Saving vision: expanding use of lentiviral gene therapy to fight blindness

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Sight is the most treasured of our senses, followed by hearing and balance. Moderate to severe vision loss affects more than 216 million people worldwide, with an estimated 36 million people suffering from blindness. Monogenic diseases caused by mutations in genes such as MYO7A are responsible for vision loss in over two million people. As current treatment options for genetic vision loss are limited or non-existent, there is a high clinical need for innovative approaches, such as gene therapy. Usher syndrome type 1B (USH1B) is caused by MYO7A mutations and patients suffer from profound deafness accompanied with loss of balance and vision. In my ERC-funded consolidator grant iHEAR and Proof-of-Concept grant MY(O)SENSES, we showed the first ground-breaking data for efficient gene transfer with lentiviral vectors into inner ear cell types and correction of hearing and balance defects caused by mutated MYO7A. The EuroVision PoC proposal aims to advance these valuable research results towards clinical application by completing pre-clinical development, securing IP rights and commercializing a formulated medicinal product to launch a first-in-human clinical LV gene therapy trial to treat USH1B patients. The EuroVision product will be the first and only single-vector treatment that addresses vision loss due to MYO7A mutations in USH1B patients, which has great potential to significantly improve quality of life for these patients as well as to benefit society through relief of costs associated with healthcare and lost productivity. Moreover, this ground-breaking therapy will lay the basis to address other monogenic eye diseases, in which large genes need to be transferred and for which there is an urgent medical need.