Sarepta Therapeutics faces mounting challenges following the deaths of three patients linked to its gene therapies, notably the Duchenne muscular dystrophy treatment Elevidys and an investigational limb-girdle muscular dystrophy candidate. The FDA requested a black box warning for Elevidys due to acute liver injury risks, prompting Sarepta to halt shipments to non-ambulatory patients and develop enhanced immunosuppression protocols. Concurrently, Sarepta announced a sweeping restructuring, cutting 36% of its workforce (~500 jobs) and redirecting R&D focus from gene therapies to siRNA platforms targeting rare genetic diseases. This strategy aims to secure financial viability amid declining revenues and regulatory scrutiny, with Sarepta projecting $400 million in annual cost savings by 2026.