RNA as a Drug Target

Discover a new paradigm in drug discovery that greatly expands the space of addressable drug targets and potential novel drugs

Existing paradigms for drug discovery have focused largely on enzymes and other proteins as drug targets. In recent years, however, different varieties of ribonucleic acids have emerged as a viable focus for target-based drug discovery, with the potential to revolutionize the strategy and approach for this essential step in the drug development process.

RNA as a Drug Target: The Next Frontier for Medicinal Chemistry offers a practice-oriented introduction to developing drug-like small molecules that selectively modulate both coding and non-coding RNAs. Beginning with a description and characterization of existing druggable RNAs, the book discusses how to approach different RNA targets for drug discovery. The result is a crucial resource for targeting RNAs and creating the next generation of life-saving pharmaceuticals.

RNA as a Drug Target readers will also find:

• A complete “toolbox” for working with RNA, from structure determination to screening and lead generation techniques
• A wide range of addressable targets and mechanisms, including splicing modulation, riboswitches, targeted degradation, and more
• Authoritative discussion of the potential of RNA-targeted small molecule therapeutics for drugging the epitranscriptome

RNA as a Drug Target provides an expert introduction to a new frontier in pharmaceutical research for medicinal chemists, biochemists, molecular biologists, and members of the pharmaceutical industry.

John Schneekloth, Jr., PhD (Jay), obtained his PhD from the chemistry department of Yale University. Following postdoctoral work at Princeton, Dr. Schneekloth joined the NCI, where his research involves using synthetic chemistry and high throughput chemical biology approaches to develop chemical probes of RNA, with a particular emphasis on targeting RNA with druglike small molecules.

Martin Pettersson, PhD, obtained his PhD in Organic Chemistry from the University of Texas at Austin. He is a medical chemistry / drug discovery leader with over 20 years of industrial experience, mainly with Pfizer (co-inventor of Paxlovid). He has then continued as Senior Director of Neuroscience/Pain, Chemistry, at Grünenthal Boston Innovation Hub, and most recently as VP of Drug Discovery at Promedigen in South Korea.