REGENXBIO is a clinical-stage biotechnology company developing gene therapies using its proprietary AAV (adeno-associated virus) NAV technology platform. The company is advancing a pipeline of one-time gene therapies for rare and serious diseases including Duchenne muscular dystrophy (RGX-202), Hunter syndrome (RGX-121), and wet age-related macular degeneration (RGX-314, partnered with AbbVie).
Company Info
Upcoming Milestones & Key Dates
FDA Type A meeting to discuss CRL and path forward for RGX-121 in Hunter syndrome
Q1 2026 Earnings — Pipeline update and RGX-202 pivotal trial enrollment
Clinical Development Pipeline
Recent News
REGENXBIO expects to resubmit the BLA for clemidsogene lanparvovec (NAVSUNLI) in Q3 2026 after the FDA confirmed existing CAMPSIITE data are sufficient for accelerated approval consideration. NAVSUNLI would be the first potential gene therapy for Hunter syndrome (MPS II).
Leadership Team
Careers & Job Openings