REGENXBIO

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates 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. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's lead product candidate is RGX-314, which is in Phase III clinical trial for the treatment of wet age-related macular degeneration. It is also developing RGX-121 that is in Phase I/II clinical trial to treat mucopolysaccharidosis type II;RGX-111, which is in Phase I/II clinical trial for treating mucopolysaccharidosis type I;RGX-181 which is in pre clinic stage for the treatment of late-infantile neuronal ceroid lipofuscinosis type II disease;RGX-202, to treat Duchenne muscular dystrophy which is in phase I/II clinical trial; and RGX-381, to treat the ocular manifestations of CLN2 disease which is in preclinical stage. REGENXBIO Inc. also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies; and has a collaboration and license agreement with Neurimmune AG to develop novel gene therapies. REGENXBIO Inc. was founded in 2008 and is headquartered in Rockville, Maryland.

REGENXBIO's mission is to make a difference in the lives of people with rare, inherited diseases through the development of novel and potentially curative gene therapy treatments.

REGENXBIO's vision is to be a leading innovator in gene therapy and to bring innovative treatment to patients with rare and devastating diseases around the world.

REGENXBIO has been recognized by numerous organizations, including the Philadelphia Business Journal awarding it the 2018 Corporate Philanthropy Award and Forbes magazine recognizing it as one of the top employers in 2018.