By FieldPulse Editorial · March 25, 2026
Tags: Clinical Trials, Pipeline
Sarepta Therapeutics released early human data showing a 90-93% reduction in disease-linked gene expression from a single dose of its FSHD drug candidate — a dramatic turn for a company that endured patient deaths and mass layoffs in 2025.
Sarepta Therapeutics is trying to write a new chapter. On March 25, the company released the first human clinical data from two siRNA programs — SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP-1003 for myotonic dystrophy type 1 (DM1) — and the early numbers were striking enough to send the stock up more than 20 percent. For SRP-1001, a single dose produced a 90 to 93 percent reduction in expression of the genes abnormally activated in FSHD1. The data also showed favorable tolerability and dose-dependent muscle exposure, two critical early signals that a drug is behav