AAVantgarde reveals safety data from groundbreaking study on Usher Syndrome type 1B

On December 9, 2024, AAVantgarde Bio, a biotechnology company based in Italy, revealed important safety data regarding the LUCE-1 trial. This trial is a Phase 1/2 open-label study designed to assess the safety and tolerability of a novel gene therapy, AAVB-081, aimed at treating retinitis pigmentosa related to Usher Syndrome Type 1B (USH1B). The trial is being conducted under the guidance of Professor Francesca Simonelli at the University Hospital of Campania 'Luigi Vanvitelli' in Naples. The LUCE-1 study primarily focuses on the effects of AAVB-081, developed using AAVantgarde's proprietary dual hybrid gene therapy platform, which delivers the MYO7A protein in affected individuals. Preliminary safety data was presented for two subjects with low visual acuity participating in the low dose cohort of the trial. The best corrected visual acuity (BCVA) of both subjects returned to their pre-treatment levels within two weeks of receiving the treatment. It was noted that all observed adverse events (AEs) were mild, with no serious complications that required further intervention. Importantly, the trial has not yet revealed any dose-limiting toxicities, which is a promising indication for the ongoing research. Professor Simonelli expressed her satisfaction with being the Principal Investigator for this groundbreaking Phase 1/2 clinical trial, noting that the preliminary safety results are encouraging as the study moves towards dose escalation. Dr. Natalia Misciattelli, the CEO of AAVantgarde, also highlighted the significance of this research, emphasizing that the study holds the potential to provide therapeutic options for patients with USH1B, a condition for which there are currently no treatments available to counter progressive vision loss. The LUCE-1 trial, identified by the clinical trial registry number NCT06591793, involves a multicenter approach to evaluate the safety, tolerability, and preliminary efficacy of different doses of the AAV8.MYO7A product. Parents or guardians of patients with Usher Syndrome Type 1B face challenges as these children are typically born deaf and experience vestibular dysfunction, leading to progressive vision loss within their early years. With surgical interventions available for their hearing impairment, many families are left without solutions for the visual degeneration associated with the disease. AAVantgarde's innovative approach aims to address this unmet need in the field of inherited retinal diseases, providing hope to these affected families. As AAVantgarde continues to explore the capabilities of its dual AAV platform technology, there is optimism that the results from the LUCE-1 trial will contribute valuable insights into gene therapies for retinitis pigmentosa related to Usher Syndrome, ultimately paving the way for future treatment options. As research progresses, the anticipation builds for more comprehensive data regarding the efficacy and long-term safety of AAVB-081 to emerge in subsequent study phases.