| Title: | Leveraging human genetic data to inform drug discovery and development. |
| Lead Institution: | Sequoia Genetics Ltd. |
| Principal investigator: |
Application 536820
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About
Scientific rationale: Drug discovery and development is slow and expensive, with a high failure rate. A lot of this inefficiency is due to difficulty in inferring causal effects of epidemiological associations and translating the findings of animal studies to humans. Human genetic data can help overcome these limitations, as they can be used to inform on the causal effects of genes coding for putative drug targets. This project will leverage large-scale human genetic association data related to potential perturbation of putative drug targets to help inform on drug target effects, and thus drug discovery and development efforts. Research question: What existing and novel drug targets are supported by human genetic evidence, and what can genetic association data tell us about potential indication mapping, biomarkers, adverse effects and population subgroup heterogeneity for these drug targets? Research aims: 1. To use human genetic evidence to identify proxies for perturbation of existing and novel drug targets, with the purpose of informing on their potential efficacy for different outcomes, secondary indications, biomarkers, adverse effects and heterogeneity in effects across population subgroups. 2. Use these insights from human genetic data to directly inform drug discovery and development efforts. 3. Triangulate findings from human genetic evidence with findings from traditional epidemiological studies.1 Publication
| Pub ID | Title | Author(s) | Year | Journal |
|---|---|---|---|---|
| 20506 | Comparison of methods for assessing effects of risk factors on disease progression in Mendelian randomization under index event bias | Linxuan Zhang (+10) | 2026 | American Journal of Human Genetics |
Enabling scientific discoveries that improve human health