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
| PROGRAM AREA | RESEARCH | PRECLINICAL | PHASE I/II | PHASE III | APPROVED | COMMERCIAL RIGHTS | PHASE | |
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Retinal Disease
Wet AMD RGX-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 subretinal treatment for wet AMD that includes the NAV AAV8 vector containing a gene encoding for a monoclonal antibody fragment.
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Worldwide |
RGX-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 subretinal treatment for wet AMD that includes the NAV AAV8 vector containing a gene encoding for a monoclonal antibody fragment. Learn More AAV-mediated antibody delivery for chronic diseases | PHASE I/II | |||||
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Worldwide |
AAV-mediated antibody delivery for chronic diseases | PRECLINICAL | ||||||
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Worldwide |
AAV-mediated antibody delivery for chronic diseases | PRECLINICAL | ||||||
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Neurodegenerative Disease
MPS II RGX-121 is our product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, which is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS).
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Worldwide |
RGX-121 is our product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, which is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS). Learn More Monogenic gene replacement | PHASE I/II | |||||
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MPS I RGX-111 is our product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I), which is designed to use the AAV9 vector to deliver the human α-l-iduronidase (IDUA) gene to the central nervous system (CNS).
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Worldwide |
RGX-111 is our product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I), which is designed to use the AAV9 vector to deliver the human α-l-iduronidase (IDUA) gene to the central nervous system (CNS). Learn More Monogenic gene replacement | PHASE I/II | |||||
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CLN2 Disease RGX-181 is our product candidate for the treatment of late-infantile neuronal ceroid lipofuscinosis Type 2 (or CLN2 disease), a form of Batten disease, which is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the central nervous system (CNS).
Learn More
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Worldwide |
RGX-181 is our product candidate for the treatment of late-infantile neuronal ceroid lipofuscinosis Type 2 (or CLN2 disease), a form of Batten disease, which is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the central nervous system (CNS). Learn More Monogenic gene replacement | PRECLINICAL | |||||
Co-Commercialization |
AAV-mediated antibody delivery for chronic diseases | RESEARCH | ||||||
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Liver-directed
HoFH RGX-501 is our product candidate for the treatment of homozygous familial hypercholesterolemia (HoFH), which is designed to use the AAV8 vector to deliver the human low-density lipoprotein receptor (LDLR) gene to liver cells.
Learn More
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Worldwide |
RGX-501 is our product candidate for the treatment of homozygous familial hypercholesterolemia (HoFH), which is designed to use the AAV8 vector to deliver the human low-density lipoprotein receptor (LDLR) gene to liver cells. Learn More Monogenic gene replacement | PHASE I/II | |||||
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Worldwide |
AAV-mediated antibody delivery for chronic diseases | RESEARCH |
Orphan Drug Designation
Rare Pediatric Disease Designation
Fast Track Designation
©2018 REGENXBIO Inc. “NAV®” is a registered trademark of REGENXBIO Inc. All rights reserved.
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