Look Towards A New Future

Oct 4, 2012

Progress in Target Validation

Target validation is one of the first and most important steps in drug discovery; poor target validation is a major factor in clinical stage attrition. Target validation involves the thorough assessment of a molecular target, evaluating its physiology, pathology, and pharmacology and determining its role in altering the disease phenotype. This report assess technological advances in the field.

Features and benefits
  • Discover how and patient-derived human primary cells and induced pluripotent stems cells lines are being used to validate new drug targets.
  • Understand how RNAi technology can be applied to both in vitro and in vivo settings to confirm the involvement of targets in disease pathways.
  • Identify the omic technologies that are being used reveal new options for modulating disease pathways and new interactions of known drugs.
  • Understand the power of high-content screening in probing functional changes occurring at the cellular level.
  • Assess the role of bioinformatics in managing the vast quantities of data that are being created through target validation projects. 
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Report Details:

Published: September 2012
No. of Pages: 44
Price:Single User License:US$2875 Corporate User License:US$10781


In recent years there has been a paradigm shift, with greater emphasis placed on the validation of a target in human cell and animal disease models prior to screening for lead compounds. This facilitates in-depth knowledge of the underlying biology of a disease and allows evaluation of upstream and downstream pathways of new targets.

RNAi-based technologies offer high throughput, a high-predictive value, relatively low cost, short experimental times, and the ability to knock down several genes in vitro and in vivo.

Improvements in molecular imaging and bioinformatics have enabled high-content screening to become more widely applied to measure phenotypic endpoints, allowing structural and biochemical changes within cells, tissues, organs, or whole organisms to be visualized in real time, leading to improvements in target validity.

Your key questions answered
  • What novel technologies have been developed to improve target validation in vivo?
  • What role do systems biology and network pharmacology have in target valdiation?
  • Which companies are leading the field in the use of in vivo models in target validation?
  • What opportunities do stem cells offer for validating targets?