Stem Cells in Drug Discovery Agenda

Registration

Stem Cells In Drug Discovery – Progress and Challenges
Devyn Smith, Chief Operating Officer, Sigilon, United States of America

How can stem cells enable drug discovery?

QC and Assay Development for Product Safety Testing Using Pluripotent Stem Cell Based Systems
Glyn Stacey, Principal Scientist and Head of Cell Biology, National Institute for Biological Standards and Control (NIBSC), United Kingdom

The development of assays for product safety testing based on human pluripotent stem cell lines requires careful evaluation of the starting cell line and qualification of the protocol to deliver the final cells for testing. In the is presentation will consider experiences in addressing these issues in the Seurat-1 EC/Colipa funded programme and new initiatives in the development of best practice for stem cell culture and assay development.

Coffee & Networking in Exhibition Hall

Oncolytic Targets Identified by Genetically Engineered hNSCs in a Rodent Model of Spinal Cord Glioma
Yang Teng, Associate Professor, Harvard Medical School, United States of America

We have investigated the oncolytic effect of genetically engineered hNSCs in a newly validated rodent model of intramedullary spinal cord gliomas (ISCG). Our findings demonstrate a stem cell-based multimodal approach to developing targeted glioma treatments following recovery neurobiology principles.

Collaborative Phenotyping at King’s College London: Hipsci and the Stem Cell Hotel
Davide Danovi, Director, Cell Phenotyping Platform, Kings College London, United Kingdom

- Using relevant cells for high content analysis
- HipSci: diverse constituents in genomics, proteomics, cell biology and clinical genetics for a national iPS cell resource to carry out cellular genetic studies.
- The Stem Cell Hotel: building a dedicated space for collaborative phenotyping within the premises of the Centre for Stem Cells & Regenerative Medicine at KCL.

Discovery of Regenerative Drugs That Modulate Endogenous Progenitor Cells
Jey Jeyakumar, Principal Scientist & Group Leader, Plasticell & Progenitor Therapeutics, United Kingdom

An overview of the applications of two validated technologies CombiCult® and ProScreen™ on the discovery of small molecule regenerative drugs for osteoarthritis, multiple sclerosis and muscular dystrophy - an open innovation partnership with GSK, will be presented.

Lunch & Networking in Exhibition Hall

Poster Viewing Session

Technology Spotlight:
Human iPSC-derived Tissues as Novel Translational Models for Drug Discovery and Screening
Mark Hewitson, Account Manager, Cellular Dynamics Intl

Human cell types differentiated from induced pluripotent stem cells (iPSCs) offer an attractive source of cellular material for both toxicity screening and drug discovery because of the biologically relevant systems they can represent in vitro. The iPSC technology is a key element for modeling human biology in a dish, which is otherwise difficult to explore using conventional cell lines, primary cells, or animal models. Our approach is to generate iPSC-derived cell types with high quality, purity, and unlimited quantities, design relevant assays with cells derived from apparently healthy donors, and develop disease models using environmental stimuli or disease-specific, patient-derived cells. Current efforts are aimed at generating large iPSC clone banks from diverse diseased backgrounds that will serve to dramatically expand access to cell models offering new tools and opportunities for phenotypic drug discovery.

High Content Screening With hiPSC-Derived Neurons
Amanda Cobos-Correa, Lab Head Screening Sciences, Novartis Institutes for Biomedical Research, Switzerland

In my talk I will present the large-scale generation of neuronal progenitors derived from Fragile X patients and their use to develop a high-content imaging assay to run a HTS to identify compounds counteracting the epigenetic gene silencing in Fragile X syndrome.

Neuromesodermal Progenitors: Tracing the Origins of Neuromuscular Diseases
Mina Gouti, Group Leader, Max-Delbrück Center for Molecular Medicine, Germany

Generation of spinal cord neurons and muscles from pluripotent stem cells with precise positional identity is crucial for neuromuscular disease modeling. I will focus on the in vitro generation of a neuromesodermal (NMP) progenitor population from pluripotent stem cells, an advancement that gives unprecedented access to the development of new models to study neuromuscular diseases.

Coffee & Networking in Exhibition Hall

Medicinal Chemistry In Regenerative Medicine: Discovery Of In Vivo-Active Small Molecule Modulators Of Endogenous Stem Cells
Angela Russell, Professor, University of Oxford, United Kingdom

The talk will give a brief overview of the area, outline how my group became involved in the field and cover contributions we have made, including the expansion of pluripotent and multipotent stem cells and how we are aiming to translate our work to the clinic for regenerative therapies. As a representative example the discovery process for new small molecule modulators to induce the differentiation of neural stem cells both in vitro and directly in vivo following systemic administration will be described.

