REGENXBIO Announces Presentation at the World Muscle Society

Core Insights - REGENXBIO Inc. announced that Chief Medical Officer, Steve Pakola, M.D., will present at the International Congress of the World Muscle Society in Vienna, Austria, from October 7-11, 2025 [1] - The presentation will include new analysis of 12-month functional data from the Phase I/II trial of RGX-202, highlighting individual patient improvements on the North Star Ambulatory Assessment (NSAA) [2][4] - RGX-202 has shown a favorable safety profile with no serious adverse events reported in the Phase I/II study, and pivotal dose participants outperformed baseline-matched external natural history controls on all functional measures [2][3] Product Overview - RGX-202 is positioned as a potential best-in-class investigational gene therapy aimed at improving function and outcomes in Duchenne muscular dystrophy [5] - It features a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain [5] - The therapy utilizes the NAV AAV8 vector and a muscle-specific promoter (Spc5-12) to support targeted expression of microdystrophin throughout skeletal and heart muscle [6] Company Background - REGENXBIO is a biotechnology company focused on advancing gene therapy to improve lives, having pioneered AAV gene therapy since its founding in 2009 [7] - The company is developing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne muscular dystrophy and other therapies in collaboration with partners [7]