Companies in #Autosomal dominant retinitis pigmentosa
Found 1 company operating in this sector.
Opus Genetics (NASDAQ: IRD), formerly Ocuphire Pharma and renamed in November 2020, is a Durham, North Carolina-based clinical-stage biopharmaceutical company (founded 2018) developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs) and other ophthalmic disorders. Lead clinical programs are OPGx-LCA5, an AAV gene therapy in Phase 1/2 for LCA5-associated retinal degeneration, and OPGx-BEST1, in a Phase 1/2 trial (BIRD-1) for BEST1-associated retinal disease — which reported positive low-dose Cohort 1 data in September 2026 and advanced to a higher-dose Cohort 2. Preclinical IRD programs include OPGx-RHO (autosomal dominant retinitis pigmentosa), OPGx-RDH12, OPGx-MERTK, OPGx-NMNAT1 and OPGx-CNGB1. The company also develops phentolamine ophthalmic solution for presbyopia and dim-light vision disturbances and APX3330, a small-molecule Ref-1 inhibitor for diabetic retinopathy. In August 2026 Opus Genetics reported a successful FDA Type C meeting on OPGx-BEST1 that aligned on pivotal-trial endpoints and a development path with potential Phase 3 dosing in 2027.