Six Eyes, One Dose, a Cane Left at the Door

Three adults and three adolescents. One injection each, under the retina, at the low dose. That is the whole human evidence Opus Genetics put on a screen in Rio de Janeiro on 15 October.

The disease is Leber congenital amaurosis type 5. A fault in the LCA5 gene means the retina does not make lebercilin. Vision goes early. The company has said about 200 people live with it. OPGx-LCA5 is an AAV8 gene therapy, a one-time subretinal dose, built to put a working copy of the gene into photoreceptors. The low dose in the Phase 1/2 study was 1 times 10 to the 10 vector genomes per eye. The trial number is NCT05616793.

The three adults still showed a visual-acuity gain at 24 months. The three adolescents showed cone-mediated sensitivity sitting in the normal range through six months. Both groups did better on full-field stimulus testing in the treated eye than in the eye that was not injected. That untreated eye is the control a trial this small can actually offer. Microperimetry showed retinal sensitivity up, and fixation shifting toward the centre of the retina, where detail lives.

The company also reported what people said they could do. Navigate without a cane. Recognise a family member. Name a colour. Those are not endpoints. They are the reason anyone is in the room.

Opus took the programme into Phase 3 at the same low dose, and did not open a higher-dose arm. Natural-history and treatment cohorts are full. Dosing is expected in the fourth quarter of 2026. The primary endpoint is retinal sensitivity at 16 central test locations. Topline six-month data are due by the end of 2027. The US Food and Drug Administration has said a licence application may be filed on the six-month number, with 12-month durability sent in while the file is open. The company's own filing says the figures are interim and may not predict the Phase 3 result.

Key Facts

• Presented 15 October 2026 at Retina in Rio by Opus Genetics
• Low dose: 1 times 10 to the 10 vector genomes per eye, one-time subretinal AAV8
• Three adults through 24 months, three adolescents through six months
• Trial NCT05616793
• Untreated eye used as the comparison on full-field stimulus testing
• Phase 3 cohorts enrolled; dosing expected in the fourth quarter of 2026
• Company estimate of the patient population: about 200

Why This Matters

Ultra-rare eye disease is where a gene therapy can be a real change for a countable group of people, and where a press cycle can outrun the evidence. Six patients and an untreated-eye comparison is the honest size of this result.

What We Don't Know Yet

Whether the Phase 3 dose, not yet given, reproduces the gain. What the acuity change was in letters or lines. The company release says improvements and does not print the chart numbers in the copy used here. Durability past 24 months in adults, and past six months in the adolescents, is not in this presentation.

Sources: Opus Genetics 8-K · BioPharm International