Opus Genetics Receives FDA Rare Pediatric Disease Designation for OPGx-LCA5 Gene Therapy
Research Triangle Park, N.C. – August 20, 2024 – Opus Genetics has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for its ocular gene therapy, OPGx-LCA5, which aims to treat patients with the inherited retinal disease Leber congenital amaurosis type 5 (LCA5). OPGx-LCA5 utilizes an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina in patients with LCA5, a condition caused by biallelic mutations in the LCA5 gene.
"We are thrilled to receive Rare Pediatric Disease designation from the FDA for our OPGx-LCA5 gene therapy," said Ben Yerxa, Ph.D., 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."
LCA5 is a form of early-onset retinal degeneration affecting approximately one in 1.7 million individuals in the United States. Currently, there are no approved treatments for LCA5-related vision loss. OPGx-LCA5 is being investigated in an open-label, dose-escalation Phase 1/2 clinical trial at the University of Pennsylvania. This study is designed to assess the safety and preliminary efficacy of OPGx-LCA5 in 15 patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.
For more information on the clinical trial, visit clinicaltrials.gov (NCT05616793)
The FDA's Rare Pediatric Disease designation is awarded to therapeutics aimed at treating serious or life-threatening rare diseases that primarily affect individuals under 18 years old. With this designation, Opus gains access to valuable incentives and support from the FDA throughout its development program. Additionally, OPGx-LCA5 will be eligible to receive a priority review voucher upon approval of any subsequent marketing application. This voucher can be sold or transferred to other companies.
About OPGx-LCA5
OPGx-LCA5 is a gene therapy designed to address Leber congenital amaurosis type 5 (LCA5), a severe early-onset inherited retinal dystrophy caused by biallelic mutations in the LCA5 gene. The LCA5 gene encodes the protein lebercilin. Research on LCA5 patients has shown a dissociation between retinal architecture and visual function, highlighting a potential for therapeutic intervention through gene augmentation.
OPGx-LCA5 employs an adeno-associated virus 8 (AAV8) vector to deliver a functional LCA5 gene to the outer retina. Currently, OPGx-LCA5 is under evaluation in a Phase 1/2 clinical trial at the University of Pennsylvania, aimed at determining its safety and preliminary efficacy in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.
