Regenxbio has announced the completion of pivotal trial enrollment and the start of commercial-scale manufacturing for its gene therapy candidate RGX-202, designed to treat Duchenne muscular dystrophy (DMD). According to the company, these milestones mark major progress toward potential regulatory submission and commercialization.
The company said that all participants in the pivotal AFFINITY DUCHENNE trial have been dosed, meeting the planned enrollment targets. The study is evaluating the safety and efficacy of RGX-202, which uses Regenxbio’s proprietary NAV AAV8 vector platform to deliver a shortened version of the dystrophin gene aimed at restoring muscle function in boys with DMD.
Regenxbio also confirmed that commercial-scale manufacturing of RGX-202 has begun at its in-house facility in Rockville, Maryland. According to the company, this represents an important step in ensuring future supply chain readiness and scalability in anticipation of potential market launch.
The company claims that RGX-202 has demonstrated encouraging data from earlier trials, with plans to present updated results from the ongoing studies in early 2026. Regenxbio added that it expects to use these findings to support future regulatory filings with the U.S. Food and Drug Administration.
According to the company, the Duchenne program is part of its broader gene therapy portfolio targeting neuromuscular and rare diseases, as Regenxbio continues expanding its manufacturing and clinical capabilities to advance next-generation treatments.
