Opus Genetics gains momentum in inherited retinal disease treatments

RTP-based Opus Genetics has announced several advances in its gene therapy programs designed to restore vision and help prevent blindness in patients with inherited retinal diseases.

In one key development, the company’s OPGx-LCA5 investigational LCA5 gene therapy program has been accepted into the U.S. Food and Drug Administration’s Rare Disease Evidence Principles (RDEP) program.

OPGx-LCA5 is a potential gene therapy for Leber congenital amaurosis type 5 (LCA5), a rare inherited retinal disease that leads to early-onset, progressive vision loss, often resulting in severe visual impairment or blindness in childhood.

RDEP is a new FDA initiative designed to provide greater speed and predictability in the review of therapies for rare diseases with very small patient populations and significant unmet medical need, that are driven by a known genetic defect. 

“Given the rarity and severity of this disease, early engagement with the FDA alongside our RMAT designation will help inform a more efficient and streamlined development pathway,” said George Magrath, M.D., chief executive officer of Opus Genetics. “We look forward to collaborating with the FDA as we pursue a potential treatment option for patients affected by this devastating inherited retinal disease.”

OPGx-LCA5 has also received Rare Pediatric Disease, Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) FDA designations.

Treating BEST1 retinal diseases

Opus Genetics also reported progress in its BEST1 retinal disease program. The company recently completed enrollment in Cohort 1 of its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy for BEST1-related inherited retinal diseases, including best vitelliform macular dystrophy (BVMD) and autosomal-recessive bestrophinopathy (ARB). 

The adaptive, open-label study is evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 at up to two dose levels in adults. Five participants are enrolled, three with BVMD and two with ARB. 

“As planned, we enrolled a mix of participants with the dominant and recessive forms of BEST disease to evaluate OPGx-BEST1 in both conditions,” said Magrath. “In the dominant participants, we completed the added step of using an in vitro platform to confirm that each participant’s disease mutation is amenable to gene augmentation.”

Three-month results from the first participant treated in the study were presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. The results highlighted positive tolerability and biological activity following subretinal administration of OPGx-BEST1.

Opus expects to announce 3-month topline data from Cohort 1 in September 2026. 

Restoring cone-mediated vision 

Additional findings presented at ARVO included 6-month results from the company’s ongoing Phase 1/2/3 study of OPGx-LCA5 in pediatric patients with Leber congenital amaurosis type 5 (LCA5), a severe, early-onset, inherited retinal degeneration.

Despite advanced disease and very poor baseline vision, the pediatric patients treated with OPGx-LCA5 demonstrated robust and consistent restoration of cone-mediated function following a single subretinal injection. These results build on prior findings in adults and suggest that even in severe pediatric disease, viable cone photoreceptors may still be preserved, pointing to a potentially broader therapeutic window.

“Our ARVO presentations reflect a meaningful shift in what gene therapy can achieve in retinal disease,” said Magrath. “We are seeing consistent evidence that we may be able to rapidly restore visual function, with encouraging clinical signals and a growing foundation across multiple programs for patients with genetic blinding diseases.”