RGNX (REGENXBIO Inc.) stock: $8.03, $530.1M market cap, $199.2M cash, $330.9M EV, $539.3M fully diluted market cap. Lead program: RGX-121-3102 (MPS II), Phase 3. Data from Fully Diluted's biotech stocks database.
Pipeline assets
ABBV-RGX-314
Phase 2/3 · Active Modality: gene therapy (AAV vector, NAV Technology Platform)
Aliases surabgene lomparvovec, sura-vec
Target VEGF
Partnerships AbbVie (collaboration to develop and commercialize ABBV-RGX-314)
Indications- Wet age-related macular degeneration (wet AMD) (Stage not disclosed)
- Diabetic retinopathy (DR) (Phase 2/3)
Status note Lead product candidate developed with AbbVie. First patient dosed in the Phase 2b/3 diabetic retinopathy trial (NAAVIGATE) June 2026. Multiple wet AMD trials ongoing including AAVIATE, ALTITUDE, ASCENT and ATMOSPHERE.
RGX-121
Phase 3 · Active Modality: gene therapy (AAV vector, NAV Technology Platform)
Aliases clemidsogene lanparvovec
Target iduronate-2-sulfatase (IDS)
Partnerships Nippon Shinyaku (collaboration for development and commercialization; double-digit royalties on net sales in licensed territories)
Indications- Mucopolysaccharidosis Type II (MPS II) (Stage not disclosed)
Status note FDA Complete Response Letter received February 2026; planned BLA resubmission. Company retains rights to any priority review voucher upon approval.
RGX-111
Stage not disclosed · Active Modality: gene therapy (AAV vector, NAV Technology Platform)
Target IDUA
Partnerships Nippon Shinyaku (collaboration for development and commercialization)
Indications- Mucopolysaccharidosis Type I (MPS I) (Stage not disclosed)
Status note FDA imposed a clinical hold on the RGX-111 program January 28, 2026. Company retains rights to any priority review voucher upon approval.
RGX-202
Stage not disclosed · Active Modality: gene therapy (AAV vector, NAV Technology Platform)
Target microdystrophin
Indications- Duchenne muscular dystrophy (Stage not disclosed)
Status note Internal lead candidate for Duchenne; AFFINITY DUCHENNE trial ongoing.
Zolgensma (royalty interest)
Approved · Active
Target SMN1 (AAV9 gene replacement)
Partnerships Novartis (NAV Technology license)
Indications- Spinal muscular atrophy (SMA), pediatric (IV gene replacement) (Approved)
Status note Commercial product developed by NAV Technology Licensee; company receives royalties on net sales.
Itvisma (royalty interest)
Approved · Active
Target SMN1 (AAV9 gene replacement)
Partnerships Novartis (NAV Technology license)
Indications- Spinal muscular atrophy (SMA), children 2+ / teens / adults (intrathecal gene replacement) (Approved)
Status note Commercial product developed by NAV Technology Licensee; company receives royalties on net sales.
Per-asset detail extracted from the 10-Q filed 2026-08-06. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.