Genetics in Drug Development course

The next live course will be running in November 2026. Registration is open HERE.

The majority of biologic and small molecule drugs perturb protein targets to exert their effects. With the recent explosion in the availability of large-scale genetic association data, it is increasingly feasible to identify genetic variants that proxy the effect of perturbing a protein drug target. Leveraging such genetic data thus offers an efficient and cost-effective approach for identifying drug targets and studying their effects. While this course is entirely self-contained, it follows on naturally from the Mendelian Randomization course. Participants who are interested in both topics may wish to apply for both courses.

For those interested in learning more on the use of genetics in drug development, an anthology curated by the course lead and members of the Burgess group can be bought from Amazon HERE.

Course objectives

This short course will provide theory and practical advice on using genetic data to:

  1. Identify drug targets
  2. Investigate the effects of drug target perturbation for different indications
  3. Explore potential adverse effects and repurposing potential for drug targets
  4. Identify biomarkers to explore drug efficacy in early stage clinical trials
  5. Identify population subgroups for which drug effects may vary
  6. Explore interactions between different drugs and exposures

Intended audience

This course is designed for all those interested in leveraging genetic data for drug development and investigation of drug effects, most notably:

  • Clinicians
  • Academics
  • Employees of pharmaceutical companies

A basic understanding of genetic epidemiology, drug development and statistics is desirable, although not essential as these topics will also be covered briefly during the course. Experience of using the R statistical software is desirable, although not essential. All code for the practical examples used during the course will be provided for R.

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Mendelian randomization course

Genetics in Drug Development course

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Course structure

The short course will be held online over one week and will consist of a series of recorded video lectures, live interactive sessions with practical examples, and participation in an online community that allows for interaction with peers and tutors throughout the course. 

The drop-in Introduction and Q&A session act as both “office hours” and networking opportunities for participants, with attendance strongly encouraged.

Access to on-demand course material will be enabled one week before the start of the course, and will continue for one month after the course finishes.

  • Talk 1: An introduction through examples
  • Talk 2: Identifying drug targets and their genetic proxies
  • Talk 3: How to proxy drug effects using Mendelian randomization studies
  • Talk 4: Colocalization for studying drug effects
  • Talk 5: Advanced methods and pharmacological considerations
  • Talk 6: Guest lecture by Prof. John Whittaker, Director of the MRC Biostatistics Unit, University of Cambridge

Sessions start at 14.00 UK time. We do not expect all attendees to be present for every session.

Every session will be recorded and accessible to participants for two weeks after the course. 

For further information about the course, please contact: burgess-group-admin@mrc-bsu.cam.ac.uk