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REGENXBIO Inc. is a clinical-stage biotechnology company, which is focused on providing curative potential of gene therapy. Its investigational gene therapies are designed to deliver functional genes to address genetic defects in cells. The Company’s investigational AAV Therapeutics include RGX-314, RGX-202, RGX-121, RGX-111 and RGX-181 and RGX-381. RGX-314 is meant for the treatment of large patient populations impacted by wet age-related macular degeneration (wet AMD), diabetic retinopathy and other chronic retinal diseases. RGX-202 is developed to treat Duchenne muscular dystrophy, one of the common fatal genetic disorders affecting children. RGX-121, RGX-111 and RGX-181 are developed to treat Mucopolysaccharidosis type II, Mucopolysaccharidosis type I, and late infantile neuronal ceroid lipofuscinosis type II (CLN2 disease), all of which are progressive, neurodegenerative lysosomal storage disorders. RGX-381 is developed to treat the ocular manifestations of CLN2 disease.
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