THERAPEUTIC PROGRAMS

Advancing the development of potentially
life-changing treatments

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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

AAV-mediated antibody delivery for chronic diseasesMonogenic gene replacement

INDICATION RESEARCH PRECLINICAL PHASE I/II PHASE III COMMERCIAL RIGHTS PHASE
Retinal Disease

Batten disease (CLN2) title icons
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, and 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, 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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