Pharmacology | In Drug Discovery And Development

Pharmacology begins long before synthesis. Using knowledge of disease pathology, pharmacologists identify biological targets—usually proteins, receptors, enzymes, or ion channels—that are implicated in a disease state. For example, in hypertension, the angiotensin-converting enzyme (ACE) is a validated target. However, a target is just a theory until validated. Pharmacologists use techniques like CRISPR gene editing or antisense oligonucleotides to "turn off" the target. If turning off the target alleviates the disease phenotype in cell cultures or animal models, the target is "validated."

Discovery pharmacology focuses on the earliest stages of the pipeline, where researchers seek to understand disease mechanisms and identify ways to intervene. Drug Discovery and Development Process - PPD pharmacology in drug discovery and development