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

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					U.S. Equal Profit Share Ex-U.S. Tiered Royalties
Wet AMD (suprachoroidal) ABBV-RGX-314						U.S. Equal Profit Share Ex-U.S. Tiered Royalties
Diabetic retinopathy (suprachoroidal) ABBV-RGX-314							U.S. Equal Profit Share Ex-U.S. Tiered Royalties
Other chronic retinal conditions								U.S. Equal Profit Share Ex-U.S. Tiered Royalties
Batten disease (CLN2) RGX-381					Worldwide
Neuromuscular Diseases
Duchenne muscular dystrophy RGX-202					Worldwide
Neurodegenerative Diseases
Hunter syndrome (MPS II) RGX-121					Worldwide
Hurler syndrome (severe MPS I) RGX-111					Worldwide
Batten disease (CLN2) RGX-181					Worldwide