THERAPEUTIC PROGRAMS
Advancing the development of potentially
life-changing treatments
We are developing gene therapy product candidates in the retinal, metabolic and neurodegenerative therapeutic areas.
Our gene therapy product candidates all utilize AAV viral vectors from our proprietary gene delivery platform, which we call our NAV Technology Platform. In addition to our internal product candidate programs, we also selectively license our NAV Vectors to other leading biotechnology companies.
Development Stage Pipeline
| Indication | Development Stage | COMMERCIAL RIGHTS | ||||
|---|---|---|---|---|---|---|
| RESEARCH | PRECLINICAL | PHASE I/II | PHASE III | |||
| Retinal Disease | ||||||
| Wet AMD (subretinal)RGX-314Commercial Rights:U.S. Equal Profit Share Ex-U.S. Tiered RoyaltiesRGX-314 is our product candidate for the treatment of wet age-related macular degeneration (AMD). RGX-314 is being developed as a novel, one-time treatment for wet AMD that includes the NAV AAV8 vector containing a gene encoding for a monoclonal anti-VEGF antibody fragment. Learn MoreAAV-mediated antibody delivery for chronic diseases | PHASE III |  U.S. Equal Profit Share Ex-U.S. Tiered Royalties | ||||
| Wet AMD (suprachoroidal)RGX-314Commercial Rights:U.S. Equal Profit Share Ex-U.S. Tiered RoyaltiesRGX-314 is our product candidate for the treatment of wet age-related macular degeneration (AMD). RGX-314 is being developed as a novel, one-time treatment for wet AMD that includes the NAV AAV8 vector containing a gene encoding for a monoclonal anti-VEGF antibody fragment. Learn MoreAAV-mediated antibody delivery for chronic diseases | PHASE I/II | U.S. Equal Profit Share Ex-U.S. Tiered Royalties | ||||
| Diabetic retinopathy (suprachoroidal)RGX-314Commercial Rights:U.S. Equal Profit Share Ex-U.S. Tiered RoyaltiesRGX-314 is our product candidate for the treatment of diabetic retinopathy. RGX-314 is being developed as a novel, one-time treatment that includes the NAV AAV8 vector containing a gene encoding for a monoclonal anti-VEGF antibody fragment. Learn MoreAAV-mediated antibody delivery for chronic diseases | PHASE I/II | U.S. Equal Profit Share Ex-U.S. Tiered Royalties | ||||
| Other chronic retinal conditionsOther chronic retinal conditionsCommercial Rights:U.S. Equal Profit Share Ex-U.S. Tiered RoyaltiesAAV-mediated antibody delivery for chronic diseases | RESEARCH | U.S. Equal Profit Share Ex-U.S. Tiered Royalties | ||||
Batten disease (CLN2) RGX-381Commercial Rights:WorldwideRGX-381 is our product candidate for the treatment of ocular manifestations in late-infantile neuronal ceroid lipofuscinosis Type 2 (or CLN2 disease), a form of Batten disease, and is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the retina.
Learn MoreMonogenic gene replacement | PRECLINICAL | Worldwide | ||||
| Neuromuscular Diseases | ||||||
| Duchenne muscular dystrophyRGX-202Commercial Rights:WorldwideRGX-202 is our product candidate for the treatment of Duchenne muscular dystrophy and is designed to use the NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin.
Learn MoreMonogenic gene replacement | PHASE I/II | Worldwide | ||||
| Neurodegenerative Disease | ||||||
Hunter syndrome (MPS II) RGX-121Commercial Rights:WorldwideRGX-121 is our product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, and is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS).
Learn MoreMonogenic gene replacement | PHASE III | Worldwide | ||||
| Hurler syndrome (severe MPS I)RGX-111Commercial Rights:WorldwideRGX-111 is our product candidate for the treatment of severe Mucopolysaccharidosis Type I (MPS I), and is designed to use the AAV9 vector to deliver the human α-l-iduronidase (IDUA) gene to the central nervous system (CNS).
Learn MoreMonogenic gene replacement | PHASE I/II | Worldwide | ||||
| Batten disease (CLN2)RGX-181Commercial Rights:WorldwideRGX-181 is our product candidate for the treatment of late-infantile neuronal ceroid lipofuscinosis Type 2 (or CLN2 disease), a form of Batten disease, and is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the central nervous system (CNS).
Learn MoreMonogenic gene replacement | PRECLINICAL | Worldwide | ||||
Orphan Drug Designation
Rare Pediatric Disease Designation
Fast Track Designation
| INDICATION | RESEARCH | PRECLINICAL | PHASE I/II | PHASE III | COMMERCIAL RIGHTS | PHASE | |
|---|---|---|---|---|---|---|---|
|
Retinal Disease
Batten disease (CLN2) RGX-381 is our product candidate for the treatment of ocular manifestations in late-infantile neuronal ceroid lipofuscinosis Type 2 (or CLN2 disease), a form of Batten disease, and is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the retina.
Learn More
|
Worldwide |
RGX-381 is our product candidate for the treatment of ocular manifestations in late-infantile neuronal ceroid lipofuscinosis Type 2 (or CLN2 disease), a form of Batten disease, and is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the retina. Learn More Monogenic gene replacement | PRECLINICAL |
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