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Regenxbio Rises After Duchenne Update And Sarepta Safety Scare

 
• By Andrew McConaghie

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

Regenxbio
• Source: Alamy

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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