Sarepta Therapeutics has enacted a major restructuring following safety concerns related to its Duchenne muscular dystrophy gene therapy Elevidys. The company is cutting approximately 36% of its workforce—about 500 employees—to achieve $400 million in annual cost savings. Sarepta plans to pivot its pipeline focus towards siRNA therapies, deprioritizing many gene therapy programs including those for limb-girdle muscular dystrophy. This move follows two reported patient deaths due to acute liver failure after Elevidys treatment, leading to the FDA requesting a black box warning for the therapy. Sarepta has paused Elevidys shipments for non-ambulatory patients and is working on enhanced immunosuppressive regimens to mitigate liver toxicity. Despite the challenges, preliminary Q2 revenue projections show continued Duchenne portfolio resilience. Leadership changes accompany the strategic shift, reflecting Sarepta’s effort to stabilize financially while addressing safety and regulatory demands.