Opus Genetics Inc IRD

Healthcare

Opus Genetics, Inc., formerly Ocuphire Pharma, Inc., is a clinical-stage ophthalmic biopharmaceutical company. The Company is engaged in developing therapies to treat patients with inherited retinal diseases (IRDs) and therapies to treat patients with other retinal and refractive disorders. Its pipeline includes adeno-associated virus (AAV)-based gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. Its most advanced gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and being evaluated in a Phase I/II open-label, dose-escalation trial, with advancing early data. The pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist to reduce pupil size, and APX3330, a novel small-molecule inhibitor of Ref-1 to slow the progression of non-proliferative diabetic retinopathy.