Regenxbio (RGNX) said Wednesday that new interim data from two additional patients with Duchenne muscular dystrophy in a phase 1/2 study of investigational gene therapy RGX-202 added to "consistent, robust" microdystrophin and transduction levels across all treated ages.
The company said microdystrophin expression was measured at 122.3% in a 3-year-old patient and 31.5% in a 7-year-old patient compared to control.
Regenxbio said it is enrolling ambulatory patients 1 year old and older for a phase 3 trial and is on track to submit a biologics license application with the US Food and Drug Administration in mid-2026.
Shares of the company were up more than 5% in recent trading.
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