Target Identification and Validation in Drug Discovery
Target identification and validation in drug discovery represent critical steps that determine the scientific foundation and strategic direction of pharmaceutical research and development. A “target” refers to a biomolecule—such as a protein, DNA, or RNA—that can bind to a drug and elicit a specific biological effect. In target identification and validation in drug discovery, identification involves the systematic selection of disease-relevant molecular targets using various analytical and computational techniques, while validation confirms whether these targets play essential roles under physiological or pathological conditions. This process not only dictates the scientific rigor and likelihood of success in drug development but also directly affects the safety and efficacy of therapeutic candidates. As such, target identification and validation in drug discovery are widely applied in novel drug discovery, targeted therapy, personalized medicine, and the development of treatments for rare diseases. With the rapid advancement of systems biology, genomics, and proteomics, researchers are now able to explore potential drug targets at greater scale and depth. Since proteins represent the predominant class of drug targets, comprehensive knowledge of their expression levels, post-translational modifications, and interaction networks is indispensable. In this context, proteomics—particularly mass spectrometry-based platforms—has played a pivotal role in accelerating target identification and validation in drug discovery.
In practical applications, target identification and validation in drug discovery typically involve two major stages. The first stage focuses on target identification and includes approaches such as candidate mining based on known mechanisms, differential expression analysis driven by large-scale datasets, and network biology inference. Proteomics technologies—including differential expression profiling (e.g., DIA, TMT), protein–protein interaction mapping (e.g., Co-IP, AP-MS), and post-translational modification analysis (e.g., phosphorylation, acetylation)—serve as core tools in this phase. These techniques enable researchers to identify protein molecules that play central roles in disease onset and progression within complex biological systems, thereby nominating them as candidate therapeutic targets.
The second stage concerns target validation, wherein the potential of candidate targets for therapeutic modulation is experimentally evaluated. This stage commonly involves gene knockout or knockdown, protein overexpression, pharmacological perturbation, CRISPR screening, and functional assays in cellular models. Proteomics continues to contribute during this phase by elucidating downstream signaling alterations upon target activation and assessing drug-target binding specificity. Mass spectrometry enables quantitative profiling of proteome dynamics, measurement of drug-target interaction strength, and assessment of subsequent biological responses. These validation efforts enhance the reliability of target selection and provide a robust foundation for downstream applications such as small-molecule screening, antibody development, and preclinical studies. Thus, target identification and validation in drug discovery serve as a foundational process at the outset of drug development and are key drivers of precision therapeutics.
In recent years, increasing efforts have been directed toward so-called “undruggable” targets, including protein–protein interaction interfaces and non-coding RNA regulatory elements, which present both significant challenges and new avenues for innovation. The identification and validation of such targets rely heavily on the integration of multi-omics technologies and the deployment of high-sensitivity mass spectrometry platforms. Accordingly, target identification and validation in drug discovery has evolved into an interdisciplinary paradigm requiring seamless collaboration among molecular biology, cellular biology, computational biology, and proteomics.
MtoZ Biolabs, with deep expertise in proteomics and precision medicine, is dedicated to providing high-efficiency, professional, and customizable services for small-molecule target identification and validation in drug discovery, supporting clients in accelerating their path toward novel therapeutic breakthroughs.
MtoZ Biolabs, an integrated chromatography and mass spectrometry (MS) services provider.
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