08:00 AM EDT, 06/05/2025 (MT Newswires) -- Regenxbio ( RGNX ) said Thursday that new interim data from a phase I/II trial of its experimental RGX-202 gene therapy for Duchenne muscular dystrophy showed "positive" functional, safety and biomarker results.

"At the same dose being used in the pivotal trial, RGX-202 participants exceeded natural history across all key measures, including the North Star Ambulatory Assessment, which is striking," Chief Medical Officer Steve Pakola said. "We are particularly encouraged by the outperformance observed in older patients."

The latest data support Regenxbio's ( RGNX ) planned biologics license application submission using the accelerated approval pathway in the middle of next year, Pakola said.

Duchenne muscular dystrophy is a severe type of dystrophy predominantly impacting boys.

The company's shares were up 4.1% in recent premarket activity.