Potency is a critical quality attribute of biological products, defined by the US FDA as the specific ability of the product to cause the required therapeutically clinical benefit. Potency assay will leverage the product's mechanism of action to define the biological activity. The absence of rigorous mechanistic data for the majority of cell and gene therapy therapeutics currently in the process research pipelines has impeded efforts to design and validate indices of product potency. The current talk tries to summarize the principal and unique challenges facing the development of functionally relevant potency assays for cell and gene therapy products.

Learning Objectives:

1. General guidance of FDA for developing potency assays

2. Understand unique challenges for developing in-vitro potency assays