Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
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.
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 · ActiveModality: 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 · ActiveModality: 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.
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.
Sponsor-matched active trials from ClinicalTrials.gov (7 as sponsor, 7 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
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