AAVantgarde completes enrollment for dual-AAV gene therapy rare eye disease trial

AAVantgarde Bio announced the completion of enrollment in its phase 1/2 first-in-human clinical trial evaluating the company’s dual-AAV gene therapy, AAVB-081, for the treatment of retinitis pigmentosa associated with Usher syndrome type 1B (USH1B).

Usher1B, caused by mutations in the MYO7A gene, is an inherited disease that affects the retina and the inner ear. Children with Usher1B are born deaf, have vestibular dysfunction, and begin to progressively lose vision in their first decade of life.

AAVantgarde’s LUCE-1 trial is a multicenter, open-label, dose-escalation and expansion study evaluating a single subretinal administration of AAVB-081, which targets mutations in the MYO7A gene, addressing the root cause of USH1B. The study enrolled 15 adult participants between 18 to 60 years of age.

AAVantgarde is testing a second therapy, AAVB-039, an intra-retinal AAV8-based dual AAV intein product, in the CELESTE study for Stargardt disease — another debilitating inherited retinal condition with no approved treatments. The company says it will have updates for both programs in 2026.