REGENXBIO Inc (RGNX)

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REGENXBIO Inc. is a clinical-stage biotechnology company. The Company's 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 or antibodies that are intended to impact disease. The Company develops products candidates across the therapeutic areas of metabolic, neurodegenerative and retinal diseases. Its product candidate, RGX-314, is meant for the treatment of wet age-related macular degeneration (wet AMD). Its product candidate, RGX-501, is meant for the treatment of homozygous familial hypercholesterolemia (HoFH). It is also developing product candidates, such as RGX-111 and RGX-121, to address the neurological symptoms of two severe genetic lysosomal storage diseases, Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II), respectively. Its product candidates utilize viral vectors from its gene delivery platform, NAV Technology Platform.