Genome-Wide Association Studies
These findings have a high sensitivity to identify tractable drug targets.
Whole Exome Sequencing
Whole exome sequencing can unambiguously identify high-yield drug targets and the required direction of effect for a pharmacological intervention.
The Effector Index is our machine-learning algorithm that quantifies the probability that a specific drug target is causal for a disease of interest using large-scale datasets from GWAS.
Drug development programs with support from Mendelian randomization evidence have a strong track-record of reaching the market. We use Mendelian randomization to assess whether a specific target is causal for the disease of interest.
Heritable rare disease is often caused by highly penetrant Mendelian mutations. We use these findings to identify and validate drug targets often pointing to the direction of effect for the required pharmaceutical intervention.