Opus Genetics receives FDA incentive for retinal gene therapy

Opus Genetics, a clinical-stage gene therapy company devoted to treating inherited retinal diseases, has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration for one of its potential therapies. 

The Research Triangle Park-based company received the designation for OPGx-LCA5, an ocular gene therapy for treating patients with LCA5, a form of early-onset retinal degeneration that causes vision loss.

The FDA designation is for therapeutics intended to treat serious or life-threatening rare diseases that primarily affect patients under the age of 18. With the designation, Opus will be eligible upon FDA approval of OPGx-LCA5 to receive a priority review voucher (PRV) that could be used to advance another internal therapy or be sold or transferred to another company. The PRV program incentivizes companies like Opus to invest in developing rare pediatric diseases that have limited markets.

“This important milestone brings us closer to delivering a potential treatment for patients with LCA5,” said Ben Yerxa, Ph.D., chief executive officer of Opus, who also serves on the North Carolina Biotechnology Center’s board of directors. “At Opus, we’re committed to advancing therapies that help treat patients with inherited retinal diseases, and this designation further validates the potential impact of our innovative ocular gene therapy approach.”

LCA5 is a type of Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene. The gene encodes a protein vital to the retina, the layer of cells within the eye that helps translate light into vision. 

The disorder affects about one in every 1.7 million people in the United States, and there are no approved treatments for it.

Clinical trial under way

Opus’ therapy, OPGx-LCA5, uses an adeno-associated virus 8 (AAV8) vector to deliver a functional LCA5 gene to cells in the outer retina. The AAV technology is based on the work of Opus’ scientific founders, Jean Bennett, M.D., Ph.D., of the University of Pennsylvania, and Eric Pierce, M.D., Ph,D., of Harvard Medical School and Massachusetts Eye and Ear.

The OPGx-LCA5 therapy is being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania to determine its safety and preliminary efficacy in 15 patients with inherited retinal degeneration due to mutations in the LCA5 gene.

“We look forward to providing updates on the Phase 1/2 clinical trial evaluating OPGx-LCA5 soon,” Yerxa said.

Opus has additional gene therapies in earlier stages of development for other retinal diseases including Best disease (vitelliform macular dystrophy), retinitis pigmentosa and different forms of LCA.

Opus, founded in 2021, is backed by the RD Fund, the venture capital arm of Foundation Fighting Blindness, a non-profit organization that supports research toward preventions, treatments and cures for blinding diseases. Other investors in the company’s recent fundraising efforts include the venture capital firms Bios Partners and Michigan Capital Network, Yerxa said.