development strategy with a focus on model cell lines and controls
• Transformation in the assay from supporting process development to drug product release and stability
• Challenges in developing pivotal stage assays in cell therapy and use of matrix potency approach for complex cell therapy products
Other Cell Types
Understanding Mechanistic Action to Develop Meaningful Assays
12:00 pm Early-Stage Potency Assay Development for Regenerative Cell Therapies
Synopsis
• Leveraging advanced technologies to increase understanding of drug product mechanism of action towards establishing a meaningful matrix of functional assays
• Developing phase appropriate early-stage potency assays for the characterization of pluripotent stem cell-derived regenerative medicines
• Moving from a characterization matrix to a suitable assay for drug product release and stability testing
12:30 pm Navigating Potency in Novel Cell Therapies: Macrophages & Mesenchymal Stem Cells
Synopsis
• Leveraging stage-appropriate potency assays
• Evolving your potency assay ready for pivotal
• Developing novel ways to assess biological activity
1:00 pm Lunch Break & Networking
Characterizing Effectively to Maximize Correlation with Clinical Efficacy
2:00 pm Analytical Assay Development for a TCR-T Cell Therapy Product with Transposase Integration
Synopsis
• Providing analytical support for TCR-T cell process
optimization
• Developing transposase-specific analytical assays
• Balancing between efficacy and safety
2:30 pm Panel Discussion: Establishing a Correlation Between Potency & Clinical Efficacy
Synopsis
• What should we be measuring beyond IFN-gamma? What about cytotoxicity, proliferation, or others?
• What measure(s) correlates best with clinical efficacy in T cell products?
• If we can’t correlate with clinical efficacy, what should we be concerned with?
Understanding Mechanistic Action to Develop Meaningful Assays
2:00 pm Assays to Measure Potency in Clonal Neoreactive T Cells (cNeT)
Synopsis
• Developing potency methods for a cNeT product and the associated challenges
• Selecting and introducing assays into a GMP environment for batch release testing
• Discussing challenges in potency method validation
2:30 pm Panel Discussion: Measuring Potency in a Non-Targeted Therapy
Synopsis
• How do you define potency for a non-targeted therapy?
• What should you measure when you don’t have a specific target cell to kill?
3:00 pm Afternoon Break & Poster Session
Creating a Phase Appropriate Potency Assay Design & Development Strategy
4:00 pm Building a Strategy for Potency Assay Development
Synopsis
• Reviewing considerations for lifecycle management of potency assays
• Supporting product comparability needs through all stages of product development
• Exploring the interplay of manufacturing process development and potency assay development
• Understanding considerations for meeting health authority requirements for potency assays
4:30 pm Panel Discussion: Potency Assay Development Over the Development Lifecycle
Synopsis
• Should we start potency assay development early? Or should we wait until clearer signals emerge later in subsequent phases
that a therapy is likely to be safe and efficacious?
• How do we translate early phase potency assays into late phase assays?
• At what stage do you begin to qualify and validate?