Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs) — rare genetic conditions that cause progressive vision loss and blindness. Opus uses AAV vectors to deliver functional gene copies directly to retinal photoreceptor cells via subretinal injection, aiming to restore or preserve vision as a one-time treatment. The company has no approved gene therapy products and generates no commercial revenue from its pipeline. Opus' two lead clinical programs target LCA5 (Leber congenital amaurosis type 5) and BEST1-associated retinal disease, both of which have no approved treatments. Opus also has five preclinical gene therapy programs targeting other forms of retinitis pigmentosa and LCA. In parallel, Opus holds rights to Phentolamine Ophthalmic Solution 0.75%, an FDA-approved eye drop sold as RYZUMVI for reversing dilated pupils after eye exams. Opus has licensed RYZUMVI exclusively to Viatris for commercialization, receiving royalties and milestone payments rather than selling the product directly. Viatris is also pursuing regulatory approval for RYZUMVI in presbyopia (age-related near-vision loss) and dim-light vision disturbances following laser surgery. Opus operates as a small, pre-revenue company with 27 full-time employees, outsourcing manufacturing to CDMOs and relying on academic and CRO partnerships for clinical trials. External grants and partner co-funding for several pipeline programs help reduce cash burn.
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