StemBANCC, comprising internationally renowned investigators, is ideally positioned to deliver a unique European biorepository and drug discovery pipeline based upon human induced pluripotent stem cells (hiPSC). This unrivalled opportunity will address the pressing therapeutic need in neurodegeneration, neurodysfunction and diabetes. We have immediate access to richly phenotyped subjects with a wealth of clinical data. Furthermore our cohorts include genetic and genome data, allowing us to carefully select subjects for hiPSC based disease model development. This clinical-genetic stratification is essential to ensure that cellular models display disease and therapy relevant phenotypes. hiPSC reprogramming is undertaken by an internationally respected centre, providing robust, reliable and timely delivery of hiPSCs; ensures the biorepository only houses quality assured hiPSC lines; and reduces unwanted variability in assays. We have leading experts in hiPSC differentiation, providing protocols with over 99% efficiency in producing cortical excitatory neurons and cardiomyocytes, which following up-scaling will meet demands of high throughput assays. StemBANCC will deliver a comprehensive ‘omics’ dataset on over 1500 stem cell lines. This includes cells taken at different lineage stages, after therapeutic challenge, monogenic and complex variants, and with matched isogenic controls. The integration of this network/pathway data will define core etiopathological processes, therapeutic generality, reveal on- and off-target effects, allow candidate therapeutic repositioning and decouple tolerance and toxicity pathways. The large-scale integrated datasets then directly guide the disease specific targeted assays, designed to maximize utility in low and high- through compound screening programs. Assay development undertaken by the StemBANCC proposed central test facility thereby provides the final component of our hiPSC platform for drug discovery, development and safety.
Call for proposal
See other projects for this call
Funding SchemeJTI-CP-IMI - Joint Technology Initiatives - Collaborative Project (IMI)