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
Orphan Drug Designation
Rare Pediatric Disease Designation
Fast Track Designation
INDICATION | RESEARCH | PRECLINICAL | PHASE I/II | PHASE III | COMMERCIAL RIGHTS | PHASE | |
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Retinal Disease
Batten disease (CLN2) 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, and is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the retina.
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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, and is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene to the retina. Learn More Monogenic gene replacement | PRECLINICAL |
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