Regenxbio Inc. is a leading clinical-stage biotechnology company focused on developing and commercializing gene therapies to treat genetic disorders through its proprietary NAV Technology Platform. This platform utilizes recombinant adeno-associated virus (AAV) vectors, including AAV7, AAV8, AAV9, and over 100 novel variants, selected for their safety profile in delivering therapeutic genes or enabling production of proteins that address underlying genetic defects. The company's investigational AAV therapeutics pipeline targets retinal diseases such as wet age-related macular degeneration and diabetic retinopathy with RGX-314; neuromuscular disorders like Duchenne muscular dystrophy with RGX-202; and neurodegenerative conditions including mucopolysaccharidosis types I and II, X-linked retinitis pigmentosa, and neuronal ceroid lipofuscinosis. Regenxbio Inc. also generates revenue through licensing its NAV vectors to partners and royalties from products like Zolgensma, supporting advancements in gene therapy for metabolic, retinal, and rare diseases. Founded in 2009 and headquartered in Rockville, Maryland, the company plays a pivotal role in advancing one-time curative treatments in the biotechnology sector.