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Approved Research

Using large-scale human genetic data to accelerate the drug development pipeline at CSL

Principal Investigator: Dr Monther Alhamdoosh
Approved Research ID: 81045
Approval date: May 12th 2022

Lay summary

CSL is a global biotechnology company with the goal of developing innovative and effective drugs for a wide range of human diseases. The process of developing a successful drug is very costly and time consuming, taking an average of $1 billion and 10 years per drug. Most drug candidates fail, mainly for reasons of lack of efficacy or safety concerns. Recently, genetics has emerged as a powerful approach to provide important evidence to support the entire development process. In this project, we will use the UK Biobank to conduct large-scale genetic analyses of diseases and health conditions of interest to CSL, across the five therapeutic areas of cardiovascular & metabolic disease, respiratory disease, transplant, immunology, and haematology. The genetic results will be used to explore (i) the potential utility of drug candidates in treating or preventing these conditions and diseases, (ii) whether new targets can be discovered, (iii) whether new biomarkers can be developed to better predict risk of such diseases, and (iv) the identification of patient groups that would likely benefit most from treatment. The anticipated public health impact of this project will be accelerated development of more effective drugs for a range of human diseases and health conditions, together with better targeting of these drugs to the suitable patients.