New Phase I/II data from its Duchenne gene therapy candidate, RGX-202, have boosted Regenxbio, just as new concerns emerged about the safety of Sarepta’s established gene therapy, Elevidys (delandistrogene moxeparvovec).
Key Takeaways
- Two more children have shown raised microdystrophin levels in RGX-202’s Phase I/II study
Regenxbio presented new data from two more young children who received RGX-202 in the Phase I/II AFFINITY DUCHENNE study at the 2025 Muscular Dystrophy Association (MDA) conference in Dallas, TX, on 19 March
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