REGENXBIO focuses on gene therapy using its NAV Technology Platform, an adeno-associated virus (AAV) gene delivery system with exclusive rights to more than 100 AAV vectors. The company generates revenue by licensing its platform, collecting royalties, and engaging in collaborations, while also developing its own one-time gene therapies for retinal, rare, and neurodegenerative diseases. Key products in later-stage trials include ABBV-RGX-314 for wet AMD and diabetic retinopathy, RGX-202 for Duchenne muscular dystrophy, and RGX-121 for Hunter syndrome, with a goal to advance five new therapies to pivotal-stage or commercial status by 2025 and FDA review for RGX-121 targeted in early 2026. REGENXBIO differentiates itself by owning a broad AAV vector library and offering end-to-end capabilities—from research to manufacturing—alongside a diversified revenue model that includes royalties, collaborations, and milestones.
Company Size
201-500
Company Stage
IPO
Headquarters
Rockville, Maryland
Founded
2009
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