Sarepta Shifts Focus to siRNA Platform, Lays Off 500 After Elevidys Receives Black Box Warning

Sarepta Therapeutics laid off over a third of its workforce, equating to approximately 500 employees, as part of a major corporate restructuring1 2 3 4 5.

The company is pivoting its research and development focus to high-impact programs within its siRNA (small interfering RNA) platform, reprioritizing away from most gene therapy programs, particularly in limb-girdle muscular dystrophy1 2 4.

Sarepta's marketed gene therapy for Duchenne muscular dystrophy, Elevidys, was linked to the deaths of two teenage patients due to acute liver failure, leading the company to add a black box warning for acute liver injury and failure to Elevidys’ FDA label1 2 4.

Sarepta paused or discontinued several development programs to boost financial sustainability, aiming to save around $400 million per year and ensure long-term positive cash flow3.

Despite safety concerns, Elevidys is not expected to be pulled from the market, with analysts citing the new black box warning as reducing regulatory risk for its continued approval1.

Strategic pipeline focus is now on siRNA-based therapies for diseases such as facioscapulohumeral muscular dystrophy, idiopathic pulmonary fibrosis, and Huntington’s disease2.