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On August 20, Opus Genetics announced that the FDA has granted its ocular gene therapy OPGx-LCA5 Rare Pediatric Disorder Qualification (RPD) for the treatment of the inherited retinal disorder LCA5. OPGx-LCA5 is an adeno-associated virus 8 (AAV8) vector designed to accurately deliver the functional LCA5 gene to the outer retina of patients with Leber congenital amaurosis (LCA) caused by a biallelic mutation of the LCA5 gene (LCA5). With this designation, OPGx-LCA5 will be eligible to receive priority review vouchers upon approval for any subsequent marketing applications that can be sold or transferred to other companies.
The LCA5 gene therapy clinical trial was first initiated by Opus, a company founded by RD Fund in 2021 as a majority shareholder in a $19 million series of seed funding to support emerging retinal disease therapies in or near early-stage clinical trials. The company is led by Dr. Ben Yerxa, former CEO of the Foundation Fighting Blindness. The gene therapy, called OPGx-001, uses a human engineered adeno-associated virus (AAV) to deliver a healthy copy of the LCA5 gene to a patient's retina, increasing the mutant copy that causes vision loss. The treatment is administered by a one-time injection just below the retina. The researchers believe the gene therapy will be effective for many years, and possibly even for a patient's lifetime. LCA5 is an early-onset retinal degeneration. It affects about one in 1.7 million people in the United States. Currently, there are no approved treatments for patients with LCA5-related vision loss. The University of Pennsylvania is currently conducting an open-label, dose-escalation Phase 1/2 clinical trial to evaluate the safety and initial efficacy of OPGx-LCA5 in 15 patients with inherited retinal degeneration due to a biallelic mutation of the LCA5 gene.
"We are pleased that our OPGx-LCA5 gene therapy has received FDA Rare pediatric disease designation. This important milestone brings us one step closer to providing a potential treatment for LCA5 patients, "said Dr. Ben Yerxa, CEO of Opus. "At Opus, we are committed to advancing therapies that help treat patients with inherited retinal diseases, and this designation further validates the potential impact of our innovative eye gene therapy approach." We look forward to providing an update soon on the Phase 1/2 clinical trial evaluating OPGx-LCA5."
BOC Sciences offers comprehensive RNA services, including raw material supply and custom synthesis, designed to support the development and application of gene therapies. We provide high-quality RNA materials such as siRNA, miRNA, and mRNA, ensuring their stability and effectiveness in gene therapy applications. With our advanced technologies and precise processes, BOC Sciences is committed to advancing gene therapy and helping clients achieve breakthroughs in treating genetic and gene-related diseases.
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OPGx-LCA5 is designed to treat a form of Leber congenital amaurosis (LCA), which is caused by biallelic mutations in the LCA5 gene (LCA5), which encodes the Lebcillin protein. LCA5 is an early onset severe hereditary retinal dystrophy. OPGx-LCA5 uses an AAV8 vector to precisely deliver a functional LCA5 transgene to the outer retina for the treatment of Leber congenital amaurosis due to biallelic mutations in the LCA5 gene. In this cohort of patients, OPGx-LCA5 demonstrated good safety and tolerability, as well as biological activity.
Opus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and goals. Supported by the RD Fund, the venture capital arm of the Foundation Against Blindness, Opus combines unparalleled insight and commitment to patient needs with a number of wholly-owned programs for orphan retinal diseases. Its AAV-based gene therapy portfolio, including the de-risking LCA5, is currently in Phase 1/2 clinical trials and aims to address some of the most neglected forms of inherited blindness while creating new orphan drug production scale and efficiency. Headquartered in Research Triangle Park, North Carolina, the company uses the best scientific knowledge and expertise of eye gene therapy pioneers to transparently deliver transformative treatments to patients..
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