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

  • Zhu Pirot Director & Technical Leader - Platform CGT Analytical Development, Bayer
  • Mariska ter Haak Senior Director, Analytical Development, IN8Bio
  • Darren Kamikura Scientific Director, Potency & Functional Characterization, Bristol Myers Squibb
  • Shibani Mitra-Kaushik Head, Product Control Analytics, Cell Therapies, Takeda Pharmaceutical Co. Ltd.

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

  • Carl Simon Biologist, National Institute of Standards & Technology - NIST

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

  • Hadar Adams Director, Analytical Development, Atara Biotherapeutics Inc.

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

  • Abid Mattoo Senior Scientist, Clinical Stage Analytical Development, Takeda Pharmaceutical Co. Ltd.

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

  • John Yu Director, Cell Product Characterization, Sana Biotechnology

Understanding Mechanistic Action to Develop Meaningful Assays

12:00 pm Early-Stage Potency Assay Development for Regenerative Cell Therapies

  • David Dilworth Senior Scientist, Assay Development, BlueRock Therapeutics

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

  • Rita Barcia Vice President - Process Development & Operations, Independent Expert

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

  • Dong Xu Associate Director, Cell Therapy Analytical Development, TScan Therapeutics

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

  • David Schwalb Director, Analytical Development, Cell & Gene Therapies, Pacira Biosciences
  • Joseph Lee Senior Director, Independent Expert
  • Mariska ter Haak Senior Director, Analytical Development, IN8Bio
  • Matthew Duvernay Senior Director, Quality Control, Cartesian Therapeutics

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? 

5:15 pm Chair’s Closing Remarks

5:30 pm End of Conference Day 1