The sweet cell of success: key considerations for the sourcing and production of pluripotent cell lines for therapeutic development

Cell sourcing and cell line production are two imperative challenges for the production of pluripotent stem cell (PSC)-based therapeutics. Regulatory guidelines published by the FDA, EMA, and other regulatory authorities around the world are still evolving to appropriately accommodate cellular therapies and have yet to achieve full harmonization between jurisdictions. In parallel, the industry continues to develop improved methods for production and characterization of PSC-based therapeutics that seek to enhance manufacturing performance and improve overall product safety. This article explores the current regulatory requirements for PSC sourcing, including donor eligibility, consent, and testing within leading and rapidly growing cell therapy jurisdictions. In the second section of this article, key considerations for PSC line derivation and banking are described, including methods for reprogramming, incorporation of genetic modifications, and cell bank production. Finally, strategies for PSC line testing are discussed, including considerations for genome editing and potency assurance, strategies to reduce downstream drug product testing requirements, and a potential approach to overcome theoretical BSE/vCJD risk in existing banked lines. Finally, thoughts on how best to align cell line production with critical product development activities, such as regulatory interactions and nonclinical studies are presented.

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