Session Chairs: Diane Blumenthal, Dianthus Biopharma Consulting, LLC, Mark Galbraith, TRANSPOSON Consulting Group, LLC., and Mayumi Holly, Tune Therapeutics

Potency is a critical quality attribute that is intended to demonstrate the ability of the drug product to effect a given result and be reflective of the intended mechanism of action. As such, Potency plays a critical role in the development of cellular and gene therapies. Potency assays present analytical, CMC, and regulatory challenges for cellular and gene therapies. The biological activity, intricacies of cellular and gene therapies, and mechanism of action are often complex or not well understood, requiring development of bespoke methods, which must meet both the needs of evaluating manufacturing consistency as well as clinical efficacy. This session will dive into key challenges of potency assays facing drug developers today.

Challenges with the industry standard potency assay for AAV (TCID50) and strategies for development of new methodologies: What information is the TCID50 providing? How can TCID50 be improved to provide more meaningful and consistent data? Can TCID50 be replaced as other assays are developed and what is the strategy for replacement?

In vitro potency assays for CAR-T cell therapies: Balancing QC-friendly assays for lot release, ensuring manufacturing consistency, short turnaround time, and clinical relevance.

US FDA Draft Guidance on Potency Assurance for Cellular and Gene Therapies and Challenges with applying this guidance. How the US FDA guidance differs from guidance provided by other agencies with particular focus on guidance provided by the EMA.

Panelists:

Gaël Debauve, UCB S.A.

Neil Haig, BioBrilliance, CMC Consulting

Max Tejada, Genentech, a Member of the Roche Group

Keith Wonnacott, Lexeo Therapeutics, Inc.

Mark Galbraith

Appearances