REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver microdystrophin via AAV8 through a one-time IV infusion.
In the interim functional results from seven participants treated at the pivotal dose level (2×1014 GC/kg), RGX-202 continues to demonstrate evidence of positively impacting disease trajectory on North Star Ambulatory Assessment (NSAA) and all timed function tests (Time to Stand, 10 Meter Walk-run, Time to Climb) at one year when compared to external control using propensity score weighting. According to REGENXBIO, biomarker data from the Phase I/II study continues to support consistent, high expression and transduction of RGX-202 microdystrophin (n=13), and data continues to support favorable safety and tolerability.
REGENXBIO has indicated that the company expects to share topline pivotal data in early Q2 2026 and plans to request a pre-BLA meeting with the FDA in mid-2026. These results are encouraging and PPMD looks forward to future updates from REGENXBIO, which we will share with the community as soon as information becomes available.
Read REGENXBIO’s press release here.
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