The overall objective of the action generated from this topic is to establish a fully self-sustainable European human iPSC banking facility, that has to be operational within the first three months of the action by seamlessly building on and incorporating existing cell lines, knowledge and infrastructure established within former European-wide initiatives (e.g. EBiSC). The bank has to be able from the start to handle and deliver a minimum of approximately 500 quality-controlled, disease-relevant (in particular for neurodegeneration, Alzheimer’s disease and other tauopathies, Parkinson’s disease, cardiovascular disease, safety, diabetes, and auto-immune and selected monogenic diseases), research-grade iPSC lines, with integrated data and cell services which will be further built on as part of the research and technology work of the action. The ultimate goal is to transform significant pre-existing European banking infrastructures into a sustainable resource for European research and development.
In Europe, the EBiSC project (https://www.ebisc.org/) funded by the Innovative Medicines Initiative Joint Undertaking (IMI JU) has demonstrated the feasibility and challenges of coordinating existing organisational capacities across Europe to fast track the establishment of a centralised network and facilities to access a standardised resource of established human inducible pluripotent stem cells (hiPSC) lines and data. EBiSC has established a unique European-based iPSC repository and has delivered harmonised and publically accessible Standard Operations Procedures (SOPs) for tissue procurement, bio-sample tracking, iPSC expansion, cryopreservation, qualification and distribution to the research community. These were implemented to create a quality managed foundational collection of lines and associated data made available for distribution.
The critical challenge addressed by this topic is to build on these important infrastructure, capabilities and knowledge to create a fully sustainable European hiPSCs distribution platform with worldwide reach.
Availability of iPSC lines derived from patients, as well as of a broad spectrum of lines from healthy donors of different ages, standardised according to how they were made and their in vitro behaviour, and the possibility of linking a gene code to cell line phenotype reflective of the disease, will enable the research community to refine original clinical diagnosis into one based on disease stratification and thereby design more precise experiments to discover novel pathogenic pathways, drug targets and new medicines. This is expected to significantly advance research and development activities across Europe by accelerating the progress of understanding certain disease aetiologies, as well as finding potential cures, thereby strengthening European competitiveness.
This European iPSC repository will be uniquely positioned to serve as the central European iPSC repository hub to accelerate and facilitate European research and development activities. Therefore, the consortium will have to continuously monitor the sale of cells produced by the banking entity, and its trend in order to develop in the runtime of the project a plan as to how to transform the repository into a self-sustainable business. Ultimately this will secure that the public and private investment will establish a resource that beyond the runtime of the project continues to support and fuel European basic research as well as drug development campaigns in pharma companies.