Regenxbio hit with another clinical hold for Hunter syndrome gene therapy
Regenxbio has been hit with another clinical hold for its Hunter syndrome gene therapy program, raising safety concerns.
REGENXBIO
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
Sector:Biotechnology / Gene Therapy
Headquarters:Rockville, Maryland, USA
Founded:2009
Website:www.regenxbio.com/
Upcoming Milestones & Key Dates
Mid-2026
FDA Meeting
FDA Type A meeting to discuss CRL and path forward for RGX-121 in Hunter syndrome
Q2 2026
Earnings
Q1 2026 Earnings — Pipeline update and RGX-202 pivotal trial enrollment
Clinical Development Pipeline
RGX-202
Target: Microdystrophin transgene delivered via AAV8RGX-121
Target: Iduronate-2-sulfatase (IDS) gene via AAV9RGX-314
Target: Anti-VEGF Fab delivered via AAV8 (subretinal or suprachoroidal)Recent News
Safety concerns spur FDA to again halt testing of Regenxbio gene therapy
Safety concerns spur FDA to again halt testing of Regenxbio gene therapy
FDA Clears Path for NAVSUNLI BLA Resubmission in MPS II, No Additional Studies Required
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).