Opus Genetics, Ocuphire Merger to Boost Advancement of Several IRD Gene Therapies

Opus Genetics, an inherited retinal disease (IRD) gene therapy company launched in 2021 by the Foundation’s RD Fund, has merged with Ocuphire, a clinical stage, ophthalmic, biopharmaceutical company. The newly formed company will be called Opus Genetics. The company’s stock will begin trading on the NASDAQ exchange on October 24 under the symbol “IRD.” Ocuphire chief executive officer, George Magrath, MD, will continue in that role with the newly formed company. Ben Yerxa, PhD, former chief executive officer at Opus, will serve as president. The new company will continue developing several emerging gene therapies for IRDs.

“We are delighted with the merger because it will enable Opus to continue its mission of developing several gene therapies for inherited retinal diseases,” says Rusty Kelley, PhD, a founding director of Opus and managing director, RD Fund, the Foundation’s venture philanthropy arm. “This is great news for the IRD gene therapy field and people affected by a variety of challenging retinal conditions.”

Opus’s most advanced program is a gene therapy for Leber congenital amaurosis 5 (LCA5), which causes significant vision loss in infancy. In a Phase 1/2 clinical trial, the LCA5 gene therapy restored vision in three legally blind adults. All three had improvements in light sensitivity and mobility. The improvements were sustained at six months. The treatment was well tolerated and no serious adverse events were reported. The company plans to enroll pediatric LCA5 patients in the trial during the first quarter of 2025.

Opus also reported it is ready to begin a Phase 1/2 clinical trial in Germany for its BEST1 gene therapy. Mutations in BEST1 can cause a few different retinal diseases including Best disease and retinitis pigmentosa (RP). BEST1-associated disease usually affects central vision and is slow progressing. Successful, Foundation-funded canine studies of BEST1 gene therapy at the University of Pennsylvania helped the company gain regulatory authorization for the clinical trial.

The company is also developing IRD gene therapies for people with mutations in: RHO, RDH12, MERTK, NMNAT-1, and CNGB1.