Although relatively new to the biopharmaceutical industry, cell and gene therapy development and manufacturing are advancing rapidly. At Informa Connect’s September 2021 Cell & Gene Manufacturing & Commercialization Conference and Exhibition, held in Boston and online, presentations reviewed concerns that arise when processing complex therapies and highlighted some innovative strategies for surmounting those obstacles. Most of those approaches described during the event used data-driven solutions, with each step building on the information gained from the previous one. High-throughput technology platforms are being designed to identify targets for solid cancers and shorten vein-to-vein timelines. Developers also are engineering hematopoietic stem cells that “uncouple” the target expression between healthy and cancer cells, thus enabling a therapy to kill only cancer cells. In this eBook, BPI managing editor Maribel Rios relates major insights from the September event. First, she focuses on a presentation describing implementation of mechanistic modeling and simulations to increase production of adenoassociated virus (AAV) vectors for use in gene therapy delivery. The second section of the eBook explores the difficulties associated with designing and performing potency assays for advanced therapy products. Such therapies are much more complex than they were even a decade ago, and knowledge to assess their functionality, activity, vector infectivity, and other parameters has not kept up with the pace of product development. Rios summarizes three presentations addressing potency assay design and workflows, including the implementation of specific analytical technologies during clinical development.

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