Accelerating Precision Drug Development and Drug Repurposing by Leveraging Human Genetics

Abstract

The potential impact of using human genetic data linked to longitudinal electronic medical records on drug development is extraordinary; however, the practical application of these data necessitates some organizational innovations. Vanderbilt has created resources such as an easily queried database of >2.6 million de-identified electronic health records linked to BioVU, which is a DNA biobank with more than 230,000 unique samples. To ensure these data are used to maximally benefit and accelerate both de novo drug discovery and drug repurposing efforts, we created the Accelerating Drug Development and Repurposing Incubator, a multidisciplinary think tank of experts in various therapeutic areas within both basic and clinical science as well as experts in legal, business, and other operational domains. The Incubator supports a diverse pipeline of drug indication finding projects, leveraging the natural experiment of human genetics.

Keywords: BioVU; PheWAS; genetics; repurposing; translational research.

Fig. 1.

Comparison of drug development approaches. Drug development is currently an extraordinarily costly endeavor with a very low success rate, much of which can be attributed to incorrect therapeutic hypotheses based on preclinical models that do not accurately predict human biology. The ADDRI's work fits within the new paradigm of drug development, which holds the promise of improved time and cost efficiency compared with the traditional approach. Human data drive the entire process in the new paradigm, and this should lead to a higher likelihood of success. ADDRI, Accelerating Drug Development and Repurposing Incubator.