REGENXBIO Inc (REGENXBIO) is a clinical-stage biotechnology company focused on developing gene therapies to address genetic defects in cells. The company's investigational therapies aim to deliver functional genes to produce therapeutic proteins or antibodies, potentially altering the course of diseases with long-lasting effects. REGENXBIO utilizes adeno-associated virus (AAV) vectors from its proprietary NAV Technology Platform, which includes a large portfolio of AAV vectors. The company serves industries related to retinal, neuromuscular, and neurodegenerative diseases.

Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam, quis nostrud exercitation ullamco laboris nisi ut aliquip ex ea commodo consequat.

Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam, quis nostrud exercitation ullamco laboris nisi ut aliquip ex ea commodo consequat.

Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam, quis nostrud exercitation ullamco laboris nisi ut aliquip ex ea commodo consequat.

Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam, quis nostrud exercitation ullamco laboris nisi ut aliquip ex ea commodo consequat.

REGENXBIO is focused on expanding the understanding and applications of AAV vectors through its NAV technology platform. The company is dedicated to discovering and developing a pipeline of investigational AAV therapeutics to alter the course of diseases and improve patient outcomes. The company's R&D efforts include engineering novel capsids, designing new NAV vectors, and optimizing enhancer and promoter combinations to enhance gene expression and therapeutic effects. REGENXBIO is also exploring the potential of NAV vectors for in vivo gene editing and delivery of small RNAs. The company's internal research and collaborations aim to identify and optimize AAV therapeutics for various disease areas, including eye diseases, neurodegenerative diseases, and neuromuscular diseases. The company operates the REGENXBIO manufacturing innovation center (RMIC) in Rockville, Maryland, the US. The company has several investigational AAV therapeutics in its pipeline, including ABBV-RGX-314 for wet AMD and DR, RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-111 for MPS I. In FY2025, the company spent US$228.3 million on its R&D activities, which grew 9.5% YoY. As of December 31, 2025, the company's NAV technology platform had 25 patent portfolios issued in the US and five European patents relating to the AAV7, AAV8, AAV9, AAVrh10, and AAVrh46 vectors and their uses. Its investigational AAV therapeutics had six patent issues in the US, one European patent issued, one pending international patent applications filed under the Patent Cooperation Treaty (PCTs), and 23 PCTs that have entered the national stage relating to its product candidates.