AAVantgarde Bio presented updated results from its LUCE-1 trial of the investigational gene therapy AAVB-081 for Usher syndrome type 1B at the EURETINA 2025 congress in Paris. The data showed the treatment has been well tolerated to date and is beginning to demonstrate potential visual benefit.

The presentation by Francesca Simonelli, MD, of the University Hospital of Campania in Naples, Italy, included safety results from 11 participants across low-, medium-, and high-dose cohorts. Among the first 4 patients with at least 180 days of follow-up, no drug-related serious adverse events or dose-limiting toxicities have been observed. Episodes of ocular inflammation were rare and reversible with steroids.

Visual outcomes were notable. All 4 patients achieved more than 1 line of improvement in best-corrected visual acuity, and 2 gained more than 3 lines in low-luminance visual acuity. Microperimetry fixation stability improved in 3 of the 4 patients. One participant has been followed for a year with sustained benefit.

LUCE-1 is a phase 1/2 open-label, dose-escalation study evaluating AAVB-081, a dual AAV vector designed to deliver the full MYO7A gene. Dr. Simonelli emphasized that continued follow-up will be needed to confirm durability and efficacy. Usher 1B affects about 20,000 patients in the United States and Europe, and currently has no approved treatments for progressive vision loss.