REGENXBIO Inc. leverages its proprietary NAV Technology Platform to discover and develop one-time gene therapies for rare and chronic diseases. The company's internal clinical pipeline includes candidates such as ABBV-RGX-314, developed in collaboration with AbbVie for the treatment of wet age-related macular degeneration and diabetic retinopathy. Other key programs include RGX-202 for Duchenne muscular dystrophy, as well as RGX-121 and RGX-111 for mucopolysaccharidosis (MPS) types II and I, respectively. In addition to its internal development efforts, REGENXBIO licenses its AAV vector technology to other biopharmaceutical companies, generating revenue through upfront fees, milestones, and royalties.
