Identifying Correct Target in Medical Chemistry

This lecture focuses on identifying the correct target, efficacy, and safety in medical chemistry. It covers topics such as the correlation between in vitro enzyme inhibition data and cell-based efficacy outcomes, the role of genetic knockdown or knockout in determining on-target specificity, and the importance of stereochemistry in drug action. Case studies, including the discovery of imatinib mesylate (Gleevec®), are discussed to illustrate key concepts in medicinal chemistry. The lecture also delves into the rules of drug-likeness, structural alerts to avoid in drug design, and the factors influencing differences in in vitro versus in vivo efficacy. Overall, it provides insights into the critical aspects of medicinal chemistry related to target identification and drug safety.