Business Description
REGENXBIO is a US-based clinical-stage company focused on developing treatments for serious genetic diseases by delivering healthy copies of defective genes directly into patients' cells. The company's pipeline includes treatments targeting inherited conditions that affect the eyes, muscles, and brain, with several programs advancing through clinical development in partnership with major pharmaceutical companies including AbbVie and Nippon Shinyaku. Beyond its own pipeline, REGENXBIO generates revenue by licensing its proprietary gene delivery technology to other drug developers. Founded in 2008 and based in Rockville, Maryland, the company is working to establish gene therapy as a viable long-term solution for patients with few or no existing treatment options.
