Opus Genetics, Inc. is a clinical-stage biopharmaceutical company dedicated to developing transformative gene therapies for patients with inherited retinal diseases (IRDs). The company's lead gene therapy candidates include OPGx-LCA5, which targets early-onset retinal degeneration caused by LCA5 mutations, and OPGx-BEST1, designed for BEST1-associated retinal diseases. In addition to its AAV-based gene therapy pipeline, Opus Genetics develops small-molecule treatments, notably Phentolamine Ophthalmic Solution 0.75%, which is approved for reversing pharmacologically induced mydriasis and is in late-stage trials for presbyopia and dim light vision disturbances. The current entity was formed following the 2024 acquisition of Opus Genetics by Ocuphire Pharma, after which the combined company adopted the Opus Genetics name and shifted its primary focus to ocular gene therapies. The company operates primarily in the United States and seeks to address rare pediatric and adult blinding conditions with limited existing treatment options.
