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	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 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 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 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 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	Co-Commercialization	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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9600 Blackwell Road, Suite 210, Rockville, MD 20850
240.552.8181

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