Enzymes

Enzymes are macromolecular biological catalysts, primarily proteins, that facilitate nearly all chemical transformations within living organisms by lowering the activation energy required for reactions (NIH, 2023). They are classified into seven main categories—oxidoreductases, transferases, hydrolases, lyases, isomerases, ligases, and translocases—based on the specific chemical reactions they catalyze (IUBMB, 2024). In the context of drug discovery, enzymes represent one of the most significant classes of therapeutic targets, with approximately 30% of all marketed pharmaceuticals acting by modulating enzyme activity (Nature Reviews Drug Discovery, 2018). Dysregulation of enzyme function, whether through overexpression, genetic mutation, or deficiency, is a fundamental driver of various pathologies, such as aberrant kinase signaling in malignancy or protease imbalance in inflammatory conditions (StatPearls, 2023). Therapeutic intervention typically involves small-molecule inhibitors that bind to active or allosteric sites to restore physiological balance, though achieving high isoform selectivity remains a primary challenge to avoid off-target toxicity (PubChem, 2024). Consequently, enzymes are central to both the understanding of disease mechanisms and the development of targeted therapies, including enzyme replacement for metabolic disorders.

Other names

Catalytically relevant proteinsBiocatalystsCatalytic proteinsEnzymatic proteins

Drugs targeting enzymes primarily act through competitive inhibition at the active site, non-competitive or allosteric modulation at distant sites to induce conformational changes, or irreversible covalent inactivation of catalytic residues (Nature Reviews Drug Discovery, 2018).