Following two recent patient deaths caused by acute liver failure, the FDA has launched an investigation into Sarepta Therapeutics' gene therapy Elevidys for Duchenne muscular dystrophy. The regulator is evaluating potential further actions but has not specified them yet. Sarepta attributes the liver injuries as known risks linked to AAV vector-based gene therapies and maintains Elevidys’ positive benefit-risk profile. In response to the safety concerns, Sarepta has suspended shipments for non-ambulatory patients and is considering enhanced immunosuppression regimens. This situation significantly impacted Sarepta's stock value, which plummeted about 80% since March.