Approved Research

Development and deployment of statistical genetic models of common complex diseases for therapeutic hypothesis generation

Lay summary

Most new drugs fail to reach the clinic and make an impact on patient lives. These drugs often fail to show efficacy in clinical trials, suggesting that the biological rationale for their development was faulty. One critical piece of information that can greatly improve the probability of success is human genetics. The key insight is that for many diseases, genetics causes disease, not the other way around.

Although the scientific community recognizes the value of human genetics for investigating the biology of disease and identifying new drug targets, the process remains laborious and time consuming. One of the key limitations is that the approaches which work for rare diseases caused by a single gene do not work for common diseases which could be caused by many genes, each with a small effect. The greatest public health burden worldwide is driven by common diseases and is poorly addressed by existing approaches to discover new drugs. 

Vesalius Therapeutics, Inc. is creating an innovative platform which can more robustly discover the causes of common diseases and more quickly identify new targets and generate new therapies. By anchoring our approach in human genetics, we can increase our probability of correctly identifying the causal mechanisms underlying patient symptoms and identifying new drugs that can successfully correct those mechanisms. To achieve our goals, we will use the UK Biobank genetic data as a rich resource to develop our platform.