THERAPEUTIC PROGRAMS

Advancing the development of potentially
life-changing treatments

diver

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
Retinal Disease

Wet AMD
RGX-314 Subretinal

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 treatment for wet AMD that includes the NAV AAV8 vector containing a gene encoding for a monoclonal anti-VEGF antibody fragment. Learn More

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 treatment for wet AMD that includes the NAV AAV8 vector containing a gene encoding for a monoclonal anti-VEGF antibody fragment. Learn More AAV-mediated antibody delivery for chronic diseases PHASE I/II

Wet AMD
RGX-314 Suprachoroidal

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 treatment for wet AMD that includes the NAV AAV8 vector containing a gene encoding for a monoclonal anti-VEGF antibody fragment. Learn More

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 treatment for wet AMD that includes the NAV AAV8 vector containing a gene encoding for a monoclonal anti-VEGF antibody fragment. Learn More AAV-mediated antibody delivery for chronic diseases PHASE I/II

Diabetic Retinopathy
RGX-314 Suprachoroidal

RGX-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 More

Worldwide

RGX-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 More AAV-mediated antibody delivery for chronic diseases PRECLINICAL

Add’l anti-VEGF treated conditions
Add’l anti-VEGF treated conditions

Worldwide

AAV-mediated antibody delivery for chronic diseases RESEARCH

CLN2 Disease title icons
RGX-381

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, which 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, which is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the retina. Learn More Monogenic gene replacement PRECLINICAL
Neurodegenerative Disease

MPS II title icons
RGX-121

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

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

MPS I title icons
RGX-111

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

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

CLN2 Disease title icons
RGX-181

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

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

Tauopathies α-synucleinopathies
Tauopathies α-synucleinopathies

AAV-mediated antibody delivery for chronic diseases RESEARCH
Liver-directed

Hereditary Angioedema
Hereditary Angioedema

Worldwide

AAV-mediated antibody delivery for chronic diseases PRECLINICAL
Neuromuscular Diseases

Undisclosed
Undisclosed

Worldwide

Monogenic gene replacement RESEARCH

  Orphan Drug Designation
  Rare Pediatric Disease Designation
   Fast Track Designation

AAV-mediated antibody delivery for chronic diseasesMonogenic gene replacement

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