Modelling Human Gastrulation: A Platform for Directing Pluripotent Stem Cell Differentiation to Clinically Relevant Cells
Roger Pedersen, Professor, University of Cambridge, United Kingdom

I will discuss how lineage decisions of human stem cells model gastrulation and thereby achieve growth factor-induced differentiation to specific cell types; how transcription factor-based forward programming provides an alternative strategy for differentiation of clinically relevant cells; and how interspecies post-implantation chimeras can be used to functionally validate human pluripotent stem cells for use in regenerative medicine.

Cancelled - 3D Human Liver Models Generated from iPSCs for Disease Modelling and Therapy
Tamir Rashid, MRC Clinician Scientist Fellow, King's College London, United Kingdom

Drinks Reception in Exhibition Hall

Conference Dinner in Hinxton Hall

End of Day 1

Investigation of Neurodegenerative Disease Utilizing Patient-Specific iPS cells
Keiko Muguruma, Research Specialist, RIKEN Center for Developmental Biology (CDB), Japan

We have developed three-dimensional culture systems for cerebellum and cerebral cortex using human pluripotent stem cells. We are investigating the pathogenesis of the intractable neurological diseases using in vitro disease models utilizing the patient-derived iPS cells.

Generation and Characterization of Patient-Specific iPSC Lines and Gene Editing of hESCs and iPSCs
Emily Titus, Director, Technology Development, Centre for Commercialization of Regenerative Medicine (CCRM), Canada

CCRM has developed standardized methods for reprogramming and gene editing. I will focus on the technical challenges and associated solutions implemented at CCRM to establish these workflows.

Coffee & Networking in Exhibition Hall

Making Adult-like Cardiomyocytes from iPSC Cells and Modeling Cardiomyopathy
Chulan Kwon, Associate Professor, Johns Hopkins University, United States of America

Target Validation Platforms for Inflammatory Disorders and Chronic Kidney Disease – the Power of Stem Cells and Precise Genome Editing
Ryan Hicks, Associate Director, AstraZeneca, United Kingdom

AstraZeneca applies advanced technologies for generation and validation of iPS cell derived models for drug discovery. Data from two cellular-platforms are presented, where we have used these tools to monitor cell-model development and compare to existing primary cell models.

Nanostructured Foams in Cell Guidance
Jens Vinge Nygaard, Head of Section & Associate Professor, Aarhus University, Denmark

Lunch & Networking in Exhibition Hall

Poster Viewing Session

Technology Spotlight:
Detection and Quantification of CRISPR Generated Genome Edits in The Production of Cell Line Models
Stephen Hague, Droplet Digital PCR Specialist Europe, Bio-Rad Laboratories Inc

Cancer Stem Cells and Hepatocytes for Drug Screening
Annamaria Lilienkampf, Research and Teaching Fellow, Edinburgh University , United Kingdom

In my talk I will introduce polymer microarray technology and describe how this approach has been used in a large number of stem cell based applications, notably polymer discovery able to support highly functional hESC-derived hepatocyte like cells and the discovery of a substrate, able to enrich CSC's.

Mapping Functional Effects of Common Genetic Variants in IPS Derived Immune Cells
Daniel Gaffney, Group Leader, Wellcome Trust Sanger Institute, United Kingdom

I will present the latest results from our work on the Human Induced Pluripotent Stem Cell Initative (www.hipsci.org), which aims to generate large numbers of IPSCs from healthy individuals and disease volunteers. I will also discuss some of our work using HIPSCI lines to study a range of cell lineage.

Coffee & Networking in Exhibition Hall

Cancelled - 3D Environments to Model Cell-Matrix Interactions Important in Development and Disease
Cathy Merry, Associate Professor of Stem Cell Glycobiology, University of Nottingham, United Kingdom

We have developed methods for functionalisation of biomaterials to display selected glycosaminoglycans in 3D environments suitable for the culture and directed differentiation of stem cells.

hiPS Cardiomyocytes in Drug Safety: Ca2+ Assay Optimization and Experimental Variability
Ivan Kopljar, Post Doctorial Research Scientist, The Janssen Pharmaceutical Companies of Johnson & Johnson, United States of America

Human stem cell-derived cardiomyocytes (hiPS-CMs) are increasingly used for screening of cardiac safety liabilities within the pharmaceutical industry. Optimization of the Ca2+ transient assay together with in-depth characterization of hiPS-CMs is essential to evaluate and improve cardiac risk assessment within drug development.

Close of Conference