"RGX-202 is the only next generation gene therapy for Duchenne in a pivotal phase trial. The new data from the age 1-3 cohort builds on the favorable safety and efficacy profile seen in ages 4 and ...

Its product candidates include the NAV Technology Platform, which consists of exclusive rights to novel adeno-associated viral vectors, and therapeutic programs such as RGX-314, RGX-202 ...

RGX-202 shows the highest reported microdystrophin levels in ambulatory patients age 8+ among gene therapies. Regenxbio plans to submit a BLA for RGX-202 in mid-2026, with Phase 3 enrollment ...