Advancing cell-targeted and immune-resistant gene therapy for frontotemporal dementia and motor neuron disease

Frontotemporal dementia and Motor neuron disease, both devastating neurodegenerative disorders, stand as formidable challenges in modern healthcare. These conditions relentlessly erode the quality of life for those affected and present significant burdens for families and healthcare systems alike. The total cost of FTD and MND is predicted to hit $25.8 billion by 2036 and $36.8 billion by 2056 globally. Despite these staggering costs, there is no cure for FTD and MND with available therapies aimed at symptom management. This project proposes to transform an experimental gene therapy into a clinical trial-ready therapy for FTD and MND patients.