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Cerebral organoids: human mini brains in a dish open up new possibilities for drug development in neurodegenerative and developmental diseases

Periodic Reporting for period 1 - Mini Brains (Cerebral organoids: human mini brains in a dish open up new possibilities for drug development in neurodegenerative and developmental diseases)

Periodo di rendicontazione: 2017-01-01 al 2018-06-30

Neurological disorders are a leading cause of disability, affecting millions of patients worldwide and constituting a disease burden that amounts to economic costs of almost 800 million Euro in Europe. Of all organs, the human brain differs most from that of common animal models, establishing specific challenges for disease modelling and compound screening. Therefore, the intrinsic complexity of brain physiology and the general lack of relevant disease models in vitro and in vivo have made it difficult to study brain disorders and constitute the bottleneck of drug discovery and development in neuroscience. The goal of this project is to evaluate the possibility of modelling human neurological disorders in cutting-edge 3D cell culture models called cerebral organoids that are derived from human patients for the analysis of disease mechanisms and compound testing. During the course of the project, we have generated organoid models for neuro-developmental disorders and tested their disease-specific phenotypes. We have identified characteristic features for several models of epilepsy that are now being tested for suitability for compound testing and high-throughput screening. As an important product from this project, we have established a business plan for commercial exploitation of the cerebral organoid model. Detailed analysis of the market situation and the competitive landscape in the field have revealed enormous opportunities for the commercialization of the 3D culture model. They have resulted in the identification of a commercial partner who will collaborate in the establishment of a commercial entity for organoid disease modelling. We expect that the work completed under this grant will pave the way for the establishment of a commercial entity during the next year.