RGX-202 / REGENXBIO - LARVOL DELTA

RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy: Topline Results from a Phase III Trial (ESGCT 2026) - No abstract available

Gene therapy • P3 data • P3 data: top line • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P2/3 | N=65 | Active, not recruiting | Sponsor: REGENXBIO Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P2/3 | N=65 | Recruiting | Sponsor: REGENXBIO Inc. | Trial primary completion date: Feb 2026 ➔ Aug 2026

Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Expanded Access to RGX-202 (clinicaltrials.gov) - P=N/A | N=0 | Temporarily Not Available | Sponsor: REGENXBIO Inc.

New trial

Single-nuclei transcriptomic analysis of skeletal muscle mRNA in mdx mice treated with RGX-202, an AAV vector encoding micro-dystrophin. (ASGCT 2026) - "In immune cells, RGX-202 treatment led to the upregulation of anti-inflammatory factors. Conclusion Ultimately, these studies may provide a better understanding of how RGX-202 impacts the intra-muscular microenvironment in dystrophin-deficient mdx mice at the molecular level."

Omic analysis • Preclinical • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy

RGX-202: Investigational gene therapy for Duchenne Muscular Dystrophy (ASGCT 2026) - "Conclusion At both dose levels, investigational RGX-202 has been well tolerated. In addition, robust RGX-202 microdystrophin expression was observed up to 12 months post-administration of both doses and treatment effects were observed up to 18 months post-administration of the pivotal dose in boys 1-12 years old with Duchenne in the phase I/II portion of the study."

Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hepatology • Immune Modulation • Immunology • Liver Failure • Muscular Dystrophy

AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD. (PubMed, Mol Ther Nucleic Acids) - P2/3 | "These findings provide preclinical evidence for the therapeutic efficacy of RGX-202 at a minimum effective dose (MED) of 1 × 1014 gc/kg in the murine DMD model. This MED served as the starting dose for the RGX-202 clinical study (NCT05693142), which has currently completed phase III enrollment."

Journal • Preclinical • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Phase I/II Clinical Data (AAN 2026) - "At both dose levels, RGX-202 has been well tolerated; Robust RGX-202 microdystrophin expression and functional improvements were observed up to 12 months post-administration in boys 1-12 years old with DMD."

Clinical data • Gene therapy • P1/2 data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hepatology • Immune Modulation • Immunology • Liver Failure • Muscular Dystrophy

Long Term Follow-up for RGX-202 (clinicaltrials.gov) - P=N/A | N=66 | Enrolling by invitation | Sponsor: REGENXBIO Inc. | N=19 ➔ 66

Enrollment change • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Gene therapy in Duchenne muscular dystrophy. (PubMed, Arch Pediatr) - "In 2023, delandistrogene moxeparvovec (Sarepta/Roche) received accelerated approval from the U.S. Food and Drug Administration for ambulatory pediatric patients, marking the first regulatory authorization of a gene therapy for DMD. By contrast, fordadistrogene movaparvovec (Pfizer) showed encouraging biomarker results but was associated with immune-mediated serious adverse events, including thrombotic microangiopathy cases and patient deaths due to acute liver failure, ultimately leading to program discontinuation. Other investigational candidates-GNT0004 (Généthon), SGT-003 (Solid Biosciences), and RGX-202 (Regenxbio)-incorporate distinct promoter designs and microdystrophin cassettes and are currently in early- to mid-phase evaluation...Gene therapy management also raises difficult economic and logistical challenges for healthcare systems. Balancing rapid patient access to potentially disease-modifying therapies with rigorous scientific and regulatory..."

Journal • Review • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hepatology • Liver Failure • Muscular Dystrophy • Myositis • Pediatrics

REGENXBIO…Initiates Commercial Production in Duchenne Gene Therapy Program (PRNewswire) - "REGENXBIO has manufactured the first batches of RGX-202 intended for commercial supply, supporting the company's expected approval and commercial launch in 2027, when the vast majority of the prevalent market is expected to be available. The company has also manufactured full supply of RGX-202 for the confirmatory trial."

Approval • Commercial • Launch • Duchenne Muscular Dystrophy

REGENXBIO Announces Completion of Pivotal Enrollment…in Duchenne Gene Therapy Program (PRNewswire) - "The pivotal portion of the multi-center, open-label Phase I/II/III AFFINITY DUCHENNE trial completed enrollment of 30 participants in October 2025....'We are committed to...sharing topline pivotal data in early Q2 of next year'."

