Target Selection

This chapter provides an overview of target identification and validation as it applies to molecular targeted drug discovery. Proper target selection and validation represent critical initial steps in any drug discovery program. To qualify as an appropriate target, the target protein must display a specific set of key features. Key features include a specific function that plays a critical role in the pathogenesis or maintenance of disease; modulation of this function must elicit a disease-modifying phenotypic outcome; the target function must be dispensable in normal (non-disease) cells or tissues; the target must be actionable, meaning that it contains pockets appropriate for drug binding; lastly, clear evidence of an unmet medical need among patients that may be fulfilled by a drug that modulates the specific target.