Opus Genetics (IRD)

The company’s pipeline is built largely around adeno-associated virus, or AAV, gene therapy programs. AAV vectors are widely used in genetic medicine because they can deliver functional genetic material to targeted cells, including cells in the eye. Opus Genetics applies this approach to retinal diseases caused by specific genetic mutations, aiming to address the underlying biological drivers of disease rather than only managing symptoms.

Its lead programs include OPGx-LCA5, which targets Leber congenital amaurosis associated with LCA5 mutations, and OPGx-BEST1, which is directed at retinal degeneration linked to BEST1 mutations. Additional candidates in development target genes including RHO, CNGB1, RDH12, NMNAT1 and MERTK, giving the company a portfolio that spans several rare inherited retinal disorders.

Opus Genetics operates as a single business focused on products related to vision health and visual performance. As a clinical-stage company, it is primarily engaged in research, preclinical development, clinical testing, regulatory planning and the manufacturing work required to advance potential therapies.

The company is distinctive because it concentrates on genetically defined eye diseases, where diagnosis can be tied to a specific mutation and where localized delivery to the retina may allow targeted treatment. Its prospects depend on clinical trial progress, safety and efficacy data, regulatory outcomes and the ability to scale specialized gene therapy development.