A Phase 1/2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Subjects with Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa

A single injection of a gene therapy into the back of the eye aims to restore the function of a faulty gene in people with Usher syndrome type 1B, a condition that causes both deafness and progressive blindness. Usher syndrome type 1B is caused by mutations in the MYO7A gene, which produces a protein essential for the health of light-sensing cells in the retina. Without it, these cells gradually die, leading to vision loss that typically begins in childhood. Current treatments only manage symptoms; none address the underlying genetic cause. This trial tests whether delivering a working copy of MYO7A directly to the retina—via a harmless virus called AAV—can slow or halt that degeneration. If the therapy proves safe and shows signs of efficacy, it would be the first treatment to target the root cause of vision loss in USH1B. Up to 15 participants will receive one of three escalating doses, with the goal of identifying a dose that offers a favourable balance of safety and benefit. Success would not restore hearing, but it could preserve sight—a direct, tangible improvement in quality of life for people who otherwise face progressive blindness.