Abstract
17O RGX-202, a phase I/II/III investigational gene therapy for the treatment of Duchenne muscular dystrophy
Neuromuscular disorders : NMD, Vol.67(Suppl), 107359
10/2026
DOI: 10.1016/j.nmd.2026.107359
Abstract
Duchenne is a progressive, lethal muscle disease in which mutations in the DMD gene that encodes dystrophin leads to muscle weakness and degeneration. RGX-202 is an investigational, one-time AAV gene therapy designed to deliver an optimized microdystrophin gene. RGX-202 is the only microdystrophin construct that encodes the C-Terminal (CT) domain found in naturally occurring dystrophin. In preclinical studies, inclusion of the CT domain preserved skeletal and cardiac muscle health. AFFINITY DUCHENNE® is a multicenter, open-label phase I/II/III trial evaluating the safety, tolerability, and efficacy of a single intravenous dose of RGX-202 for ambulatory boys with Duchenne. The phase I/II study enrolled boys aged 1-11 years to one of two dose levels (DL): DL1, 1 × 1014 genome copies (GC)/kg body weight (n=3), or DL2, 2 × 1014GC/kg (n=10). The phase III trial enrolled boys ages 1 and older at DL2. Interim data from the Phase I/II trial were previously reported (March 11, 2026). RGX-202 was well tolerated in the Phase I/II study. Common drug-related AEs included vomiting (n=7), fatigue (n=6), and nausea (n=4). There were no serious adverse events (SAEs) or adverse events of special interest (AESIs) reported. Functional outcomes were analyzed using multiple validated methods to estimate expected disease progression without treatment, including the cTAP disease progression model and external control comparisons using coarsened exact matching and propensity score weighting. Participants in DL2 (n=7; boys aged 8+, n=5) demonstrated improvement in function at 1 year and exceeded external controls (propensity score weighting). MRI-assessed left ventricular ejection fraction and other cardiac measures remained stable through 1 year (n=7). Enrollment in the phase III trial completed in October 2025 with topline data anticipated in Q2 of 2026. Confirmatory trial enrollment continues. Study results from the Phase I/II/III study will be presented.
Details
- Title: Subtitle
- 17O RGX-202, a phase I/II/III investigational gene therapy for the treatment of Duchenne muscular dystrophy
- Creators
- Steve Pakola - Regenxbio, Rockville, USAAravindhan Veerapandiyan - Arkansas Children’s Hosp, Little Rock, USACrystal Proud - The King's CollegeNancy Kuntz - Ann and Robert H. Lurie Children’s Hosp of Chicago, Chicago, USAAmy Harper - Virginia Commonwealth UniversitySusan Iannaccone - The Univ of Texas Southwestern Med Center, Dallas, USACarolina Tesi-Rocha - Stanford Sch Med, Stanford, USAErika Finanger - Oregon Health & Science UniversityCuixia Tian - Cincinnati Children's Hospital Medical CenterHan Phan - Rare Disease Research LLC, Atlanta, USAEleonora D’ambrosio - Univ Massachusetts Chan Med Sch, Worcester, USADimah Saade - University of IowaJahannaz Dastgir - Regenxbio, Rockville, USAKoji Takahashi - Regenxbio, Rockville, USAOlivier Danos - Regenxbio, Rockville, USARegenxbio RGX-202 Study Team
- Resource Type
- Abstract
- Publication Details
- Neuromuscular disorders : NMD, Vol.67(Suppl), 107359
- DOI
- 10.1016/j.nmd.2026.107359
- ISSN
- 0960-8966
- Publisher
- Elsevier B.V
- Language
- English
- Date published
- 10/2026
- Academic Unit
- Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9985240281502771
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