Sarepta slashes workforce, scales back research after Duchenne gene therapy setbacks

Sarepta Therapeutics is laying off about 500 employees and halting several research programs after safety concerns and deaths linked to its Duchenne muscular dystrophy gene therapy, Elevidys.

Why it matters: The cuts reflect a sharp pivot by Sarepta to stabilize finances and preserve its future, following an 80% stock drop and regulatory challenges. The fate of Elevidys, a first-of-its-kind therapy, is now uncertain, impacting patients and the broader gene therapy field.

Backstory: Elevidys was the first gene therapy approved in the U.S. for Duchenne muscular dystrophy in 2023. Despite initial promise, recent patient deaths triggered FDA action and reduced the eligible patient pool. Sarepta halted sales to higher-risk non-ambulatory patients and paused confirmatory trials.

• The FDA also announced this week that a black box warning over liver failure risks will be added to Elevidys.

Big picture: Sarepta’s future now depends on navigating FDA scrutiny, maintaining revenue from its other Duchenne drugs, and advancing new RNA-based therapies. The layoffs and research pause are part of a bid to save $400M annually and retain profitability amid growing uncertainty.

• Elevidys sales dropped from $375M in Q1 to $282M in Q2 and are expected to stabilize at around $500M per year.

What’s next: The company intends to focus its resources on RNA drugs developed with Arrowhead for rare neurological disorders.