biostudies-literatureAzzag KNIAID NIH HHSNational Institutes of HealthNIAMS NIH HHSDuchenne Research FundNIH HHS5099-5117https://www.ebi.ac.uk/biostudies/studies/S-EPMC12338975biostudies-literatureUnknown33(10)Pluripotent stem cell (PSC)-derived therapies are in clinical trials of terminally differentiated or transiently required cell types, but to date no PSC-derived trial contributing tissue-specific stem cells or any PSC-based skeletal muscle regeneration trial has been approved. We describe a process in accordance with the Current Good Manufacturing Practice (CGMP) to generate large-scale cryopreserved PAX7-induced myogenic progenitors, which reconstitute both fibers and satellite cells, from PSCs. We subjected the clinical-grade cell product MyoPAXon to biodistribution, toxicity, and tumorigenicity studies in mice under Good Laboratory Practice conditions with no adverse effects and demonstrate long-term engraftment (>1 year) and efficacy in dystrophic mice. Transplantation of 37-60 millionMolecular therapy : the journal of the American Society of Gene TherapyPreclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies.PMC12338975R34 AR081536R01 AR081882R01 AR078571U24 AI126683P40 OD028116R01 AR078624Norris BFlory CRamachandran SKim HSeelig DKyba MKiley JPerlingeiro RCRHocum Stone LLEverett JShappa Faustich JWagner JEAhlquist ASchumacher RJMagli AKang PBRangarajan PBushman FMcKenna DHAzzag KSumstad DKadidlo DCrist SBGraham MLfalsePreclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies.Pluripotent stem cell (PSC)-derived therapies are in clinical trials of terminally differentiated or transiently required cell types, but to date no PSC-derived trial contributing tissue-specific stem cells or any PSC-based skeletal muscle regeneration trial has been approved. We describe a process in accordance with the Current Good Manufacturing Practice (CGMP) to generate large-scale cryopreserved PAX7-induced myogenic progenitors, which reconstitute both fibers and satellite cells, from PSCs. We subjected the clinical-grade cell product MyoPAXon to biodistribution, toxicity, and tumorigenicity studies in mice under Good Laboratory Practice conditions with no adverse effects and demonstrate long-term engraftment (>1 year) and efficacy in dystrophic mice. Transplantation of 37-60 million2025-01-01T00:00:00Z2025 Oct2026-07-15T13:13:30.338Z2026-07-05T03:08:14.213ZS-EPMC123389754068227210.1016/j.ymthe.2025.07.007