Regenxbio Rises After Duchenne Update And Sarepta Safety Scare

Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.

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...

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