About Us

Leaders in AAV Gene Therapy


REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.

Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering improved patient outcomes.

We are currently developing gene therapy product candidates for the treatment of retinal, metabolic, and neurodegenerative diseases. Our 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.

Mission & Values

Gene therapy has the potential to address the underlying cause of disease, providing single-dose therapies with long-lasting results. REGENXBIO is focused on developing therapies for diseases that have significant unmet needs. Our management team includes leaders who are experienced in building and operating innovative healthcare ventures and have expert knowledge in the development of AAV gene therapy.

Our core values drive everything we do: