In Vivo Target Validation Using Biological Molecules in Drug Development

Handb Exp Pharmacol. 2016;232:59-70. doi: 10.1007/164_2015_17.


Drug development is a resource-intensive process requiring significant financial and time investment. Preclinical target validation studies and in vivo testing of the therapeutic molecules in clinically relevant disease models can accelerate and significantly de-risk later stage clinical development. In this chapter, we will focus on (1) in vivo animal models and (2) pharmacological tools for target validation.

Keywords: Bleeding disorders; Gene therapy; Gene-editing; Hemophilia; Monoclonal antibodies; Mouse models; Non-mammalian models; Recombinant proteins; Target validation.

Publication types

  • Review

MeSH terms

  • Animals
  • Disease Models, Animal
  • Drug Discovery*
  • Drug Evaluation, Preclinical
  • Mice
  • Molecular Targeted Therapy*