Cambridge Healthtech Institute’s Inaugural

RNA as a Small Molecule Target

Expanding the Boundaries of Druggable Targets

April 17, 2020

RNA molecules are crucial for delivering cellular information and genetic regulation, but until recently, the drug discovery world has emphasized protein drug targets. Our lack of knowledge in RNA biology prevented us from exploring possibilities of RNA drug targets, but with recent advances in technologies, such as sequencing, new therapeutic strategies are being explored. Join us at the Inaugural RNA as a Small Molecule Target symposium, part of Drug Discovery Chemistry, as we discuss methods and tools to identify specific, potent, novel, small molecule binders of RNA.

Preliminary Agenda


Targeting Pre-mRNA Splicing with Small Molecules

Marla Weetall, PhD, Vice President, Pharmacology, PTC Therapeutics

Repurposing Tools for RNA – And What to Consider When Doing It

Jenifer Kaplan, PhD, Scientist II, Biophysics and Assay Development, Arrakis Therapeutics

Targeting Structurally and Functionally Diverse RNAs with Drug-Like Small Molecules

John Schneekloth Jr. (Jay), PhD, Senior Investigator, Chemical Biology Laboratory; Head, Chemical Genetics Section, Center for Cancer Research, National Cancer Institute, NIH

RNA-targeted Small Molecule Synthesis

Amanda E. Hargrove, PhD, Assistant Professor of Chemistry, Assistant Professor of Biochemistry, Duke University


Identification of Small Molecules that Promote Exon 10 Skipping in MAPT Pre-mRNA

Martin Pettersson, PhD, Research Fellow, Research Project Leader, Medicinal Chemistry, Pfizer

Discovering Novel RNA Binding Proteins for Small Molecule Drug Discovery

Pramod Pandey, PhD, Principal Scientist, Merck Research Labs Exploratory Science Center

RNA Splicing Modulation… Application to CD33

Tom Chappie, Associate Research Fellow, Pfizer

* The program is subject to change without notice, due to unforeseen reason.

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