Shares of Sarepta Therapeutics SRPT plunged over 30% in Friday pre-market after a clinical trial participant died following treatment with a gene therapy, marking the third death linked to the company’s experimental treatments.
What Happened: Sarepta Therapeutics confirmed the death of a 51-year-old man who was part of a Phase 1 trial for a gene therapy aimed at treating limb-girdle muscular dystrophy, according to BioCentury. The patient died of liver failure last month after being administered the experimental treatment.
The company, already under increased scrutiny for safety issues, had not disclosed this death in a recent corporate update. The update revealed the company’s plan to lay off hundreds of employees after two patients died while receiving treatment for Duchenne muscular dystrophy.
The Food and Drug Administration (FDA) is currently investigating the deaths of the two teenagers who were treated with Elevidys, Sarepta’s approved Duchenne muscular dystrophy gene therapy, which now comes with a black box warning regarding acute liver injury and failure.
In a statement, Sarepta acknowledged the inherent risks in clinical trial participation and expressed gratitude towards the “courageous patients and families who participate.” The company also stated, "While we do everything possible to ensure patient safety, there is inherent risk in clinical trial participation.”
Why It Matters: The recent death adds to the growing safety concerns surrounding Sarepta’s gene therapies. The company’s stock had surged in July 2025, following a strategic restructuring and cost-cutting measures aimed at focusing on high-value, high-impact programs.
However, the FDA’s request for a black box warning for acute liver injury and failure on Elevidys, Sarepta’s approved gene therapy for Duchenne muscular dystrophy, had already raised eyebrows.
The undisclosed death of the trial participant, coupled with the ongoing FDA investigation into the deaths of two teenagers treated with Elevidys, may further impact investor confidence in the company and its gene therapies.
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Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.
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