Abstract

Precise Genome Editing and Stem Cell Technologies - Novel Tools for Novel Medicines

Lorenz Mayr, Vice President, AstraZeneca Ltd

Recent advances in Precise Genome Editing (PGE) and stem cell technologies have enormous potential to revolutionise the drug discovery process at all stages, from target identification through to toxicological studies. The ability to generate physiologically relevant cells in limitless supply makes induced pluripotent stem cells (iPSC) and embryonic stem cells (ESC) an attractive alternative to currently used recombinant cell lines or primary cells in all processes from hit finding/screening to lead optimisation and safety testing/toxicology/DMPK. Recent advances with precise genome editing of stem cells have further accelerated the use of these cells towards personalised medicines.. This presentation will describe the use of cell lines derived from human embryonic stem cells (ESC) and induced pluripotent stem cells (iPSC) for drug discovery at AstraZeneca. We will discuss in detail: • Generation & banking of stem cell lines • Precise genome modification technologies • Target identification studies • High-throughput screening • Toxicology testing • Personalised therapies • Summary & Outlook.