REGENXBIO announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker data for RGX-202, REGENXBIO’s potential best-in-class, investigational gene therapy for Duchenne muscular dystrophy. The functional data demonstrate consistent benefit among dose level 2 participants at 9 and 12 months following treatment with RGX-202.
Aravindhan Veerapandiyan, M.D., of Arkansas Children’s Hospital said:
“These findings suggest that the microdystrophin expression observed with RGX-202 is leading to meaningful functional improvements, even in individuals with DMD who are expected to experience functional decline. These Phase I/II results, demonstrating functional improvements and favourable safety profile, underscore the potential of RGX-202 as a treatment option for individuals with DMD. It is both encouraging and essential to have innovative therapies that can help preserve muscle integrity and substantially delay disease progression. I’m enthusiastic about the continued development of RGX-202 and the promise it holds for the Duchenne community.”
Please read the full press release for more information.
We remain steadfast in our commitment to supporting individuals and families living with Duchenne. If you have any questions or concerns regarding this news, please do reach out to us at info@actionduchenne.org.
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