Drug discovery for Parkinson’s disease using a translational screening platform

Parkinson’s disease (PD) is the most common neurodegenerative motor disease and is characterised by the aggregation of alpha-synuclein (α-syn) in the brain. In this project, we develop and validate a translational screening platform for α-syn aggregation inhibitors for PD. The primary assay of the platform is a phenotypic assay based on α-syn aggregate induction in neuroglioma cells using recombinant α-syn fibrils. The assay has already been validated in a 384 well format on the automated screening platform of CD3. We will extend the platform with our in-house developed iPSC-based secondary assays and our in-house in vivo PD rodent model. The platform will be stress-tested using reference and repurposing compounds. Secondly, we will use this platform in a high-throughput screening campaign (collaboration with CD3) to identify at least one candidate series eligible for lead generation.

Keywords:drug discovery, Parkinson's disease, iPSC, alpha-synuclein

Disciplines:Neurological and neuromuscular diseases