RGX-202 / REGENXBIO - LARVOL DELTA

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

RGX-202: An investigational AAV8 gene therapy coding for a novel microdystrophin as a treatment for Duchenne muscular dystrophy (WMS 2022) - "Additional inclusion criteria are based on dystrophin gene mutations in exons 18-58, a functional assessment, and AAV seronegative status. Endpoints include safety, immunogenicity assessments, RGX-202 microdystrophin levels in muscle, functional assessments (North Star Ambulatory Assessment and timed function tests), and muscle preservation (MRI)."

Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Muscular Dystrophy • Pediatrics

[VIRTUAL] A Novel AAV8Based Gene Therapy for Duchenne Muscular Dystrophy: Preclinical Studies in the Mdx Mouse (ESGCT 2021) - "Our data demonstrates that treatment with RGX202 resulted in the expression of an active microdystrophin, which improves muscle function and reduces dystrophic pathology in mdx mice. We are currently developing RGX202 as a candidate for microdystrophin gene therapy for DMD."

Preclinical • Cardiovascular • CNS Disorders • Congestive Heart Failure • Developmental Disorders • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Genetic Disorders • Heart Failure • Immunology • Inflammation • Muscular Dystrophy • Psychiatry • MRI

RGX:202, a novel investigational microdystrophin gene therapy for Duchenne Muscular Dystrophy (New Directions 2021) - No abstract available

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

[VIRTUAL] A Novel AAV8 Vector for Microdystrophin Gene Therapy of Duchenne Muscular Dystrophy: Preclinical Studies in the Mdx Mouse (ASGCT 2021) - "Creatine kinase levels as a marker of muscle damage were also dose-dependently decreased at weeks 7 and 12. Taken together, these results suggest the potential of RGX-202 as a promising candidate for clinical development of microdystrophin gene therapy in DMD patients."

Preclinical • Cardiovascular • Congestive Heart Failure • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Heart Failure • Immunology • Inflammation • Muscular Dystrophy • MRI

[VIRTUAL] RGX-202, an AAV8 Vector Encoding an Extended Microdystrophin Efficiently Restores Dystrophin Associated Protein Complex and Corrects Satellite Cells in a Dystrophic Mouse Model (ASGCT 2021) - "In mdx mouse, treatment with RGX-202 was shown to restore the DAPC at the sarcolemma and prevent the pathological cycle of degeneration / regeneration that ultimately results in muscle wasting. This initial characterization provides a rationale for the further development of RGX-202 as a microdystrophin gene therapy treatment for DMD."

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

REGENXBIO Announces Update on RGX-314 and Pivotal Program for the Treatment of Wet AMD and New Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy (PRNewswire) - "...REGENXBIO announced a new program, RGX-202, a novel, potentially best-in-class, one-time gene therapy for the treatment of Duchenne Muscular Dystrophy (DMD)....Proof of concept data from preclinical studies of RGX-202 in the mdx mouse model of DMD demonstrates broad and robust expression of microdystrophin in muscle, recruitment of key proteins to the muscle cells, improvements in muscle histology, as well as meaningful increases in muscle strength and function....Investigational New Drug (IND) application enabling studies are being completed and REGENXBIO expects to submit an IND to the FDA in mid-2021."

IND • Preclinical • Duchenne Muscular Dystrophy