Opus Genetics Receives FDA Rare Pediatric Disease Designation for Gene Therapy OPGx-LCA5

Opus Genetics announced that the FDA has granted rare pediatric disease (RPD) designation for its ocular gene therapy, OPGx-LCA5. This therapy is designed to treat patients with Leber congenital amaurosis (LCA) type 5, a rare inherited retinal disease caused by biallelic mutations in the LCA5 gene.

OPGx-LCA5 utilizes an adeno-associated virus 8 (AAV8) vector to deliver a functional LCA5 gene directly to the outer retina of affected patients. The goal is to restore or preserve vision in individuals suffering from LCA5-related vision loss.

LCA5 is a severe, early-onset form of retinal degeneration that affects approximately one in 1.7 million people in the US. Currently, there are no approved treatments for this condition.

“We are thrilled to receive rare pediatric disease designation from the FDA for our OPGx-LCA5 gene therapy. This important milestone brings us closer to delivering a potential treatment for patients with LCA5,” Ben Yerxa, PhD, Chief Executive Officer of Opus Genetics, said in a company news release. “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.”

The FDA'srare pediatric disease designation is granted to therapies targeting serious or life-threatening rare diseases that primarily affect individuals under the age of 18. This designation provides Opus Genetics with various incentives and support throughout the development of OPGx-LCA5. Additionally, it makes the therapy eligible to receive a priority review voucher upon approval, which can be used for any subsequent marketing application or sold or transferred to another company.

Opus is conducting an open-label, dose-escalation phase 1/2 clinical trial at the University of Pennsylvania to evaluate the safety and preliminary efficacy of OPGx-LCA5 in 15 patients with LCA5-related vision loss. For more information on the trial, visit clinicaltrials.gov (NCT05616793).