Regenxbio reports positive Duchenne gene therapy trial results, targets FDA approval by 2027

Regenxbio announced that its gene therapy for Duchenne muscular dystrophy achieved the required endpoints in a pivotal clinical trial.

The data met the study's predefined criteria, which the company says positions the program for a regulatory filing. The biotech now plans to submit a marketing application to the U.S. Food and Drug Administration with a target approval date in 2027.

If approved, Regenxbio would become one of the first companies to bring a gene-based treatment for Duchenne to market, potentially expanding therapeutic options for patients with the rare, progressive disease.