Blanco Maria-Jesus, Gardinier Kevin M
Sage Therapeutics, Inc., Medicinal Chemistry, 215 First Street, Cambridge, Massachusetts 02142, United States.
Novartis Institutes for BioMedical Research, Global Discovery Chemistry, 22 Windsor Street, Cambridge, Massachusetts 02139, United States.
ACS Med Chem Lett. 2020 Jan 14;11(3):228-231. doi: 10.1021/acsmedchemlett.9b00582. eCollection 2020 Mar 12.
The classical toolbox for drug discovery is continuously expanding beyond traditional small molecules. New chemical modalities including RNA therapeutics, protein degraders, cyclopeptides, antibody drug conjugates, and gene therapy have matured, demonstrating clinical success and are now considered early in target appraisal. In this Viewpoint, we highlight recent progress in the field.
药物研发的经典工具库正在不断扩展,超越了传统小分子药物的范畴。包括RNA疗法、蛋白质降解剂、环肽、抗体药物偶联物和基因疗法在内的新化学模式已经成熟,展现出临床成效,目前被视为靶点评估的早期阶段。在本观点文章中,我们重点介绍了该领域的最新进展。