Sarepta Therapeutics shares rise on early promise for rare disease drugs

Damian Garde is a reporter at large, live and feature journalism, covering the global drug industry and contributing to STAT’s industry-leading events.

Sarepta Therapeutics’ next crop of medicines for muscular dystrophy appear to be safe and effective in early clinical trials, the company said, a positive though incremental update for a drugmaker rebuilding its business after a tumultuous 2025.

Shares of Sarepta rose more than 20% in early trading Wednesday after the company presented preliminary data on a pair of treatments for rare, muscle-wasting diseases. Sarepta, which laid off more than one-third of its workforce last year after multiple patient deaths derailed its top-selling gene therapy for muscular dystrophy, has staked its future on a pipeline of unproven treatments for related conditions.

The early data on two of those drugs, SRP-1001 and SRP-1003, will be followed by more substantive updates later this year, Sarepta said, and pivotal studies will come in 2027.