AbstractAttrition is a major challenge in drug discovery and development, with more than half of clinical studies failing due to lack of efficacy (Nelson et al. 2015). Analyses of historical pipeline data revealed that drug targets with human genetic evidence of disease association are twice as likely to lead to approved drugs (King et al. 2019). Therefore, leveraging human genetic data to identify new therapeutic targets is anticipated to have a measurable impact on their successful clinical development. To this end, AstraZeneca launched the ambitious Integrated Genomics Initiative in 2016, run by its Centre for Genomics Research. This Initiative has the bold aim to analyse 2 million human genomes by 2026 and to embed human genomics in its research and development pipeline. In this talk, I will provide an overview of the Initiative
s strategy, involving close collaborations with academic partners and global consortia, and review our latest progress. I will highlight recent insights from rare-variant analyses of complex diseases, such as chronic kidney disease, based on whole-exome sequencing data in large cohorts of patients and healthy controls. Finally, I will present our analytical engines and tools, allowing us to investigate systematically over 2 billion gene-phenotype associations in the UK Biobank data. In summary, the Genomics Initiative offers the opportunity to transform drug target discovery and has the potential to deliver new, innovative treatments for patients.