One component of the high attrition rate in Drug Discovery stems from the selection of inappropriate biological targets that are not well validated nor supported by compelling scientific evidence. Thus, a robust validation of the biological target at an early stage is essential to increase confidence in its suitability as an efficacious mechanism of intervention in a pathological condition.
Aptuit’s multidisciplinary approach to target validation includes studies based on biological or pharmacological tools, and/or based on the distribution of the target in anatomically relevant areas. Moreover, substantiation of the initial hypothesis can be carried out through an in vivo approach, such as disease-relevant animal models, and/or through the most advanced gene manipulation techniques.
Biological results can be integrated with preliminary safety predictions and analytical confirmation of the pharmacological tool exposure in the biophase (plasma, brain, target tissues) in order to assess potential risks associated with the target and to define an early translation to clinical effects. Our medicinal chemists can support the identification and/or synthesis of pharmacological tool compounds according to client indications.
All the methodologies employed to validate the biological target can be performed as individual experimental packages, custom-tailored to meet clients’ specific needs, or as a fully integrated set of biological/safety/PK studies. The following disciplines support stand alone or integrated services for Target Validation: