IRD

Opus Genetics, Inc., a clinical-stage biopharmaceutical company, develops gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs) and other types of therapies for additional ophthalmic disorders. It develops OPGx-LCA5, an early-onset retinal degeneration that is in Phase 1/2 clinical trial for the treatment of LCA5-associated IRD; and OPGx-BEST1 to treat BEST1-associated retinal disease. The company also develops various pre-clinical IRD programs, including OPGx-RHO, a gene therapy that targets autosomal dominant retinitis pigmentosa caused by RHO mutations; OPGx-RDH12 that is designed to restore protein expression and halt functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase gene; OPGx-MERTK, an AAV vector for the treatment of MERTK IRD; OPGx-NMNAT1, a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 gene; and OPGx-CNGB1, an AAV gene therapy for retinitis pigmentosa due to mutations in the CNGB1 gene. In addition, it develops Phentolamine Ophthalmic Solution for reversal of mydriasis, as well as for the treatment of presbyopia and dim light or night vision disturbances and APX3330, a small-molecule inhibitor of Ref-1 for the treatment of diabetic retinopathy. Opus Genetics, Inc. was formerly known as Ocuphire Pharma, Inc. and changed its name to Opus Genetics, Inc. in November 2020. The company was founded in 2018 and is headquartered in Durham, North Carolina.