Sarepta Therapeutics is cutting approximately 36% of its workforce—about 500 jobs—following the deaths of two Duchenne muscular dystrophy patients treated with its gene therapy Elevidys. The company is shifting focus from gene therapies to siRNA-based treatments for rare genetic diseases. Elevidys use has been restricted due to acute liver failure risks, prompting an FDA-mandated black box warning. Sarepta seeks FDA approval for an enhanced immunosuppression regimen and plans new clinical trials to mitigate liver toxicity in non-ambulatory patients. Despite investor concerns, the restructuring aims to improve financial stability.