Addressing a long-standing challenge for medicinal chemists, this practical reference provides the concepts and tools for developing drug-like small molecules that selectively modulate non-coding RNAs. The introductory parts describes the wide range of druggable RNAs, available techniques for their characterization, binding assay development as well privileged structures in small molecule RNA binders. The main part systematically covers different RNA targets and how to approach them from a drug discovery angle, from splice modulation to riboswitches to targeted degradation, translation blockers and RNA modification. Throughout, the first successful examples of RNA-targeting drug candidates from the development pipeline of small and large pharmaceutical companies are discussed. This seminal reference on a new paradigm in drug discovery is required reading for every medicinal chemists who needs to stay abreast of current developments.
