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Regenxbio Says New Data Support Potential Gene Therapy for Duchenne Muscular Dystrophy
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Regenxbio Says New Data Support Potential Gene Therapy for Duchenne Muscular Dystrophy
Mar 19, 2025 8:40 AM

11:07 AM EDT, 03/19/2025 (MT Newswires) -- 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 ( RGNX ) 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.

Price: 8.20, Change: +0.40, Percent Change: +5.13

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