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Opus Genetics is a clinical-stage biopharmaceutical company dedicated to developing first-in-class gene therapies for inherited retinal diseases (IRDs). The company was formed in 2024 through the acquisition of the original Opus Genetics by Ocuphire Pharma, which subsequently rebranded to the Opus Genetics name to focus on genetic medicine. Its lead gene therapy candidate, OPGx-LCA5, is currently in Phase 1/2 clinical trials for the treatment of Leber congenital amaurosis 5, a rare form of childhood blindness. Additionally, the company maintains a portfolio of clinical-stage small molecule assets, including Nyxol for mydriasis reversal and APX3330 for diabetic retinopathy. Led by CEO Dr. George Magrath, Opus Genetics leverages an adeno-associated virus (AAV) platform to address ultra-rare genetic eye disorders.
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