Predicting drug efficacy: knockouts model pipeline drugs of the pharmaceutical industry

Curr Opin Pharmacol. 2003 Oct;3(5):563-70. doi: 10.1016/j.coph.2003.04.002.


One of the major challenges for the pharmaceutical industry is to develop innovative drugs to new targets from the human genome. A systematic approach for target selection could significantly increase the rate of successful new drug development, thereby enhancing industry productivity. It has previously been shown that mouse knockout phenotypes for the targets of the 100 best-selling pharmaceutical drugs correlate well with known drug efficacy. Furthermore, physiological validation of novel pipeline targets of the pharmaceutical industry has been provided using mouse knockout data. These data demonstrate an excellent correlation between knockout phenotype and anticipated drug efficacy, establishing an important marker for superior new drug targets from the genome.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Design*
  • Drug Industry
  • Genome, Human*
  • Humans
  • Mice
  • Mice, Knockout / genetics*
  • Mice, Knockout / metabolism
  • Phenotype
  • Technology, Pharmaceutical / methods*