Headquartered in Rockville, MD Fiscal year ends December NASDAQ: RGNX regenxbio.com
REGENXBIO Inc. is a clinical-stage biotechnology company focused on adeno-associated virus (AAV) gene therapy. It is developing a late-stage pipeline of one-time treatments for rare and retinal diseases.
Lead programs:
- **RGX-202:** for Duchenne muscular dystrophy, in the Phase I/II/III AFFINITY DUCHENNE trial. A BLA submission under the accelerated approval pathway is planned for Q1 2027.
- **Surabgene lomparvovec (sura-vec, ABBV-RGX-314):** for wet age-related macular degeneration and diabetic retinopathy, developed with AbbVie. Pivotal wet AMD data are expected in Q4 2026, and the Phase IIb/III NAAVIGATE diabetic retinopathy study is enrolling.
- **RGX-121 (clemidsogene lanparvovec) and RGX-111:** for MPS II (Hunter syndrome) and MPS I, both partnered with Nippon Shinyaku. RGX-121 is currently on FDA clinical hold.
The company earns license and royalty revenue from its AAV platform, including royalties on Novartis' ZOLGENSMA and ITVISMA. ZOLGENSMA royalties have declined since U.S. patent expiry in January 2026, while ITVISMA royalties are supported by patents extending to 2037. It also earns service revenue and partner milestone payments. REGENXBIO operates an in-house Manufacturing Innovation Center and is producing intended commercial supply of RGX-202 there. Updated
The smaller-than-expected loss came from a $38.0 million Sarepta litigation gain: Zolgensma royalties fell $21.2 million after U.S. patents expired in January, leaving revenue 41.6% short of estimates. Cash plus the settlement funds operations only into late Q4 2027, and pivotal wet AMD data for sura-vec lands before year end.
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REGENXBIO Inc. is a clinical-stage biotechnology company focused on adeno-associated virus (AAV) gene therapy. It is developing a late-stage pipeline of one-time treatments for rare and retinal diseases.
The Wall Street consensus 12-month price target for RGNX is $21.40.
