10069789Development of a Novel Gene Therapy for Neurodegenerative DiseasesClosedCollaborative R&DInnovate UKAmyotrophic Lateral Sclerosis (ALS) is a fatal motor neuron disease that causes death in patients within 3-5 years of diagnosis. Riluzole is the only approved drug in the UK that extends survival by 6 months. Increasing research in the pathophysiology of ALS has led to a deeper understanding of molecular mechanisms underlying the disease progression. Recently several exciting targets have been identified. Many of these targets are not druggable using the conventional small molecule and monoclonal antibody-based approach. RNA targeting gene therapy has emerged as a promising modality, extending druggable targets for the treatment of ALS. Drishti Discoveries is an Advanced Therapy company developing disease-modifying gene therapy for rare diseases using a proprietary miRNA-adapted shRNA technology. We are applying this RNA-targeting technology to develop a long-term treatment for ALS. Our data so far show we can significantly silence our gene of interest and rescue motor functions in an acute ALS mouse model. In this project, we aim to further progress the programme. We will confirm the efficacy of our candidates in an independent ALS mouse model to bring our innovative gene therapy product one step closer to the clinic for patients in urgent need.