Added value to drug discovery via human iPSC disease models
Despite significant financial investments and technological advances in the past decades, the output of approved drugs has been relatively inefficient. The lack of physiologically relevant and predictive cell-based assays is one of the major obstacles. The use of recombinant cell lines or animal models does not fully recapitulate the complexity of human disease. In addition, primary human tissues are typically difficult to obtain and not available in the large quantities required for medium- or high-throughput screening.
Human induced pluripotent stem cell (hiPSC)-based disease models are advantageous platforms thanks to their functional relevance and scalability. The pharmaceutical industry is now broadly recognizing the benefits of applying hiPSC biology in the drug discovery pipeline, and the use of this technology is growing.
Nevertheless, implementation of hiPSC-derived disease models in drug discovery projects involves several critical considerations. The first critical step is lineage specification of hiPSCs to cell types that closely recapitulate key disease features. Secondly, cell manufacturing at a commercial scale must be achieved. Thirdly, reproducible and high-throughput assays that measure the disease biology must be developed.
During this webinar we discuss the advantages, challenges and solutions of implementing hiPSC-technology into drug discovery and development - addressing disease modelling, large-scale manufacturing, assay development and phenotypic screening.
Presented by
Arie Reijerkerk, PhD,
Director Manufacturing Technology
Arie joined Ncardia in 2015 and together with an expert project team he has been instrumental in the establishment of Ncardia’s platform for process development and large-scale manufacturing. Earlier in his career, he obtained a PhD in Internal Medicine, and spend more than 10 years in basic and applied cardiovascular research in academia. Before joining Ncardia, he was responsible for the early pre-clinical development of drugs passing vascular barriers in the brain at the clinical-stage company to-BBB technologies BV. As Director Manufacturing Technology he currently supervises multiple project teams with the goal of delivering custom manufacturing and process development services and coordinates scientists and business developers in client project acquisition and setting up partnerships. Recently, he managed the successful technology transfer of Ncardia’s bioreactor-based large-scale manufacturing process for cardiomyocytes to a pharmaceutical partner.
Elena Matsa, PhD,
Director Discovery Technology
Elena is the Director of Discovery Technology at Ncardia. She obtained her PhD in stem cell biology in 2010, and subsequently worked as a post-doctoral researcher at the University of Nottingham, and the Stanford University School of Medicine. She has extensive experience and high impact publications in modelling of human cardiac disease in iPSC-derived cardiomyocytes. Currently, Elena supervises the activities of Ncardia’s Discovery Technology Department, which runs disease modeling, and drug discovery and safety assessment projects in oncology and cardiovascular, skeletal, metabolic and neural disease areas.
