Sarepta Therapeutics faces a crisis after the death of a third patient linked to its gene therapies this year. The latest fatality involved a 51-year-old man with limb girdle muscular dystrophy (LGMD) in the Phase I DISCOVERY trial evaluating SRP-9004. This followed two deaths in patients treated with Sarepta’s marketed Duchenne muscular dystrophy therapy, Elevidys. Despite an FDA request to halt Elevidys shipments, Sarepta rejected compliance, citing no new safety signals in ambulatory patients. The deaths, tied to acute liver failure, raise profound challenges for Sarepta’s pipeline and regulatory relations, igniting broader concerns for AAV-based gene therapy risks.