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Human iPSC-derived functional cells are revolutionising phenotypic drug discovery and development

Effective drug discovery and development relies largely on the availability of predictive pre-clinical model systems; the absence of which has contributed to late-stage drug failures and high expenditures. New industry acceptance of human induced pluripotent stem cell (hiPSC)-derived cardiomyocyte assay systems for regulatory safety pharmacology is now resulting in broad use of the technology in drug discovery and development. Here, we demonstrate an end-to-end solution covering large-scale manufacturing of hiPSC-derived cells enabling phenotypic high-throughput screening (HTS) in a relevant human cellular disease model.

phenotypic

Despite many technological developments over the past decades, the output of commercial drug discovery and development has been relatively ineffective; which has been referred to as the productivity crisis in pharmaceutical industry by many experts.1,2 The reality is that this crisis makes the modern drug development process very expensive and the costs have shown a significant upward trend for decades. It was estimated that the accumulated costs per approved new compound are in the region of $2,558 million. Interestingly, $1,098 million of this is spent in the pre-clinical development phase. A significant contributor to these high costs per approved drug is the extremely low success rates in clinical drug development; only 11.8% of drug candidates entering Phase I are likely to be approved.3 This number is even lower (~1%) if the whole drug development process, from target discovery to market launch, is taken into account.

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