THERAPEUTIC PROGRAMS

Advancing the development of potentially
life-changing treatments

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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 ANTICIPATED MILESTONE PHASE
Retina

RGX-314

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
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 Complete dosing of Cohort 4 Q4 2018 and initiate Phase II trial in 2019 PHASE I/II
Metabolic

RGX-501

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
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 Additional data Q4 2018 PHASE I/II
Neurodegenerative Diseases

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
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 Begin enrollment in Phase I trial Q4 2018 PHASE I/II

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
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 Begin enrollment in Phase I/II trial Q4 2018 PHASE I/II
Additional Lead Program Candidate

Undisclosed

Designation in 2H 2018
Designation in 2H 2018 Designation in 2H 2018 RESEARCH

  Orphan Drug Designation
  Rare Pediatric Disease Designation
   Fast Track Designation

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