Allogeneic islet products for type 1 diabetes: Navigating nonclinical and manufacturing regulatory expectations

Le résumé fourni par la source

Next-generation allogeneic islet cell products-including stem cell (SC)-derived, gene-edited, and encapsulated islets-offer scalable and curative solutions to restore physiological glucose control in type 1 diabetes. The critical path to success for these complex therapies hinges on robust nonclinical pharmacology and toxicology evidence and rigorous, scalable, and well-controlled manufacturing. We discuss strategies for essential nonclinical data for First-in-Human trials, emphasizing SC-specific risks, question-specific model selection, stage-by-stage process characterization, and safety, identity, purity, and potency testing. This paper serves as a resource for developers, highlighting best practices and regulatory expectations to advance transformative T1D cell therapies from bench to clinic.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.