Sarepta Therapeutics is undergoing a major restructuring by laying off approximately 500 employees, about 36% of its workforce, and pausing research on several gene therapy programs for limb-girdle muscular dystrophy. This strategic pivot, prompted by safety concerns following two patient deaths due to liver failure linked to its Duchenne muscular dystrophy gene therapy Elevidys, aims to save over $400 million annually and improve financial flexibility. The FDA has requested a black box warning for Elevidys for acute liver injury, particularly in older, non-ambulatory patients, leading Sarepta to halt treatment for this group and suspend a confirmatory Phase 3 study. The company plans to seek FDA approval for an enhanced immunosuppressive strategy to mitigate liver toxicity, while continuing collaboration with Arrowhead Pharmaceuticals on skeletal muscle disease therapies. Investors responded positively to the restructuring announcement despite Sarepta’s share price decline earlier in the year.