Rare disease drugmaker · based in Rockville, United States · founded 2009 · ticker RGNX · regenxbio.com
Regenxbio works on 3 rare diseases we track, including Duchenne Muscular Dystrophy, Hunter Syndrome and 1 more. It has 4 active clinical trials, though none are looking for new volunteers right now.
REGENXBIO is a gene therapy company that builds treatments on its NAV adeno-associated virus (AAV) technology. Its most advanced rare disease program is RGX-202 for Duchenne muscular dystrophy, and REGENXBIO said on November 5, 2026 that it plans to submit the application for accelerated approval in the first quarter of 2027. RGX-121 for Hunter syndrome (MPS II) received a complete response letter from the FDA in February 2026.
Regenxbio pipeline
Each row lists the diseases Regenxbio is studying at that stage, from the ones closest to approval down to the earliest work.
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
These are Regenxbio trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
4 trials underway, not taking new volunteers
Underway, not taking new volunteersPhase 2/3Updated a few months agoNCT05693142
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne.
This is an observationalscreening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety, efficacy, and pharmacodynamic dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.
In August 2026 the FDA placed RGX-121 on clinical hold for the second time that year, after spine MRI findings in 5 participants in its CAMPSIITE study, and BioPharma Dive reported on August 24, 2026 that REGENXBIO no longer expects to resubmit the application in the near term. RGX-111 for MPS I is partnered with Nippon Shinyaku.
With AbbVie, REGENXBIO also develops surabgene lomparvovec (ABBV-RGX-314) for wet age-related macular degeneration and diabetic retinopathy, which are common eye diseases.
Where Regenxbio trials take place
The trials above run at 30 study sites in 3 countries. Tap a country to see its states, cities and hospitals.
United States
25▼
Canada
4▼
Brazil
1▼
Rare diseases Regenxbio works on
Each disease page lists every open trial, not only Regenxbio's, plus approved treatments.
Hunter syndrome is an X-linkedlysosomal storage disorder caused by deficiency of iduronate-2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in cells and tissues.
How common: 1 in 100,000 to 150,000 males; females with disease are rare
Mucopolysaccharidosis Type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which breaks down glycosaminoglycans (GAGs).
How common: Approximately 1 per 100,000 live births; Hurler form is the most common phenotype
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Regenxbio's own site is regenxbio.com, and our help paying for treatment page lists drugmaker programs and charity funds.
1 shared disease, including Duchenne Muscular Dystrophy
Questions about Regenxbio
REGENXBIO is a gene therapy company that builds treatments on its NAV adeno-associated virus (AAV) technology. Its most advanced rare disease program is RGX-202 for Duchenne muscular dystrophy, and REGENXBIO said on November 5, 2026 that it plans to submit the application for accelerated approval in the first quarter of 2027. RGX-121 for Hunter syndrome (MPS II) received a complete response letter from the FDA in February 2026. In August 2026 the FDA placed RGX-121 on clinical hold for the second time that year, after spine MRI findings in 5 participants in its CAMPSIITE study, and BioPharma Dive reported on August 24, 2026 that REGENXBIO no longer expects to resubmit the application in the near term. RGX-111 for MPS I is partnered with Nippon Shinyaku. With AbbVie, REGENXBIO also develops surabgene lomparvovec (ABBV-RGX-314) for wet age-related macular degeneration and diabetic retinopathy, which are common eye diseases.
Regenxbio has active programs across 3 rare diseases: Duchenne Muscular Dystrophy, Hunter Syndrome, Mucopolysaccharidosis Type I. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Regenxbio is a rare disease specialist headquartered in Rockville, United States, founded in 2009. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Duchenne Muscular Dystrophy, Hunter Syndrome, Mucopolysaccharidosis Type I.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Regenxbio, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
None that we track as of November 2026. No Regenxbio label in the FDA's openFDA database matches a rare disease on Trial Friend, which usually means the company has no approved product yet or its approved drugs are for common conditions.
In development: the trials list on this page shows what Regenxbio is recruiting for now, and the FDA calendar lists any application once a decision date is public.
Regenxbio was founded in 2009 and is headquartered in Rockville, United States. The company has been active for 17 years. It trades publicly under the ticker symbol RGNX. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.