AM23-SN-15-O: Critical Role of Analytics/Analytical Development for Cell and Gene Therapy Product Acceptance and Licensure (Enduring)

Blood banking and product safety testing has evolved over decades to ensure safe products and maximize testing efficiency. Innovations in the space have allowed for qualitative analysis of products and faster analytical processes based on scientific and technical discoveries. The Advanced Therapies development space is newly emerging, with very few licensed products, all in a narrow range of classes. In recent years, we have seen products stall at the licensure finish line because of an inadequate set of characterization and potency assays. In this space, 80% of the questions related to licensure filings are related to the CMC section – production and analytical variables. As the range of product types expands, the complexity of these analytics and the potential roadblocks to product licensure can only be expected to increase. These analytical development and testing challenges affect developers of Advanced Therapies throughout the development and production cycle – first starting materials, moving through in-process testing, and finally, focusing on finished product testing. Further, multiple levels of testing are required, including safety, quality, identity, purity & potency, with potency being especially challenging. This session will cover the critical burden of analytical development and testing in alleviating roadblocks to the licensure and commercialization of Advanced Therapy products. It will also look at the value of a strong analytical partner throughout development and production. Topics covered will include the importance of early analytical development, criticality of potency assays, description of newly developed flexible potency assay platforms, qualification of traditional donor screening assays for use in cell therapy safety testing, impact of a robust analytical development plan on successful licensure discussions with regulators and insights into possible future analytical requirements for cell and gene therapy products.