Sarepta Therapeutics is facing a third confirmed death tied to its experimental gene therapies. The company paused its Phase 1 trial for SRP-9004 on July 17, one day after internal documents revealed that a 51-year-old man had died from acute liver failure in June. He was enrolled in the limb-girdle muscular dystrophy trial. Sarepta did not mention the death in its July 16 corporate update, which announced a $400 million cost-cutting plan, 500 layoffs, and a pivot away from most gene therapy programs. The omission has triggered backlash across biotech circles and investor forums.
The company later admitted the death occurred and said regulators were informed “in an appropriate and timely manner.” That statement came only after Bloomberg and BioCentury published the details. Sarepta’s CEO claimed the death was unrelated to the restructuring, but the timeline is tight. The trial was paused within 24 hours of the corporate update. SRP-9004 was deprioritized. No mention of the fatality was made during the investor call.
This marks the third death linked to Sarepta’s gene therapy portfolio in 2025. Two teenage boys died earlier this year after receiving Elevidys, the company’s approved treatment for Duchenne muscular dystrophy. Both cases involved liver failure. The FDA responded by requiring a black box warning for acute liver injury and failure on Elevidys packaging. Sarepta halted shipments to non-ambulatory patients and suspended dosing in its Phase 3 ENVISION study.
The latest death adds pressure to SRP-9003, another limb-girdle therapy still in development. Analysts warn that the same viral vector used in Elevidys and SRP-9004 could trigger similar toxicity. Sarepta says it will still seek FDA approval for SRP-9003 in late 2025. That plan now faces scrutiny.
Sarepta’s stock dropped 26.85% in premarket trading on July 18. Shares are down more than 80% year-to-date. The company’s restructuring includes a shift toward siRNA programs for Huntington’s disease, idiopathic pulmonary fibrosis, and myotonic dystrophy. Gene therapy is no longer the flagship.
The patient who died was a father of two. He volunteered for a trial targeting an ultra-rare disease. His death was not disclosed until reporters forced the issue. Sarepta’s silence is now part of the story.
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