Dec 2, 2022

Opus Genetics to Launch Gene Therapy Clinical Trial for LCA5 Patients

Eye On the Cure Research News

The LCA5 gene therapy will be the first emerging Opus treatment to move into a human study

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Opus Genetics, a company developing gene therapies for people with inherited retinal diseases, has received authorization from the US Food & Drug Administration (FDA) to launch a clinical trial for its emerging gene therapy for Leber congenital amaurosis 5 (LCA5), which causes significant vision loss in children with mutations in the gene that expresses the protein lebercillin. The Phase 1/2 clinical trial, enrolling nine adult patients, will take place at the University of Pennsylvania. The company plans to begin patient enrollment in the first quarter of 2023.

The LCA5 gene-therapy clinical trial will be the first launched by Opus, a company originally conceived and formed by the Foundation Fighting Blindness. Founded in 2021, Opus received $19 million in seed funding from the Foundation’s RD Fund, a venture philanthropy fund for 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 LCA5 gene therapy will use 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 will be delivered through a one-time injection underneath the retina. Researchers believe gene therapies will be effective for many years, perhaps the life of the patient. Preclinical data, from studies in animal and human cell models, have demonstrated preservation of retinal structure and visual function when OPGx-001 was administered prior to peak disease severity.

“We are delighted to see Opus moving into the clinic with its LCA5 program, founded by our distinguished colleague and gene therapy luminary Dr. Jean Bennett,” says Rusty Kelley, managing director of the RD Fund. “Excitingly, Opus is also developing gene therapies for LCA13, which is caused by RDH12 mutations, and LCA9, which is caused by NMNAT-1 mutations. The company’s goal is to continue building sequentially a pipeline of new gene therapies to address a variety of unmet inherited retinal diseases, including LCA, macular dystrophies, and retinitis pigmentosa.”