Sarepta Therapeutics faces mounting scrutiny following the confirmation of a third patient death linked to its gene therapies. The latest fatality involved a 51-year-old man treated for limb-girdle muscular dystrophy in a Phase I trial, who succumbed to acute liver failure. This follows two teenagers who died after receiving Elevidys, Sarepta’s FDA-approved gene therapy for Duchenne muscular dystrophy. In response, the FDA has requested Sarepta to halt shipments of Elevidys, citing concerns over liver risks, and is contemplating a market withdrawal. Sarepta’s CEO Douglas Ingram defended the company’s transparency despite criticism over delayed disclosures. Industry experts and former FDA officials acknowledge the complexity but highlight the serious safety signals associated with these AAV-based therapies.