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Allogeneic islet products for type 1 diabetes: Navigating nonclinical and manufacturing regulatory expectations
Chengyuan Press1, Kevin D'Amour2, Nicholas Mamrak1
1Breakthrough T1D, New York, NY, USA.
Abstract:
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.
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