Disease-relevant target protein

The term 'disease-relevant target protein' is a generic descriptor used in pharmacology and drug discovery to identify any protein whose functional modulation is expected to yield a therapeutic effect in a specific disease state [1, 2]. These proteins are the primary focus of drug development efforts, ranging from traditional small-molecule inhibitors and monoclonal antibodies to advanced modalities such as Proteolysis Targeting Chimeras (PROTACs) and molecular glues [1, 16]. A protein is deemed disease-relevant based on genetic, biochemical, or clinical evidence linking its activity to disease progression or maintenance [2, 3]. While the term does not refer to a single biological entity, the identification and validation of such targets are fundamental to the pharmaceutical industry [12, 13]. Challenges associated with these targets include the 'undruggability' of proteins lacking traditional binding pockets and the necessity of achieving high selectivity to avoid systemic toxicity [2, 4].

Other names

Drug targetTherapeutic targetPathogenic proteinNeosubstrateProtein of interest

Various mechanisms including inhibition, activation, and induced degradation.