Abstract

Working Together to Unlock the Potential of Precise Genome Editing

Lorenz Mayr, Vice President, AstraZeneca Ltd

The recent discovery of the CRISPR/Cas9 technology (Clustered Regularly Interspaced Short Palindromic Repeats / CRISPR-associated protein 9) technology enables precise genome editing (PGE) in eukaryotic genomes at unprecedented ease, precision and short timelines. The technology has tremendous potential for drug discovery with its wide applications for target discovery & target validation, generation of in-vitro and in-vivo models of disease, bespoke models for safety & DMPK and its future applications in gene therapy as a therapeutic agent to treat disease at the molecular level with high efficacy and high safety. AstraZeneca has built a strong CRISPR/Cas platform with internal activities and external partnerships to make full use of this revolutionary technology in its drug discovery processes across all therapeutic areas. We will present case studies from the AstraZeneca-IMED research areas of Oncology and Autoimmune diseases and we will show the impact of the CRISPR/Cas technology in the fields of target validation, isogenic cell line engineering, mechanistic disease modelling, genome-wide target discovery and will review our partnerships with the academic community with the use of both pooled and arrayed gRNA libraries.