First Patients Dosed in Gene Therapy Trial for USH1B

Tuesday, January 14 2025 | 14 h 11 min | News

AAVantgarde Bio, an Italy-based biotechnology company announced that the first patient has been dosed in their Phase ½ clinical trial. This trial will test the safety of a new gene therapy (AAVB-081) for Usher Syndrome Type.

This trial is the first to test what is called a dual-vector gene delivery system. In gene therapy, the delivered gene is “packaged” in a viral vector. MYO7A is too big to fit into most vectors, so in this case the gene is split into two, with each half delivered in a separate vector. Once in the cell the two halves of the gene join to form one functional gene.