Plain-English translation of NCT07185256 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing OPGx-BEST1, a one-time gene therapy treatment injected directly into the back of the eye for people with two rare inherited eye diseases: Best vitelliform macular dystrophy (BVMD) or autosomal-recessive bestrophinopathy (ARB). Both conditions are caused by mutations in the BEST1 gene and lead to progressive vision loss. The medication works by delivering a healthy copy of the BEST1 gene to the cells in the eye that are affected by the disease.
Currently, there are no approved treatments for BVMD or autosomal-recessive bestrophinopathy, and people with these conditions face progressive vision loss with limited options. This trial is exploring whether this new gene therapy can be safely delivered to the eye and may help preserve or improve vision in patients with these genetic disorders.
You likely qualify if…
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If you qualify, you will have the gene therapy injected into one eye during a surgical procedure on Day 1. After surgery, you will return for follow-up visits on Days 2, 7, 14, 30, and 45, then again at 3 months, 6 months, and yearly for 5 years. At each visit, your eye doctor will examine your eye and test your vision to see how the treatment is working and to monitor for any side effects.
AI-generated summary from trial data · Jun 6, 2026 · Not medical advice
United States