UshTher Final Meeting

Usher syndrome (USH) is the most common combination of deafness and blindness due to retinitis pigmentosa. USHIB, caused by mutations in the large MYO7A gene, is among the most severe and frequent forms of USH. While deafness can be improved with cochlear implants, blindness remains untreatable. The overall goal of UshTher is to develop a phase I/II, first-in-human, clinical trial of gene therapy for USHIB retinitis pigmentosa based on dual AAV. It would be the first time the dual AAV vector approach is tested in humans. Towards this ambitious objective, UshTher has assembled a very competitive consortium with leaders in the fields of retinal gene therapy from bench to bedside including SMEs with expertise in the development of gene therapy products.