Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Stage not disclosed · ActiveModality: gene therapy
Target LCA5
Partnerships University of Pennsylvania (LCA5/RDH12 license agreement)
Indications
Leber congenital amaurosis (LCA5 mutations)(Stage not disclosed)
Catalysts
— on or prior to March 31, 2028
Status note AAV-based gene therapy evaluated for treatment of LCA caused by LCA5 gene mutations; note financing tranches tied to FDA application acceptance and approval milestones.
OPGx-BEST1
Stage not disclosed · ActiveModality: gene therapy
Target BEST1
Partnerships Iveric Bio (asset purchase; BEST1/RHO licenses from Penn/University of Florida)
Indications
Retinal diseases (BEST1 mutations)(Stage not disclosed)
Status note AAV-based gene therapy evaluated for retinal diseases associated with BEST1 mutations (DUO-1001 trial referenced).
Phentolamine Ophthalmic Solution 0.75%
Approved · ActiveModality: small molecule
Aliases RYZUMVI
Target adrenergic receptors (alpha-1, alpha-2)
Partnerships Viatris (exclusive license to develop/manufacture/commercialize the refractive product candidate)
Indications
Pharmacologically induced mydriasis(Approved)
Presbyopia(Stage not disclosed)
Decreased vision under mesopic conditions after keratorefractive surgery(Stage not disclosed)
Status note FDA-approved and commercially launched (Apr 2024) as RYZUMVI for pharmacologically induced mydriasis; also in development for presbyopia and low-light visual disturbances after keratorefractive surgery.
OPGx-RDH12
Preclinical · ActiveModality: gene therapy
Target RDH12
Partnerships University of Pennsylvania (LCA5/RDH12 license agreement)
Indications
Leber Congenital Amaurosis(Phase 2)
Leber Congenital Amaurosis (LCA)(Phase 2)
Status note Preclinical; co-funded by Foundation Fighting Blindness and NIH. RDH12-associated early-onset retinal disease.
OPGx-MERTK
Preclinical · ActiveModality: gene therapy
Target MERTK
Partnerships Foundation Fighting Blindness Retinal Degeneration Fund (Jun 2025 funding agreement, up to $2M non-dilutive)
Indications
Retinitis pigmentosa (MERTK variants)(Stage not disclosed)
Status note Preclinical; co-funded by Global RDH12 Alliance. MERTK-related autosomal recessive retinal dystrophy.
OPGx-RHO
Preclinical · ActiveModality: gene therapy
Target RHO
Partnerships Iveric Bio (asset purchase; RHO license from Penn/University of Florida)
Status note Preclinical. CNGB1-related autosomal recessive retinitis pigmentosa.
OPGx-NMNAT1
Preclinical · ActiveModality: gene therapy
Target NMNAT1
Partnerships Massachusetts Eye and Ear Infirmary (Nov 2021 license for NMNAT1 program)
Indications
Retinal disease(Stage not disclosed)
Status note Preclinical. NMNAT1 enzyme for NAD+ regeneration; clinical development activities expected at Cleveland Clinic Abu Dhabi in 2026.
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 (3 as sponsor, 0 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.
Get the weekly biotech catalyst rundown. Cash runways, financings, and trial readouts for all 714 stocks.