Mar 26, 2024

Opus Reports Vision Improvements for Patients in LCA5 Gene Therapy Clinical Trial

Eye On the Cure Research News

The company plans to administer a higher dose of the emerging gene therapy to the next group of patients.

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Opus Genetics has announced vision improvements for the first three adult patients in its Phase 1/2 gene therapy clinical trial for Leber congenital amaurosis 5 (LCA5), a rare, severe, inherited retinal disease. Some of the patients, who had been almost totally blind since birth, can now see and identify objects for the first time. The company has also reported positive safety data for the trial thus far.

Opus plans to administer the next highest dose of its LCA5 gene therapy to the next cohort of adult patients in mid-2024. The company also has plans sometime in the future to dose patients as young as 13 years of age.

The Phase 1/2 clinical trial is led by Tomas S. Aleman, M.D., at the Center for Advanced Retinal and Ocular Therapeutics (CAROT), Scheie Eye Institute, Department of Ophthalmology of the Perelman School of Medicine, University of Pennsylvania.

“We are delighted to see some vision bestowed to LCA5 patients, enabling them to see objects they’ve never seen before,” says Rusty Kelley, PhD, managing director of the RD Fund, the Foundation’s venture philanthropy arm. “While the trial is still at an early stage, the preliminary data is encouraging for patients and Opus, which is achieving its goal of developing effective gene therapies for inherited retinal diseases which aren’t being addressed by other companies.”

The LCA5 gene therapy clinical trial is the first launched by Opus, a company founded in 2021 by the RD Fund, as a majority shareholder in a $19M Series Seed financing to support emerging retinal disease therapies in or nearing early-stage clinical trials. The company is led by Ben Yerxa, PhD, former chief executive officer of the Foundation.

Known as OPGx-001, the gene therapy uses a human-engineered adeno-associated virus (AAV) to deliver healthy copies of the LCA5 gene to the retinas of patients, augmenting the mutated copies causing vision loss. The therapy is administered through a one-time injection underneath the retina. Researchers believe gene therapies will be effective for many years, perhaps the life of the patient.

Though LCA5 patients have severe vision loss at birth, they have surviving retinal structure which researchers believe can be harnessed for improved vision using gene therapy.