The identification of a new drug target, such as an enzyme associated to a disease process, is often the initial step in the discovery and development of first-in-class drugs. Once a new target has been found, several strategies are employed to validate the target and support decisions, such as launching a large-scale drug development programme, conducting a proof-of-concept trial in humans, or collaborating with another company. Inadequate early-stage validation of therapeutic targets has been linked to costly clinical failures1 and low medication approval rates. The use of genomics to inform drug discovery and development pipelines has sparked both excitement and skepticism during the last three decades. Target validation can be characterized in a variety of ways depending on context, but it usually refers to a technical assessment of whether a target plays a crucial part in a disease process and whether pharmacological modulation of the target is efficacious in a given patient population.