Opus Genetics

Company profile

Opus Genetics company profile

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. The company's pipeline includes gene therapies based on adeno-associated virus (AAV) aimed at treating inherited retinal conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa. Its leading candidate, OPGx-LCA5, is currently in a Phase 1/2 trial for mutations related to LCA5 and has displayed promising early outcomes. Other initiatives encompass OPGx-BEST1, a gene therapy targeting BEST1-related retinal degeneration, as well as a small molecule therapy for diabetic retinopathy that is ready for Phase 3, which has been developed under a Special Protocol Assessment with the FDA.