drug target

A drug target is the specific molecular handle in the body that a drug grabs hold of to do its job. If the disease is a malfunctioning machine, the target is the particular lever the drug pushes or jams to fix how the machine runs.

Technically, it is a biomolecule whose activity a drug modulates to produce a desired therapeutic effect. Most targets are proteins, such as receptors, enzymes, ion channels, or transporters, though nucleic acids and other molecules can serve as targets too.

Choosing the right target is one of the most consequential decisions in a project, because a beautifully optimized molecule aimed at the wrong target will never cure the disease. This is why teams spend great effort on target validation, gathering evidence that changing the target really alters the disease.