FDA Grants Rare Pediatric Disease Designation to Opus Genetics for OPGx-LCA5 Therapy

Opus Genetics, a clinical-stage gene therapy company focused on inherited retinal diseases, has announced that its gene therapy OPGx-LCA5 has been granted Rare Pediatric Disease (RPD) designation by the U.S. Food and Drug Administration (FDA). This therapy is being developed to treat Leber congenital amaurosis (LCA5), a severe retinal disorder caused by biallelic mutations in the LCA5 gene.

The CEO of Opus Genetics, Ben Yerxa, Ph.D., expressed enthusiasm about receiving the RPD designation, highlighting it as a significant step toward providing a treatment for those affected by LCA5. He emphasized the company's dedication to advancing therapies for inherited retinal diseases and noted that this designation underscores the potential impact of Opus’s innovative gene therapy. Anticipation is building for forthcoming updates on the ongoing Phase 1/2 clinical trial of OPGx-LCA5.

LCA5 is a rare genetic condition that leads to early-onset retinal degeneration, affecting about one in 1.7 million individuals in the United States. Currently, there are no approved treatments available for vision loss associated with LCA5. OPGx-LCA5 aims to address this gap and is under investigation in an open-label, dose-escalation Phase 1/2 clinical trial at the University of Pennsylvania. This trial is designed to assess the safety and preliminary efficacy of the therapy in 15 patients with inherited retinal degeneration due to mutations in the LCA5 gene.

The FDA’s RPD designation is granted to treatments targeting serious or life-threatening rare diseases primarily affecting patients under 18 years old. This designation provides Opus Genetics with various incentives and support from the FDA throughout the development process. Upon approval, OPGx-LCA5 would be eligible to receive a Priority Review Voucher, which can accelerate the review process for future marketing applications or be sold or transferred to other companies.

OPGx-LCA5 specifically targets a form of Leber congenital amaurosis caused by mutations in the LCA5 gene, which encodes the protein lebercilin. This condition, characterized by early-onset severe retinal dystrophy, presents an opportunity for therapeutic intervention via gene augmentation. OPGx-LCA5 employs an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the retina.

Opus Genetics is a company deeply committed to addressing inherited retinal diseases through gene therapy. Supported by the RD Fund, the venture arm of the Foundation Fighting Blindness, Opus leverages its extensive knowledge and dedication to patient needs. Its portfolio includes AAV-based gene therapies for various orphan retinal diseases, with the OPGx-LCA5 program currently undergoing a Phase 1/2 clinical trial. Based in Research Triangle Park, North Carolina, Opus Genetics aims to develop transformative treatments by harnessing cutting-edge science and the expertise of leaders in ocular gene therapy.

The company’s mission is to address some of the most neglected forms of inherited blindness and enhance the manufacturing scale and efficiency of orphan products. By focusing on innovative approaches to gene therapy for retinal diseases, Opus Genetics strives to bring new hope and potential treatments to patients affected by these debilitating conditions.