Small molecule target

A small molecule target is a general term for a biological macromolecule, typically a protein, whose activity is modulated by a low-molecular-weight organic compound to achieve a therapeutic effect [1, 6]. These targets encompass a wide variety of functional classes, including enzymes, G protein-coupled receptors (GPCRs), ion channels, and nuclear receptors, which together account for the majority of approved drugs [1, 2]. Small molecules are characterized by their ability to diffuse across cell membranes, allowing them to interact with both extracellular and intracellular targets [5, 7]. The interaction usually involves the binding of the small molecule to a specific pocket or active site on the target, leading to inhibition, activation, or allosteric modulation of its biological function [1, 9]. Identifying and validating these targets is a critical step in drug discovery, as it allows for the optimization of drug potency and selectivity while minimizing off-target effects [3, 4, 6]. While proteins are the most common targets, recent research has also expanded to include RNA and protein-protein interactions as viable small molecule targets [5, 9, 10].

Other names

Drug targetMolecular targetTherapeutic targetBiological targetProtein target

Small molecules typically act through enzyme inhibition, receptor antagonism or agonism, ion channel modulation, or allosteric regulation of protein function.