Therapeutic Pipeline

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

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
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U.S. Equal Profit Share Ex-U.S. Tiered Royalties

Wet AMD (suprachoroidal)

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

Diabetic retinopathy (suprachoroidal)

ABBV-RGX-314
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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