Abstract

Using Pluripotent Stem Cells in the Age of Genome Editing

Herbert Mueller-Hartmann, Head of Global Technology Development, Lonza Bioscience

In light of the decline of drug candidates in Pharma R&D output, there is an increasing demand for cell-based in vitro-systems that provide a better model for the physiological situation in vivo. The hope is that new cell-based models will better reflect physiological disease mechanisms, which will lead the way to identifying new types of pharmaceuticals. The challenge for advanced cell-based models is that they need to be robust, scalable, and compatible with assays that generate reproducible data. Induced pluripotent stem cells (iPSCs) represent one solution to the challenge of robustness, in that they provide a means of generating large numbers of theoretically homogeneous differentiated cells for use in cell-based assays. New strategies for genetic engineering of these cells -- CRISPR/Cas9 genome editing for example -- are helping advance the capability of generating cells with specific disease features. Additionally, 3D cell culture methods promise another level of biological relevance for cell-based models. Finding the right strategy to incorporate new technologies that enable higher-value in vitro-models will be important for advancing drug discovery and pre-clinical development.