RESEARCH TRIANGLE PARK, N.C.—Opus Genetics, a clinical-stage gene therapy company developing treatments for inherited retinal diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation (RPD) for its ocular gene therapy OPGx-LCA5 to treat patients with the inherited retinal disease LCA5. OPGx-LCA5 is an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene, the announcement said.

“We are thrilled to receive Rare Pediatric Disease designation from the FDA for our OPGx-LCA5 gene therapy," said Ben Yerxa, PhD, chief executive officer of Opus. "This important milestone brings us closer to delivering a potential treatment for patients with LCA5. 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. We look forward to providing updates on the Phase 1/2 clinical trial evaluating OPGx-LCA5 soon,” Yerxa said.

LCA5 is a form of early-onset retinal degeneration that affects approximately one in 1.7 million people in the U.S. Currently, there are no approved treatments for individuals with LCA5-related vision loss, according to the company. OPGx-LCA5 is currently being studied in an open-label, dose-escalation Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in 15 patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene, Opus said.

The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. By obtaining this designation, Opus said it has access to valuable incentives and support from the FDA during its development program. With this designation, OPGx-LCA5 will be eligible to receive a priority review voucher upon approval for any subsequent marketing application that can be sold or transferred to other companies, the company said.