The company intends to leverage the accelerated approval pathway, citing positive clinical trends and strong biomarker data. The upcoming application will feature a comprehensive safety dataset drawn from 63 participants across pivotal and confirmatory studies, alongside efficacy results from 30 pivotal study patients. CEO Curran Simpson noted that the data demonstrates significant functional improvements and a robust correlation between microdystrophin expression and clinical outcomes.

RGX-202 is engineered to include the C-Terminal domain, a feature the company claims distinguishes it from other therapies by better preserving muscle function. With manufacturing already underway at its facility in Maryland, the company is positioning its supply chain for a potential market launch in late 2027. Recent trial data indicated that over 93% of patients achieved 10% microdystrophin expression by the 12-week mark, meeting the program's primary endpoint.