RESEARCH TRIANGLE PARK, N.C., Aug. 3, 2026

Opus Genetics, Inc. has completed patient enrollment in its registrational Phase 3 clinical trial evaluating OPGx-LCA5, an investigational AAV8-based gene therapy for LCA5-associated inherited retinal disease (IRD). The milestone advances the company’s lead ophthalmology program toward a potential Biologics License Application (BLA) following prior alignment with the U.S. Food and Drug Administration (FDA) through the Rare Disease Evidence Principles (RDEP) program. The company plans to begin patient dosing in the fourth quarter of 2026, with topline six-month efficacy results expected by the end of 2027. If successful, Opus Genetics believes the therapy could become the first approved treatment for patients with LCA5-associated inherited retinal disease, a rare inherited disorder that causes severe vision loss and blindness beginning in early childhood.

FDA-Aligned Registrational Trial Targets First Approved LCA5 Therapy

The Phase 3 registrational study is evaluating the safety and efficacy of a one-time subretinal administration of OPGx-LCA5 in patients with genetically confirmed LCA5-associated inherited retinal disease. Developed in collaboration with the FDA through the Rare Disease Evidence Principles (RDEP) program, the study incorporates innovative evidence-generation methods designed for ultra-rare diseases, including a six-month run-in period during which participants serve as their own control before receiving treatment. Based on discussions with the FDA, Opus Genetics expects that a future Biologics License Application may be supported by six-month efficacy data, while 12-month durability data could be submitted during the regulatory review process. This streamlined development strategy is intended to accelerate access to gene therapies for patients with rare inherited retinal disorders.

Gene Therapy Aims to Restore Vision in Rare Inherited Blindness

OPGx-LCA5 is designed to treat Leber congenital amaurosis caused by biallelic mutations in the LCA5 gene, which encodes the lebercilin protein essential for normal retinal function. The investigational therapy utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional copy of the LCA5 gene directly to retinal cells, with the goal of restoring gene function and preserving vision. Previous research has demonstrated that patients with this rare genetic disorder often retain retinal structure despite severe visual impairment, suggesting an opportunity for gene augmentation therapy to improve visual outcomes. The program has received multiple FDA regulatory incentives, including Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT) designations, reflecting both the seriousness of the condition and the potential of the therapy to address a significant unmet medical need.

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Enrollment Milestone Strengthens Opus Genetics’ Gene Therapy Pipeline

Completion of enrollment marks an important clinical and regulatory milestone for Opus Genetics as it advances a growing portfolio of AAV-based gene therapies targeting inherited retinal diseases. Beyond OPGx-LCA5, the company’s pipeline includes investigational programs for BEST1, RDH12, MERTK, RHO, CNGB1, and NMNAT1 gene-associated retinal disorders. The company also expects OPGx-LCA5 may qualify for a Rare Pediatric Disease Priority Review Voucher upon regulatory approval, potentially providing additional strategic value. With dosing scheduled to begin later in 2026 and pivotal efficacy results anticipated in 2027, Opus Genetics continues to position itself at the forefront of developing one-time gene therapies designed to restore vision and prevent blindness in patients affected by rare inherited retinal diseases.

Source: Opus Genetics press release