REGENXBIO, Inc. is a clinical-stage biotechnology company, which engages in the development, commercialization, and licensing of recombinant adeno-associated virus gene therapy. The company is headquartered in Rockville, Maryland and currently employs 353 full-time employees. The company went IPO on 2015-09-17. The firm has developed a pipeline of gene therapy programs using its proprietary adeno-associated virus (AAV) gene therapy delivery platform (NAV Technology Platform) to address genetic diseases. The company is focused on its internal development pipeline in three areas: retinal, neuromuscular, and neurodegenerative diseases. Its investigational AAV therapeutics include ABBV-RGX-314, RGX-202, RGX-121, and RGX-111. The company is developing ABBV-RGX-314 in collaboration with AbbVie to treat large patient populations impacted by wet age-related macular degeneration, diabetic retinopathy (DR) and other chronic retinal diseases characterized by loss of vision. The company is developing RGX-202 to treat Duchenne muscular dystrophy (Duchenne). The firm is developing RGX-121 to treat Mucopolysaccharidosis type II (MPS II), a progressive, neurodegenerative lysosomal storage disorder.
