AAVantgarde has unveiled the design of its innovative LUCE-1 trial for Usher Syndrome Type 1B at the 9th Annual Retinal Cell and Gene Therapy Innovation Summit. This marks a pivotal moment for the field of retinal gene therapy. The trial, led by Francesca Simonelli at the Ophthalmology Clinic of the University of Campania “Luigi Vanvitelli,” has already dosed its first patient with AAVB-081.

The study utilizes the groundbreaking “dual AAV” platform developed by Alberto Auricchio and his team at Tigem, which allows for the transfer of large genes crucial for treating complex genetic conditions. This first-in-human (FIH) Phase 1/2 trial for retinitis pigmentosa associated with USH1B is a significant step toward addressing the unmet needs of patients affected by blindness and hearing loss.