Opus Genetics Reports Positive Gene Therapy Data in Pediatric Patients with Ultrarare Eye Disease

CHAPEL HILL, N.C.-Sep 30, 2025 – Opus Genetics has announced new clinical data for its investigational adeno-associated virus (AAV) vector-based gene therapy designed to treat children with Leber Congenital Amaurosis Type 5 (LCA5), an ultrarare inherited retinal disease that affects approximately 200 people in the U.S.

The company is developing the gene augmentation therapy, which uses an AAV8 vector and the same promoter technology as Roche’s Luxturna, to deliver a functional LCA5 gene to address the photoreceptor impairment that causes severe vision loss.

Pediatric Data Shows Encouraging Early Improvement

The new data set expands on Opus’ previous findings in adults and focuses on the first three adolescent patients enrolled, aged 16 to 17, who had severe vision impairment at baseline.

Key findings include:

• Average Improvement: The cohort showed an average improvement of 0.3 logMAR as of the data cutoff. Each 0.1 logMAR shift represents a one-line change on a standard eye test chart.
• Individual Responses: Two patients achieved improvements of 0.2 and 0.7 logMAR at three months post-treatment. The third patient saw a 0.5 logMAR improvement after only one month.
• Durability Comparison: Opus noted that the early pediatric improvements compare favorably to the company’s adult cohort, which demonstrated durable vision improvements extending through 18 months of follow-up.

Safety Profile Supports Path Forward

The top-line pediatric safety data largely reinforced the encouraging signs reported in adults. No adverse events were found to be related to the study drug. One pediatric participant experienced a worsening of a preexisting cataract, which Opus attributed to the surgical procedure used for gene therapy delivery, not the drug itself. The worsening did not obscure improvements in retinal sensitivity.

Following this data release, Opus Genetics plans to meet with the FDA in the fourth quarter to determine the next clinical development steps. The company previously stated that an accelerated clinical development pathway toward a filing for FDA approval could be possible if efficacy results in children align with the results observed in adults.

The positive data was well-received by investors, with shares in Opus Genetics rising more than 3% in premarket trading Tuesday morning.