Using functional genomics to guide drug discovery

The pharmaceutical industry is keen on reducing the staggering rate of failure for targets in the drug discovery pipeline. One approach is to improve the identification and selection of potential targets, so drug development teams can focus on more hopeful candidate targets from the beginning. In this respect, results from many types of functional genomics experiments can provide information or evidence for the relationship between potential targets and their associated disease at the:

• DNA level (single nucleotide polymorphism (SNP) and copy number variations, epigenetics)
• RNA level (gene expression microarrays and RNA-seq)
• protein level (DNA/RNA-protein interactions e.g. ChIP-seq)

Functional genomics in cancer studies

Using functional genomics to guide drug discovery has been particularly useful for cancer studies. For example the discovery that the gene HER2 is over-expressed in certain types of breast cancers led to the development of the drug Herceptin (The HER2 Journey). More recently, high throughput analyses and meta-analyses of data from breast cancer samples have uncovered many additional targets for which existing drugs can be re-purposed, significantly speeding up the process of drug discovery (7).