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Leveraging human genetic data to inform drug discovery and development.
Safe People
Organisation name
Sequoia Genetics Ltd
Organisation sector
Commercial
Applicant name(s)
Dipender Gill
Funders/ Sponsors
Safe Projects
Project ID
OFHS260020
Lay summary
This study will use large-scale genetic data from the Our Future Health programme to help identify and evaluate potential new drug targets for diseases where better treatments are needed. Genetic differences between people can act as natural experiments that help researchers understand how specific genes influence health and disease. By analysing these genetic differences across many participants, we can investigate whether modifying the activity of proteins that are coded for by those genes may have beneficial or harmful effects. First, we will identify DNA changes that can act as reliable indicators of how specific genes influence disease risk. This can help highlight genes whose proteins may serve as promising targets for new medicines or help validate targets already being explored by researchers. Second, we will examine the likely health effects of variation in the genes coding for these targets, including both potential benefits and possible side effects. Understanding these effects early can help guide the development of safer and more effective treatments. Finally, we will explore whether computers that learn from data (machine learning) can help us identify promising drug targets and predict their likely therapeutic outcomes. Together, this work aims to support the discovery and prioritisation of new medicines.Knowledge Gaps: Most drugs fail in clinical trials because their targets aren't validated in humans. Current drug development relies heavily on animal models, which don't always predict how drugs will work in people. Human genetic data has the potential to overcome these limitations. Significance of Research: Human genetic information offers another way to study disease and potential treatments. Natural differences in genes between people can show how changes in certain biological processes affect health. In some cases, these natural differences can mimic the effects of medicines that increase or reduce the activity of a gene. Studying these patterns can help researchers understand which genes or biological pathways may be good targets for new treatments. Anticipated Research Significance: The Our Future Health programme provides a unique opportunity to do this research at a very large scale. It includes genetic information and health data from many volunteers across the UK. By analysing these data together, we can identify patterns that link genes with health outcomes and gain insights into how diseases develop. This information can help guide future research into new medicines and improve our understanding of human health.
Public benefit statement
This research supports the discovery and development of safer and more effective medicines for common health conditions. These include illnesses that affect many people in the UK, such as heart disease, diabetes, respiratory conditions, immune-related diseases, brain disorders, and kidney disease. These conditions lead to poor health, disability, and early death, and many still lack effective treatments. By studying genetic and health information from the Our Future Health programme, this research will explore how natural differences in genes influence health and disease. These genetic differences can provide clues about whether changing certain biological processes in the body might improve health or cause unwanted effects. Learning this early in the research process can help identify which treatments are most likely to work. This approach may help reduce the number of medicines that fail during clinical trials and allow researchers to focus on the most promising treatments. Longer term, this could speed up the development of new medicines, ensure they are safer and more effective. It may also help identify groups of people who are more likely to benefit from certain treatments or experience side effects. Overall, this work aims to contribute to better treatments, and longer, healthier lives for the public.
Request category type
Public Health Research
Other approval committees
Project start date
29/04/2026
Latest approval date
24/04/2026
Safe Data
Dataset(s) name
Safe Setting
Access type
TRE