Potency testing is a critical part of the assessment and release of cell therapy products. These functional assays provide quantitative assessments of the biological activity of a cell or gene therapy product that are associated with that product’s in vivo mechanism(s) of action (MOA). Current state-of-the-art analytical methods still rely mainly on in vitro assays including immune-assays, cell-based proliferation, cytokine release as well as cytotoxicity assays and involve complex technology such as flow cytometry. In some cases, these assays may lack the required robustness, simplicity, sensitivity and/or throughput required to function well in the QC GMP environment. In other cases these assays reflect an average measurement that represents the entirety of a sample and do not provide information regarding the function associated with individual cell phenotypes. Next generation potency assays are needed which are easy to use, robust and provide a more comprehensive evaluation of the quality attributes of the Drug Substance (i.e. Viral Vector, plasmid) and Drug Product.

This session will involve case studies highlighting innovative approaches being used to develop potency assays in support of programs from early phase to late phase and commercialization. Assay design strategies focused on reflecting a cell therapy product’s complex MOA, while balancing this with attributes that are important for a method to function effectively to perform QC release and stability testing will also be included. Points to consider for critical reagents used to support potency assays for cell/gene products as well as viral vector will also be discussed.

Session Speakers:

Rethink the Potency Paradigm for Gene Therapy Products

Xiaohui Lu, Ultragenyx

Success Story: Luxturna Potency Assay Development to Validation

Ravindra Kumar, Spark Therapeutics

Unleashing the Power: Assessing CAR T Cell Therapy Potency and Maximizing Life Cycle Management

Seema Bansal, Bristol-Myers Squibb

Additional Panelists:

Andrew Byrnes, CBER, FDA

Max Tejada

Appearances

Parallel Session 3: Lifecycle Approaches to Potency Assay Selection

Session Chairs

Speakers