Sarepta Shares Dive Post Patient Death in LGMD Gene Therapy Trial

Sarepta Therapeutics’ shares plummeted 36% after a third patient treated with its LGMD gene therapy SRP-9004 died from acute liver failure in a Phase 1 trial. This follows two earlier patient deaths linked to Sarepta's Duchenne gene therapy Elevidys, prompting pipeline development pauses and scrutiny. Sarepta rejects FDA’s request to halt Elevidys shipments, citing no new safety signals in ambulatory patients. The situation underscores ongoing safety challenges in AAV-based gene therapies.