Regenxbio
Regenxbio works on 6 rare diseases tracked on Trial Friend, including Batten Disease, Duchenne Muscular Dystrophy, Geographic Atrophy and 3 more, with 4 active clinical trials.
Regenxbio is a clinical-stage biotechnology company advancing gene therapies for rare and retinal diseases using its proprietary AAV gene therapy delivery platform. The company's pipeline includes therapies for mucopolysaccharidosis disorders, Duchenne muscular dystrophy, and inherited retinal diseases.
Focus areas at Regenxbio
As a rare disease specialist, Regenxbio has active clinical trial programs and drug development efforts across 6 rare diseases, including Batten Disease, Duchenne Muscular Dystrophy, Geographic Atrophy, Homozygous Familial Hypercholesterolemia, Hunter Syndrome, and 1 additional rare conditions. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows active and recruiting rare disease studies sponsored by Regenxbio, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Regenxbio is headquartered in Rockville, United States, founded in 2009, publicly traded under the ticker symbol RGNX. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Regenxbio Drug Pipeline
Regenxbio has 4 active clinical trials across 2 development stages. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Regenxbio Clinical Trials (4)
Active and recruiting clinical trials sponsored by Regenxbio, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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FDA-Approved Drugs
Regenxbio Trial Locations
Regenxbio clinical trials are running at 30 sites in 3 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (6)
Diseases targeted by Regenxbio's clinical trial and drug development programs
Batten disease is a rare inherited neurological disorder caused by mutations in genes affecting lysosomal function, leading to accumulation of lipofuscin (age pigment) in neurons. Progressive vision l...
Duchenne muscular dystrophy is an X-linked genetic disorder causing progressive muscle weakness and degeneration, beginning in early childhood. The defective dystrophin protein normally protects muscl...
Geographic atrophy is the advanced form of dry age-related macular degeneration, characterized by progressive loss of retinal pigment epithelium, photoreceptors, and choriocapillaris in well-defined a...
Homozygous Familial Hypercholesterolemia is a severe genetic disorder causing extremely high cholesterol levels from birth, leading to early heart disease and cholesterol deposits in tendons and aroun...
Hunter syndrome is an X-linked lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in cells and tissues. This multisystem dis...
Mucopolysaccharidosis Type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which breaks down glycosaminoglycans (GAGs). This results in accumulation o...
Patient Resources
Organizations and resources related to Regenxbio's rare disease focus areas
Frequently Asked Questions About Regenxbio
Common questions about Regenxbio's rare disease programs, clinical trials, and treatments.