RegenXBio continues to report positive biomarker data from their AAV-microdystrophin gene therapy trial

AAV-microdystrophin gene therapy trial

RegenXBio reported new interim data from 2 additional participants in the Phase 1/2 portion of the AFFINITY DUCHENNE trial of RGX-202, an AAV8-microdystrophin gene therapy. In a 3 year old participant, microdystrophin levels at 12 weeks were 122.3% of control, and in a patient aged 7 years old microdystrophin expression was measured to be 31.5% of control. 

In all, RGX-202 has been well tolerated in 12 individuals, with no serious adverse events reported. In November 2024, data from the first 5 individuals treated showed functional improvements compared to natural history controls.  

RegenXBio indicates that we can expect to see additional Phase 1/2 data, including additional functional data, in the first half of this year. They expect to pursue FDA approval via the accelerated approval pathway in 2026.  

Press Release: https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-reports-positive-biomarker-data-affinity-duchenner

Letter to the community:

REGENXBIO RGX-202 Community Letter_vFINAL 24Mar2025Download