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How do scientists identify possible targets for new drugs?

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A lab technician wearing protective gloves and face shield lifts a rack of deep-frozen cells from a cryopreservation tank emitting vapor in a laboratory setting. — pharmaceutical industry A technician withdrawing deep-frozen cells for in vitro culture at a laboratory of a German pharmaceutical company.

Scientists identify potential drug targets through rigorous basic research and advanced genomic analysis, which are essential for developing effective medical treatments.

Research and Discovery

The process often begins with basic research conducted by scientists and physicians in universities and public institutions. By studying physiology, biochemistry, and microbiology, researchers gain a deeper understanding of natural substances and bodily processes. This research helps identify specific biological components—such as enzymes, receptors, or transport processes—that play a role in disease and can serve as targets for new drugs.1

Genomic Insights

Modern genomics has revolutionized target identification. By analyzing entire genomes, scientists can pinpoint genes associated with hereditary diseases and identify proteins that are expressed in specific tissues or disease states. Techniques like functional genomics and the use of microarrays allow researchers to map the "transcriptome," revealing which genes are active during different stages of development or disease progression.2

Targeted Therapies

In fields like oncology, understanding the specific molecules involved in tumor growth allows scientists to design targeted therapies. By focusing on molecules responsible for specific cell behaviors, researchers can create drugs that are more precise and less toxic than traditional treatments.3