Measurement of the biological activity is crucial for lot release, stability and any comparability exercise of all biological medicinal products. Potency assays should be implemented as early in clinical developments as possible because correlation with clinical performance could help in future identification of sub potent batches. However, development of an appropriate potency assay is often challenging, especially for products with complex or not well-defined mechanisms of action.

Gene editing (GE) tools are used to achieve targeted modifications of genome sequences resulting in gene inactivation, modifications or insertions at specific locations, either in vivo or ex vivo. This allows the generation of very diverse cell phenotypes that can be used in the clinic to treat a high array of diseases. Thus, developing specific potency assays for gene edited medicinal products can have additional complications.

This session will aim to review FDA’s recently released draft guideline on potency assurance for cell and gene therapy products. It will also cover issues to be considered when designing a potency assay for ex vivo genome-edited products or products that are intended to edit the genome in vivo, considering the different GE tools and possible mechanisms of actions. The session will also present the particular challenges of developing a potency assay for a GE product intended to knock-out a gene.

Session Speakers:

Potency Assurance for Cellular and Gene Therapy Products

Andrew Byrnes, CBER, FDA

Potency Development for an in Vivo AAV Gene Editing Therapy

Debaditya Bhattacharya, ElevateBio

Approaches to Potency Assays for CRISPR Genome Editing Therapeutics

Kristy Wood, Intellia Therapeutics, Inc.

Additional Panelist:

Keith Wonnacott, Lexeo Therapeutics, Inc.

Marcos Timon

Appearances