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Opus Genetics

IRD Rare disease Lead: Approved 1 Biotech Distilled article ↓

Ophthalmology Gene Therapy Small Molecule

Most recent annual filing: 2026-03-15

Pipeline (12 active programs)

OPGx-LCA5

Gene Therapy Target: LCA5

Leber Congenital Amaurosis Phase 1 subretinal University of Pennsylvania

OPGx-BEST1

Gene Therapy Target: BEST1

Best Vitelliform Macular Dystrophy Phase 1 subretinal
Autosomal Recessive Bestrophinopathy Phase 1 subretinal

OPGx-RHO

Gene Therapy Target: RHO

Retinitis Pigmentosa Preclinical

OPGx-RDH12

Gene Therapy Target: RDH12

Leber Congenital Amaurosis Preclinical

OPGx-MERTK

Gene Therapy Target: MERTK

Retinitis Pigmentosa Preclinical Cleveland Clinic Abu Dhabi

OPGx-NMNAT1

Gene Therapy Target: NMNAT1

Leber Congenital Amaurosis Preclinical

OPGx-CNGB1

Gene Therapy Target: CNGB1

Retinitis Pigmentosa Preclinical

RYZUMVI

phentolamine ophthalmic solution 0.75%

Small Molecule Target: alpha-1 adrenergic receptor, alpha-2 adrenergic receptor

Pharmacologically Induced Mydriasis Approved topical Viatris
Presbyopia NDA Submitted topical Viatris
Vision Disturbances After Keratorefractive Surgery Phase 3 topical Viatris

APX3330

Small Molecule Target: APE1/Ref-1

Diabetic Retinopathy Phase 2 oral

Biotech Distilled coverage (1)

2026-06-17 Opus Genetics (IRD) — Scientific Deep Dive for OPGx-LCA5, OPGx-BEST1, and the IRD Gene Therapy Pipeline

Articles open on Substack. Some deep dives are subscriber-only.