Masitinib, Onpattro, and Beyond: The Central Role of Synthetic Organic Chemistry

This presentation is directed toward a general audience, and thus it eschews specialist details of how and why molecular targets are selected and synthesized. Instead, it illustrates how curiosity-driven research in synthetic organic chemistry has inspired solutions to biomedical problems in the fields of degenerative, proliferative and infectious disease, nucleic acid therapeutics, cardiovascular health, and so on. In particular, it outlines how research on the synthesis of thiopeptide natural products enabled the development of new kinase inhibitors, cytotoxic agents, and anti-infective resources; how synthetic work on mitomycinoids and tetrodotoxin inspired the quantum leap that launched the field of nucleic acid therapeutics; and how a synthetic organic culture plays a central role in the development of the medicines of the future.