AAVantgarde Bio has completed enrollment in LUCE-1, a phase 1/2 clinical trial evaluating its investigational gene therapy AAVB-081 for retinitis pigmentosa associated with Usher syndrome type 1B. The multicenter, open-label study enrolled 15 adults aged 18 to 60 years and is designed to assess safety, tolerability, and preliminary efficacy following a single subretinal administration.

AAVB-081 uses a dual–adeno-associated virus serotype 8 (AAV8) platform to deliver the MYO7A gene, which is too large to fit into a single standard AAV vector. The approach relies on 2 vectors, each carrying half of the gene, which recombine in the cell nucleus to generate functional protein. MYO7A mutations are responsible for Usher 1B, a condition characterized by congenital deafness, vestibular dysfunction, and progressive vision loss beginning in childhood.

Investigators described the completion of dosing as a key step toward generating early clinical data. Usher 1B currently has no approved treatments for retinal degeneration, although surgical options exist for hearing loss.

AAVantgarde is also recruiting patients for its CELESTE trial, which is evaluating a separate dual-AAV gene therapy, AAVB-039, for ABCA4-associated Stargardt disease. The company has indicated that updates from both programs are expected in 2026. RP