Regenxbio Inc is a biotechnology company. Its main activity is the development and commercialization of recombinant adeno-associated virus gene therapy to correct an underlying genetic defect. The diseases that Regenxbio platform targets are metabolic (homozygous familial hypercholesterolemia), neurodegenerative conditions (mucopolysaccharidosis), and retinal diseases (wet age-related macular degeneration, X-linked retinitis pigmentosa).