Chengyuan Press, Kevin D'Amour, Nicholas Mamrak, David Pepperl, Robert H Kutner, Diana M Colleluori, Melanie L Graham, Michael A Brehm, Nasir Hussain, Esther Latres, Marjana Marinac
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引用次数: 0
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.
期刊介绍：
Stem Cell Reports publishes high-quality, peer-reviewed research presenting conceptual or practical advances across the breadth of stem cell research and its applications to medicine. Our particular focus on shorter, single-point articles, timely publication, strong editorial decision-making and scientific input by leaders in the field and a "scoop protection" mechanism are reasons to submit your best papers.
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