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Regenxbio Sets Sights On 2026 Filing For DMD Gene Therapy

 
• By Jessica Merrill

The company has reached agreement with the US FDA on a pivotal trial for RGX-202 through an accelerated pathway.

(Shutterstock)

Regenxbio believes it has a best-in-class gene therapy in development for Duchenne muscular dystrophy (DMD) and functional improvement data from Phase I/II testing of RGX-202 suggest that could be so. The company announced the first functional data for patients treated with RGX-202 on 18 November and said it has reached alignment with the US Food and Drug Administration on a pivotal trial and potential pathway for accelerated approval.

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