REGENXBIO Inc.

REGENXBIO Inc. is a clinical-stage biotechnology company dedicated to improving lives through the curative potential of gene therapy. The company's core business involves the development, commercialization, and licensing of recombinant adeno-associated virus (AAV) gene therapies, leveraging its proprietary NAV® Technology Platform. Headquartered in Rockville, Maryland, REGENXBIO focuses on delivering one-time treatments for patients suffering from common and rare diseases.

REGENXBIO's pipeline includes gene therapy candidates for retinal, metabolic, neuromuscular, and neurodegenerative diseases. Key product candidates in development include ABBV-RGX-314 for wet age-related macular degeneration (AMD) and diabetic retinopathy, RGX-202 for Duchenne muscular dystrophy, and RGX-121 for mucopolysaccharidosis II (Hunter syndrome). The company also licenses its NAV® vectors to other biotechnology firms, enabling broader development of new medicines.

In early 2026, REGENXBIO reported positive interim data from its Phase I/II AFFINITY DUCHENNE® trial of RGX-202. However, the company faced regulatory challenges in January 2026 when the U.S. Food and Drug Administration (FDA) placed clinical holds on its RGX-111 and RGX-121 programs following the detection of a brain tumor in a participant treated with RGX-111, leading to significant market reaction. Curran Simpson serves as the President and Chief Executive Officer, guiding the company's late-stage pipeline and strategic initiatives amidst these developments.