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REGENXBIO Announces Presentation at the World Muscle Society
ROCKVILLE, Md., Sept. 29, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced Chief Medical Officer, Steve Pakola, M.D., will present at the
About this update from Regenxbio Inc.
[{"type":"text","content":"\n ROCKVILLE, Md., Sept. 29, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced Chief Medical Officer, Steve Pakola, M.D., will present at the International Congress of the World Muscle Society taking place in Vienna, Austria, October 7-11, 2025.\n The podium presentation will share new analysis of previously presented 12-month functional data from the Phase I/II trial of RGX-202, including individual patient improvement on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model from the Collaborative Trajectory Analysis Project. As reported, RGX-202 demonstrated a favorable safety profile with no serious adverse events or adverse events of special interest observed in the Phase I/II study. Pivotal dose participants exceeded baseline-matched external natural history controls on all functional measures.\n Using multiple models of natural history disease progression, these results further demonstrate the potential of RGX-202 to serve as a differentiated, best-in-class gene therapy for the treatment of Duchenne muscular dystrophy.\n \n Presentation: RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical DataPresenter: Steve Pakola, M.D., Chief Medical Officer, REGENXBIOSession: Short Oral Presentations 1: Updates on SMA and DMD Trials- Accompanying Poster: P425Date/Time: October 8, 3:30 CET\n The presentation will be available on the Publications page of REGENXBIO's website, www.REGENXBIO.com.\n \n About RGX-202 RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain.\n Additional design features such as codon optimization may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured by REGENXBIO using its proprietary, high-yielding NAVXpress® suspension-ba...