What is target validation?

Applicants should have experimental data that validates the identified target. 

Points to consider:

  • Do you have a target, pathway and the ability to modulate these?
  • Importantly, do you have any evidence that your target is clinically relevant e.g. evidence from GWAS studies or evidence of mutations that cause a human phenotype?
  • Have potential toxicity issues been considered?

If you are unsure whether your research is at this stage or if you require funding to get to this stage, please contact a member of the team to discuss at [email protected]

Why does target validation matter?

The target is fixed at the beginning of a lengthy and expensive discovery and development programme. Investors need evidence to support the choice of target. Importantly, investors will need to see evidence that shows the target is involved in the human disease.

There are different types of target validation. Whilst it is unlikely that you will have validation at every level, having a combination of data will strengthen your application. Notably, all five levels of evidence relate to causation.

  • Theoretical: prediction of what might be expected to happen when a target is modulated based on general understanding of a biological pathway
  • In vitro: evidence that modulating the target results in a particular change in an in vitro system
  • In vivo: evidence that modulating the target results in a particular change in an animal model
  • Human: direct evidence the target causes the disease; usually, but not always, genetic evidence
  • Clinical: Proof-of-clinical-concept in man e.g. data from a previous clinical trial for a drug with undesirable properties