Now as Director, how do you see Open Targets going forward?
On the public side, I think that we’ve made a real impact with the Open Targets Platform and Open Targets Genetics, and we want to ensure that those will continue to be maintained.
Within the consortium, the driving question behind our work is: how do we take the existing research programme and convert it into targets or outputs that can be useful to our partners and feed into the drug discovery process? We want to use our experimental research programme to deliver cutting edge experiments and produce information about targets in disease.
From the genetics side, this includes understanding how to take forward common disease GWAS to identify the genes that are likely to be targets, as well as gene modulation and synthetic lethality screens in cancer or in neurodegenerative disease. Following on from that, we need to pinpoint the cellular locations of the effects, to understand where the genes we’ve identified are acting to cause disease and where we can intervene to modulate the disease. In that line of investigation, approaches like single cell sequencing or single cell expression quantitative traits will be essential.
There are bioinformatics associated with all of those processes, to make sense of the data we are generating. Our next steps in this area are to improve the way that we integrate data, to better identify the correct targets in disease. This will involve developing our machine learning capabilities, as well as combining the experimental data we are generating with the systematic integration of data from public resources.
Ultimately, our aim is to create a complete package of data pinpointing the most likely targets to be of interest in disease, including parameters such as tractability, cellular location, network expansion, etc. But the question is such an open-ended one that we won’t be able to address all aspects; we do our best to address individual problems, while bearing in mind how all the parts fit together.
And finally, what has been the most memorable moment of your career?
My involvement with the Human Genome Project has afforded me a few brushes with fame. In 1999, after we published the sequence of chromosome 22, I was invited to give a talk in Lisbon on live television. That evening, we went out for a drink, and when I went up to the bar, a man I’d never met before turned to me and said “You were that guy on television, weren’t you? Let me get you a drink!” And I thought: this is fame!
None of that has happened to me since, but it does show that there are occasionally unexpected rewards for being involved in science.