REGENXBIO is a clinical-stage biotechnology company specializing in gene therapy. It uses its proprietary NAV® Technology Platform to create therapies that deliver genes to cells, aiming to address genetic defects or enable cells to produce therapeutic proteins. Their product candidates target diseases such as retinal, metabolic, and neurodegenerative conditions. REGENXBIO’s gene therapies are designed for single administration with the goal of providing long-lasting effects. The company also licenses its NAV® Vectors to other biotechnology companies for further applications in gene therapy.