Description
Regenxbio is a clinical-stage biotechnology company focused on providing curative potential of gene therapy.
The Company's investigational gene therapies are designed to deliver functional genes to address genetic defects in cells.
Its investigational AAV Therapeutics include: RGX-314, which is developing in collaboration with AbbVie to treat large patient populations impacted by wet age-related macular degeneration, diabetic retinopathy and other chronic retinal diseases characterized by loss of vision; RGX-202, which is developing to treat Duchenne muscular dystrophy (Duchenne), one of the most common fatal genetic disorders affecting children; RGX-121, RGX-111 and RGX-181, which is developing to treat Mucopolysaccharidosis type II (MPS II), Mucopolysaccharidosis type I (MPS I), and late infantile neuronal ceroid lipofuscinosis type II (CLN2 disease)., and RGX-381, which is developing to treat the ocular manifestations of CLN2 disease.
