REGENXBIO(RGNX) Prnewswire·2025-03-19 13:15Core Insights - REGENXBIO Inc. reported positive interim data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, presented at the 2025 MDA Clinical & Scientific Conference [1][2] Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with RGX-202 being the only next-generation gene therapy in pivotal trials for Duchenne muscular dystrophy [2][9] - The company aims to improve lives through the curative potential of gene therapy and has a late-stage pipeline for various rare diseases [12] Clinical Trial Updates - The AFFINITY DUCHENNE trial is progressing towards completing enrollment in 2025, with a Biologics License Application (BLA) submission anticipated by mid-2026 [2][7] - New biomarker data from patients aged 1-3 showed microdystrophin expression levels of 122.3% compared to control in a 3-year-old patient, indicating robust expression across all ages [4][7] - RGX-202 demonstrated the highest reported vector genome copies (4.9-55.4) among approved or investigational gene therapies [6] Safety and Efficacy - As of February 21, 2025, RGX-202 was well tolerated with no serious adverse events (SAEs) reported, and common drug-related adverse events included nausea, vomiting, and fatigue [7][8] - The treatment has shown functional improvements exceeding external natural history controls, with patients demonstrating stable or improved function on the North Star Ambulatory Assessment (NSAA) [8] Future Prospects - The company plans to share additional Phase I/II functional data in the first half of 2025, reinforcing the potential of RGX-202 to alter the course of Duchenne muscular dystrophy [2][8]