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. The expressed protein is designed to neutralize vascular endothelial growth factor (VEGF) activity, modifying the pathway for formation of new leaky blood vessels and retinal fluid accumulation. Wet AMD is characterized by loss of vision due to excess blood vessel formation between two layers of cells in the retina. This excess blood vessel formation results in fluid leakage that can result in physical changes in the structure of the retina and changes in vision. As this process becomes more severe, blindness can result from scar formation due to hemorrhaging. 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. The expressed protein is designed to neutralize vascular endothelial growth factor (VEGF) activity, modifying the pathway for formation of new leaky blood vessels and retinal fluid accumulation. Wet AMD is characterized by loss of vision due to excess blood vessel formation between two layers of cells in the retina. This excess blood vessel formation results in fluid leakage that can result in physical changes in the structure of the retina and changes in vision. As this process becomes more severe, blindness can result from scar formation due to hemorrhaging. Learn More Topline data late 2018 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. HoFH is a monogenic disorder caused by abnormalities in the function or expression of the LDLR gene. HoFH patients have very low levels or are completely deficient of LDLR, resulting in very high total blood cholesterol levels. This leads to premature and aggressive plaque buildup, life threatening coronary artery disease (CAD) and aortic valve disease. 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. HoFH is a monogenic disorder caused by abnormalities in the function or expression of the LDLR gene. HoFH patients have very low levels or are completely deficient of LDLR, resulting in very high total blood cholesterol levels. This leads to premature and aggressive plaque buildup, life threatening coronary artery disease (CAD) and aortic valve disease. Learn More Topline data late 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). MPS I is a rare recessive genetic disease caused by deficiency of IDUA, an enzyme required for the breakdown of polysaccharides heparan sulfate and dermatan sulfate in the lysosomes of cells. Many patients develop symptoms related to glycosaminoglycan storage in the CNS, which can include excessive accumulation of fluid in the brain (hydrocephalus), spinal cord compression and cognitive impairment. 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). MPS I is a rare recessive genetic disease caused by deficiency of IDUA, an enzyme required for the breakdown of polysaccharides heparan sulfate and dermatan sulfate in the lysosomes of cells. Many patients develop symptoms related to glycosaminoglycan storage in the CNS, which can include excessive accumulation of fluid in the brain (hydrocephalus), spinal cord compression and cognitive impairment. Learn More Begin enrollment in Phase I trial mid-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). MPS II is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme IDS. In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Developmental progression begins to plateau between three and five years of age, with regression reported to begin around six and a half years. 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). MPS II is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme IDS. In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Developmental progression begins to plateau between three and five years of age, with regression reported to begin around six and a half years. Learn More Begin enrollment in Phase I/II trial mid-2018 PHASE I/II
Additional Lead Program Candidate

Undisclosed

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