EBiSC: The European Bank for induced pluripotent Stem Cells

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1 EBiSC: The European Bank for induced pluripotent Stem Cells Sadallah Fatiha Japan Health Sciences Foundation Visit IMI JU Office, Brussels, Belgium

2 EBiSC Establish a European repository for research grade human induced pluripotent stem cells (ipsc) at unprecedented scale A repository to be run on a self sustaining, not for profit basis and on terms of open access for all qualified users A repository to catalyse and support collaboration & propel the ERA to the forefront of applied human disease-related ips research

3 What is the EBiSC project? A 35 million, IMI funded project through which 26 leading European organisations will establish a central facility for the collection, testing and distribution of ips cells to researchers.

4 Who is in the consortium? 6 EFPIA members 6 SMEs 8 Universities 5 public agencies 1 charity funded institute 9 countries Large enough to have a structuring effect on the EU Research Landscape Manageable as a multi-centre consortium

5 What is the EBiSC project? Problem Statement ipsc lines are being produced in a huge number of separate research projects and hold great potential for developing a personalized However this is hampered by variable quality and consistency of their production, lack of access ease and incomplete clinical and genotype/phenotype accompanying data Planned Solution EBiSC will establish a central, not for profit EU facility for the standardized collection, testing and distribution of a large number of diverse research grade ips cell lines EBiSC Copyright EBiSC Consortium 5

6 Why create EBiSC? With EBiSC : better use of research assets Research projects creating ipscs EBiSC Other researchers provide samples of ipsc lines to EBiSC Creates distribution stocks & ensures quality get ipscs of known quality, faster & at less cost

7 What will EBiSC do? EBiSC : improving the research landscape in Europe Research projects creating ipscs EBiSC Other researchers Consent forms & contracts which meet needs of all stakeholders Common standards for processing and testing cell lines Establish central facilities which use best cell culture technologies to operate at scale Data management system which provides extensive data to users but controls access Create a catalogue of cell lines which meet user needs

8 What does the catalogue look like currently? Gene editing workstream Build control lines for disease modelling Osteoporosis-pseudoglioma syndrome Musculoskeletal Neuro- Degenerative psychiatric Alzheimer s disease Parkinson s disease Autism spectrum disorder Creatine transporter deficiency Fronto-temporal dementia Spinal muscular atrophy Machado Joseph (ataxia) Hereditary spastic parapelegia Primary erythromelalgia Retinitis pigmentosa Acquired aplastic Anemia Fanconi anemia Epidermolysis bullosa Blood Auto immunity Cardiovascular Brugada syndrome Long QT syndrome Catecholaminergic polymorphic Ventricular tachycardia (CPVT) Type 2 diabetes MODY Obesity Gaucher disease Metabolic Minor genetic variants Polymorphisms implicated from GWAS Understanding normal variation underpinning disease susceptibility & mechanism

9 What should the catalogue look like towards end of 2016? Clinic ipsc specialist Researcher Volunteer donates tissue Derive ipsc lines from tissue Replication creates large numbers of cells Differentiatio n creates cells of specific types Physiological cells used in research 250 de novo lines/gene edited derivatives within project 87 FP7 projects as potential users 32 FP7 projects as potential suppliers 21 national/international funded projects with EBiSC partners 3 IMI funded projects (StemBANCC, EU-AIMS, MIP-DILI) 2,500 distinct patient derived/disease model lines induced pluripotent stem cell

10 What is EBISC likely to achieve? Hot Start lines from 7 ipsc centres Sanger HIPSCI lines New EBiSC Commissioned ipsc lines ipsc lines from other EU funded projects eg FP7, H2020, IMI First cells shipped to researchers from EBiSC Standard contracts Main central facility operational Standard Quality Control testing programme Extended web portal operational Harmonised production protocols v1 Foundational collection EBiSC ipsc catalogue Beyond 2016: EBiSC will continue to expand its catalogue to meet user demand leading to a self financing operation by And beyond Europe Engage with similar major ipsc banking and production initiatives

11 How will EU stakeholders benefit? A common European approach for ipsc based research Establishing a central resource will define and disseminate best practice for ipsc based research to tissue donors, their clinicians, research funders, patients etc as well as to researchers strengthen profile of current European practice for ipsc based research provide a focal point for academics and SME s for technology innovation enable faster more cost effective research. Haplobank Mechanism of disease Stratifying Patients Pre-clinical efficacy models Predicting off target effects

12 Thank you Fatiha Sadallah Principal Scientific Manager