Therapeutic area: CNS, neurodegenerative disorders
Disease: Parkinson’s Disease
Monogenic inroad: Alpha Synuclein (SNCA) and LRRK2 (G2019S)
Cellular Model: Patient-derived cell lines
Project Origin: University of Luxembourg, Prof. Rejko Krüger
Medical need: Parkinson’s disease (PD) is the second most common neurodegenerative disorder currently affecting between 7-10 million people worldwide. The annual cost of PD in Europe is in excess of 14 billion euros (Olesen et al, Eur J Neurol, 2012). SNCA mutations are associated with autosomal dominant forms of PD. SNCA is deemed as one of the major causative genes in different neurodegenerative disorders.
Goal: The program aims for development and implementation of physiologically highly-relevant cellular disease models (assays). Assay development is based on human differentiated induced pluripotent stem cells (iPSC), and tailored AI-based image analysis methodologies. The assays are rendered applicable for HTS-HCS, and will be used for the development of disease modifying treatments.
Development stage: The assays have been validated through a set of compounds and are currently automated.