Therapeutic Programs

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		ANTICIPATED MILESTONE	PHASE
Retina RGX-314 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. 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	Phase I/IIa data and initiation of Phase IIb trial in late 2019	PHASE I/II
RGX-314 Diabetic Retinopathy		IND submission in 2H 2019	RESEARCH
Neurodegenerative RGX-121 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). 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	Interim data update in 2H 2019	PHASE I/II
RGX-111 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). 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 in mid-2019	PHASE I/II
RGX-181 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	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	IND submission in 2H 2019	RESEARCH
Metabolic RGX-501 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	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	Interim data update in 2H 2019	PHASE I/II

Orphan Drug Designation
Rare Pediatric Disease Designation
Fast Track Designation

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9600 Blackwell Road, Suite 210, Rockville, MD 20850
240.552.8181

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