Drug target

A biological molecule or process that a drug acts on to produce an effect. Targets can include proteins, nucleic acids, and cellular pathways.

Receptor (biochemistry): A protein or other molecule that binds a signaling molecule and changes cellular activity. Many drugs act by activating or blocking receptors that regulate cell behavior.
Target identification: The process of finding biological molecules or processes that may be altered to treat a disease. It establishes which biological candidate a drug-development program will pursue.
Drug discovery: The process of finding and developing compounds that can prevent, diagnose, or treat disease. Target choice helps shape screening strategies and the design of candidate medicines.
Off-target effect: A drug effect caused by interaction with a biological site other than the intended target. It explains effects that cannot be attributed to the drug’s intended target.
Undruggable target: A biological target considered difficult to modulate with conventional drug-like molecules. Some disease-linked targets lack accessible binding sites or other tractable features.
Enzyme: A biological catalyst that accelerates a chemical reaction without being consumed. Drugs can inhibit or alter enzyme activity to change biochemical reactions.
Target validation: The process of testing whether changing a proposed biological target can affect disease-relevant outcomes. Validation tests whether acting on the proposed target is likely to matter therapeutically.
Structure-based drug design: Drug design that uses a target’s three-dimensional structure to guide compound selection or modification. A known target structure can reveal binding sites for rational compound design.
Polypharmacology: The action of one drug on multiple biological targets. It contrasts with the idea that each drug acts through one isolated target.
Human genetics in drug discovery: The use of human genetic evidence to prioritize and assess therapeutic targets. Genetic evidence can strengthen or challenge claims that a target causes disease.

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