- 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.