Genetics-Driven Research: Benefits and Challenges


  • More relevant to human biology than animal models of disease.
  • Increased potential for large effect sizes.
  • Higher clinical success rates.
  • Increased potential for first-in-class therapies.
  • Enhanced speed in some cases.
    • Less need to validate target through assays.
    • Confidence in target enables accelerated investment.
  • Insights into safety and potential side effects.


  • Targets may involve unexplored biology.
  • Targets may be difficult to drug.
  • Challenges with biology and druggability take time to solve.
  • May be less informative for some therapeutic areas.
  • Therapeutic hypotheses still need to be proven.
    • Can pharmacology mimic gene variant present from birth?
  • Not all variants provide both efficacy and safety insights.

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