A gene therapy that wins in the clinic but fails in the market or, worse, never reaches market, is not a success. For example, in July 2025, the FDA issued Complete Response Letters (CRL) to Ultragenyx’s UX111 AAV gene therapy for Sanfilippo syndrome and Capricor’s deramiocel cell therapy for Duchenne muscular dystrophy, citing CMC deficiencies and manufacturing facility observations as primary findings.1,2
In Ultragenyx’s case, the FDA acknowledged that robust clinical data existed, but the program…
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