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.