Regenxbio On Track For Pivotal Study After Interim RGX-202 DMD Data

Aug 01 2024

• By Alaric DeArment

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The company is hoping to become second to market with a DMD gene therapy after Sarepta’s Elevidys, which recently had a label expansion.

• Source: Shutterstock

Regenxbio Inc. is closer to becoming a potential competitor to Sarepta Therapeutics, Inc. in gene therapy for Duchenne muscular dystrophy (DMD) with Phase I/II interim data for its candidate RGX-202. Despite Sarepta’s Elevidys (delandistrogene moxeparvovec-rokl) currently having a monopoly on gene therapy treatment for DMD, there is likely enough room for RGX-202 to be second to market.

The Rockville, MD-based biotech announced interim data from the Phase I/II AFFINITY DUCHENNE trial in DMD patients aged 1-11. RGX-202...

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