REGENXBIO Announces Publication of Preclinical Results Demonstrating Functional Benefits of Novel Microdystrophin Construct in RGX-202 Investigational Gene Therapy for Duchenne Muscular Dystrophy

Core Insights - REGENXBIO Inc. announced preclinical results showing that a microdystrophin gene therapy construct with the C-terminal (CT) domain provides improved functional benefits for patients with Duchenne Muscular Dystrophy compared to a construct without the CT domain [1][4][5] - RGX-202 is the only investigational microdystrophin gene therapy candidate that includes the CT domain, making it closest to naturally occurring dystrophin [2][8] Group 1: Research Findings - The preclinical study published in Molecular Therapy Methods and Clinical Development demonstrated that the microdystrophin with the CT domain was maintained at higher levels in transduced muscles and effectively recruited the dystrophin-associated protein complex to the muscle membrane [4][5] - The incorporation of the CT domain enhances the microdystrophin design, allowing for higher accumulation levels in muscle and potentially improving functional benefits [5][7] Group 2: Clinical Trial Insights - Interim results from the Phase I/II AFFINITY DUCHENNE trial indicated that RGX-202 showed consistent evidence of positively changing the disease trajectory in patients with Duchenne and had a favorable safety profile [5][6] - REGENXBIO is currently enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial and plans to submit a Biologics License Application (BLA) via the accelerated approval pathway in mid-2026 [6][9] Group 3: Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline that includes RGX-202 for Duchenne, among other treatments for rare diseases [11] - The company has pioneered AAV gene therapy since its founding in 2009 and has treated thousands of patients with its AAV platform [11]