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 Drug Target conference, part of Discovery on Target, as we discuss RNA as a novel target site for therapeutics.

Preliminary Agenda

Small Molecules to Target RNA

FEATURED PRESENTATION: Non-Coding RNA as a Small Molecule Drug Target
Elliott Nickbarg, PhD, Principal Scientist, Pharmacology Department, Merck & Co, Inc.

Directly Targeting RNA with Small Molecules
Meizhong Jin, PhD, Senior Director, Chemistry, Arrakis Therapeutics

Targeting Structurally and Functionally Diverse RNAs with Drug-Like Small Molecules
John S. Schneekloth Jr. (Jay), PhD, Principal Investigator, Chemical Biology Laboratory; Head, Chemical Genetics Section, Center for Cancer Research, National Cancer Institute, NIH

RNA-Protein Complexes

RNA Splicing Target Identification  
Tom Chappie, Associate Research Fellow, Pfizer

Identification of Development Candidate eFT226, a First in Class Inhibitor of eIF4A RNA Helicase
Justin Ernst, PhD, Director of Medicinal Chemistry, Effector Therapeutics

Modulating the Epitranscriptomic RNA Modifications for Cancer Therapy
Pawel Sledz, PhD, Senior Scientist, Department of Biochemistry, University of Zurich

Finding the Druggable Structures

Enabling Modulation of RNA Biology in Human Disease with Small Molecules
Razvan Nutiu, PhD, Investigator, Chemical Biology & Therapeutics, Novartis

Structure-Based Discovery of New Functions in Large RNAs
Kevin Weeks, PhD, Kenan Distinguished Professor of Chemistry, University of North Carolina

Oligonucleotides to Target RNA

FANA ASO Therapy to Silence Foxp3, Impair Treg Function and Promote Anti-Tumor Immunity
Wayne Hancock, PhD, Professor of Pathology and Laboratory Medicine, University of Pennsylvania, Chief of the Division of Transplant Immunology, Children’s Hospital of Philadelphia (CHOP)


For more details on the conference, please contact:
Mana Chandhok

Conference Producer

Cambridge Healthtech Institute

Phone: (+1) 781-972-1350



For partnering and sponsorship information, please contact:
Rod Eymael

Manager, Business Development

Cambridge Healthtech Institute

Phone: (+1) 781-247-6286