Clinical data • Enrollment closed • Duchenne Muscular Dystrophy

Functional improvements seen in patients after muscular dystrophy gene therapy (BioNews) - P1/2/3 | N=65 | AFFINITY DUCHENNE (NCT05693142) | Sponsor: REGENXBIO Inc. | "As part of the Phase I/II AFFINITY DUCHENNE trial, five boys aged six to 12 years were treated with RGX-202, a single-dose gene therapy that aims to provide patients with a functional version of the microdystrophin gene. The five patients demonstrated improved physical performance, as well as increased microdystrophin expression. Additionally, no significant adverse side effects were observed, indicating that RGX-202 may drastically improve outcomes for patients with DMD."

P1/2 data • Duchenne Muscular Dystrophy

RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data (ASGCT 2025) - "In the Phase I/II patients, RGX-202 has been well tolerated and demonstrated both robust RGX-202 microdystrophin expression and functional improvements. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Clinical data • Gene therapy • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Development of a Commercial Manufacturing Process for RGX-202, a Systemically Delivered AAV for the Treatment of Duchenne Muscular Dystrophy (ASGCT 2025) - "The RGX-202 commercial manufacturing process has been successfully executed at the REGENXBIO Manufacturing Innovation Center. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data (AAN 2025) - "These participants also exceeded available external natural history controls matched for age and baseline function. Conclusions In the Phase I/II patients, RGX-202 has been well tolerated and demonstrated both robust RGX-202 microdystrophin expression and functional improvements."

Clinical data • Gene therapy • Late-breaking abstract • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

REGENXBIO REPORTS POSITIVE BIOMARKER DATA FROM AFFINITY DUCHENNE TRIAL OF RGX-202 GENE THERAPY (PRNewswire) - P1/2/3 | N=65 | AFFINITY DUCHENNE (NCT05693142) | Sponsor: REGENXBIO Inc. | "Positive biomarker data in patient aged 1-3 add to consistent, robust microdystrophin and transduction levels across all treated ages. Patient aged 3 years at dosing had expression level at 122.3% compared to control....With a differentiated novel construct and proactive short course immune modulation regimen, RGX-202 continues to demonstrate encouraging safety profile with no SAEs or AESIs....REGENXBIO plans to share additional interim functional data in the first half of 2025...Phase III portion of AFFINITY DUCHENNE trial enrolling ambulatory patients aged 1 and above, on track for BLA submission mid-2026."

FDA filing • P1/2 data • P3 data • Duchenne Muscular Dystrophy

REGENXBIO Announces Presentations at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (PRNewswire) - "REGENXBIO Inc...today announced new interim biomarker data from the Phase I/II portion of the AFFINITY DUCHENNE trial of RGX-202 for the treatment of Duchenne muscular dystrophy will be presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Dallas, TX, March 16-19, 2025."

Biomarker • P1/2 data • Duchenne Muscular Dystrophy

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants with Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P2/3 | N=65 | Recruiting | Sponsor: REGENXBIO Inc. | Phase classification: P1/2 ➔ P2/3 | N=15 ➔ 65 | Trial completion date: Dec 2025 ➔ Aug 2028

Enrollment change • Phase classification • Trial completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Long Term Follow-up for RGX-202 (clinicaltrials.gov) - P=N/A | N=19 | Enrolling by invitation | Sponsor: REGENXBIO Inc.

Gene therapy • New trial • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Nonclinical and clinical updates on RGX-202, an AAV8 vector encoding a novel microdystrophin for the treatment of Duchenne Muscular Dystrophy (New Directions 2024) - No abstract available

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data (ASGCT 2024) - "RGX-202 has been well tolerated in 3 participants up to 25 weeks post-administration of RGX-202, with readily detectable RGX-202 microdystrophin levels at 12 weeks."

Clinical data • Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data (AAN 2024) - "RGX-202 has been well tolerated in 3 participants up to 25 weeks post-administration of RGX-202."

Clinical data • Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Quantitation of Dystrophin and RGX-202 Microdystrophin by a Validated Hybrid LBA/LC-MS Assay (MDA 2024) - "This assay, as well as an orthogonal method that uses a Capillary Western Immunoassay (JESS) are currently used to support the first-in-human clinical study Affinity Duchenne. RGX-202 microdystrophin Data from the Affinity Duchenne study will be presented."

Late-breaking abstract • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: REGENXBIO Inc.

Gene therapy • New P1/2 trial • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy