REGENXBIO Inc

REGENXBIO Inc. (http://regenxbio.com/) is a clinical-stage biotechnology company. The Company’s gene therapy product candidates are designed to deliver functional genes to address genetic defects in cells, enabling the production of therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates use adeno-associated virus (AAV) vectors from its gene delivery platform, called NAV Technology Platform. Its product candidate includes RGX-314, RGX-111, RGX-121, RGX-202, RGX 181 and RGX-381. RGX-314 is meant for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR) and other anti-VEGF treated conditions. 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). RGX-202 is its product candidate for the treatment of duchenne muscular dystrophy (DMD).

• REGENXBIO Inc