Therapeutic Pipeline

We are developing gene therapy product candidates in the retinal, metabolic and neurodegenerative therapeutic areas.

Our NAV® Technology Platform 

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

Orphan Drug Designation
Rare Pediatric Disease Designation
Fast Track Designation
Regenerative Medicine Advanced Therapy Designation
AAV-mediated antibody delivery for chronic diseases
Monogenic gene replacement
Retinal Disease
 research preclinical phase I/II phase III Commercial Rights

Wet AMD (subretinal)

ABBV-RGX-314
aabvie logo
U.S. Equal Profit Share Ex-U.S. Tiered Royalties

Wet AMD (suprachoroidal)

ABBV-RGX-314
aabvie logo
U.S. Equal Profit Share Ex-U.S. Tiered Royalties

Diabetic retinopathy (suprachoroidal)

ABBV-RGX-314
aabvie logo
U.S. Equal Profit Share Ex-U.S. Tiered Royalties

Other chronic retinal conditions

aabvie logo
U.S. Equal Profit Share Ex-U.S. Tiered Royalties
Neuromuscular Diseases

Duchenne muscular dystrophy

RGX-202

Worldwide
Neurodegenerative Diseases

Hunter syndrome (MPS II)

RGX-121

Worldwide