REGENXBIO to Present Promising Clinical Findings at World Muscle Society Conference

REGENXBIO's Significant Presentation at World Muscle Society

REGENXBIO Inc., a leading biotechnology company, is making waves in the field of gene therapy with its upcoming presentation at the International Congress of the World Muscle Society. The event, scheduled to take place from October 7 to 11, 2025, in Vienna, Austria, will feature Dr. Steve Pakola, the Chief Medical Officer of REGENXBIO, as a key speaker.

Dr. Pakola's presentation will focus on the latest analyses from the Phase I/II trial of RGX-202, a novel investigational gene therapy aimed at treating Duchenne muscular dystrophy (DMD). This presentation is particularly important as it includes 12-month functional data that previews the potential of RGX-202 in providing effective treatment for patients afflicted by this devastating genetic disorder.

Insights from the Phase I/II Clinical Trial

The Phase I/II trial has shown promising results, especially regarding the safety profile of RGX-202. Notably, there have been no serious adverse events reported, marking a significant milestone in the study. Furthermore, participants have demonstrated improvement beyond the expected outcomes when compared to external natural history controls, as assessed by the North Star Ambulatory Assessment (NSAA). This enhancement in functional performance is analyzed using the Collaborative Trajectory Analysis Project (cTAP) disease progression model.

The RGX-202 therapy is designed to deliver a microdystrophin construct, which encodes essential areas of naturally occurring dystrophin, including the critical C-Terminal (CT) domain. By focusing on gene expression and reducing immunogenicity through codon optimization, the therapy presents a unique advancement in the treatment landscape for DMD.

Pivotal Data Highlights

Dr. Pakola will present interim clinical data under the title "RGX-202: An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy." The session is part of a broader effort to update attendees on trials relating to spinal muscular atrophy (SMA) and DMD, highlighting the growing portfolio of gene therapies targeting these debilitating conditions.

Scheduled for October 8 at 3:30 PM CET, Dr. Pakola's talk will shine a light on the feasibility and effectiveness of RGX-202 through empirical evidence, as well as detail its implications for future therapeutic options in DMD. An accompanying poster with further data will provide additional context for those interested in the technical aspects of the study.

The Path Ahead for RGX-202 and REGENXBIO

As the only gene therapy in advanced stages of development specifically for DMD, RGX-202's innovative approach distinguishes it from competing modalities. Using the NAV® AAV8 vector and a muscle-specific promoter, the therapy aims to enhance the delivery system for microdystrophin throughout not only skeletal muscles but also the heart, making its therapeutic reach potentially transformative.

REGENXBIO continues to pave the way for innovative treatments in genetic disorders, using its proprietary NAVXpress® platform to manufacture RGX-202 at high yields. With thousands of patients already treated through its AAV platform, including Novartis' ZOLGENSMA®, REGENXBIO stands at the forefront of gene therapy revolution.

As the medical community and patients eagerly await the outcomes of the upcoming presentation in Vienna, RGX-202 embodies not just a treatment option, but a beacon of hope for those affected by Duchenne muscular dystrophy. The anticipation surrounding the results and their potential to redefine care for DMD is palpable, marking a pivotal moment in the journey towards more effective gene therapies.

For more information, the presentation will be available on the REGENXBIO website post-event.