Opus Genetics receives Pediatric Disease Designation from FDA for OPGx-LCA5 to treat rare inherited retinal disease LCA5

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Opus Genetics announced the FDA has granted Rare Pediatric Disease designation (RPD) for its ocular gene therapy OPGx-LCA5 to treat patients with the inherited retinal disease LCA5.

According to the company, 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 (LCA5).¹

Ben Yerxa, PhD, CEO of Opus, said the company is pleased to receive Rare Pediatric Disease designation from the FDA for its OPGx-LCA5 gene therapy.

“This important milestone brings us closer to delivering a potential treatment for patients with LCA5,” Yerxa said in the 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.”

LCA5 is a form of early-onset retinal degeneration that affects approximately 1 in 1.7 million people in the United States. There currently are no approved treatments for individuals with LCA5-related vision loss. OPGx-LCA5 is currently being studied in an open-label, dose-escalation Phase 1/2 clinical trial (NCT05616793) at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in 15 patients who were diagnosed with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

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 securing the designation, Opus has access to incentives and support from the FDA during its development program.

With the 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.

About OPGx-LCA5

The therapy is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5 is an early-onset severe inherited retinal dystrophy. Studies in LCA5 patients have uncovered evidence for the dissociation of retinal architecture and visual function in the disease, pointing to an opportunity for therapeutic intervention through gene augmentation.

Moreover, OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina.¹

Reference:

1. Opus Genetics. Opus Genetics Receives Rare Pediatric Disease Designation from the U.S. FDA for Ocular Gene Therapy OPGx-LCA5 to Treat Rare Inherited Retinal Disease LCA5. GlobeNewswire News Room. Published August 20, 2024. Accessed August 20, 2024. https://www.globenewswire.com/news-release/2024/08/20/2932762/0/en/Opus-Genetics-Receives-Rare-Pediatric-Disease-Designation-from-the-U-S-FDA-for-Ocular-Gene-Therapy-OPGx-LCA5-to-Treat-Rare-Inherited-Retinal-Disease-LCA5.html