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Pre-Conference Workshop Day

Workshop A - Monday | December 2, 2019

Empowering RNA Drug Target Validation: “An Essential Initial Step for Every Drug Discovery Effort”
09:30 – 11:30

(miR-21 and its role in cancer will be used as a case study for the discussion)

How do we ensure that the RNA we are targeting is a high value drug target?

  • As drug hunters how do we validate a RNA as a drug target? Translational medicine in the area of RNA Medicine is still in its nascency, there are very few examples in literature that gives us strong confidence of the role RNA plays in disease.
  • How do we as drug hunters in industry collaborate best with academic medical research institutes to accelerate the research in translational medicine to best elucidate the role of RNA in disease?
  • What are the best tools to elucidate the role of RNA in disease?

Drug discovery on target

  • Once we validate a specific RNA as a drug target how do we generate the appropriate drug like tool molecules to ensure we guide our drug discovery effort with specificity? Drug discovery on Target is critical and always a challenge, particularly with small molecules that are promiscuous in their nature.
  • Mechanism of Action; are CRISPR and oligonucleotides appropriate control tools when you develop small molecules targeting RNA?
  • Cell based systems for screening molecules, compare and contrast, do they correlate?

2D Cell Cultures vs. 3D Cell Cultures

3D Organoids vs. Mouse Models

Johan Pontin

Workshop Leader

Johan Potin
Founder and Former CEO
The RNA Medicines Company

Workshop B - Monday | December 2, 2019

Exploring Drug Discovery Approaches to RNA
12:30 – 14:30

Shifting the paradigm from targeting protein to targeting RNA requires rethinking several aspects of drug discovery including target selection, lead discovery, assays to support lead optimization as well as in vivo pharmacodynamic markers.  This workshop will provide an overview of all the published approaches to drugging RNA as well as highlight key technologies that may prove useful for initiating new programs.  This will be an interactive workshop where participants are welcome to share any learnings as well as discuss and debate the best directions to take in this highly challenging and exciting new area.

Key questions the workshop aims to address:

  • How should novel targets be selected/discovered for RNA-targeted approaches?
  • What mechanisms are possible or likely to work?  Discuss the various known approaches of affecting splicing, 5' UTR interference to decrease transcription, binding to mRNA etc?
  • What are the pitfalls when transitioning from targeting protein to targeting RNA (what mindsets need to change)?
  • What are the key technologies useful for drugging RNA?  What is the role of structure-based design, computational methods, biophysics, cell-based assays etc?
  • What are appropriate pharmacodynamic (PD) markers for RNA targeted therapy and what level of PD can we expect (partial or full PD responses?).

Workshop Co-Leaders

Margaret Scott

Margaret Porter Scott
Vice President, Biochemical & Cellular Pharmacology

John Moffat

John Moffat
Senior Scientist, Biochemical & Cellular Pharmacology

Workshop C - Monday | December 2, 2019

Enabling Targeting Non-Coding RNAs with Small Molecules: Why, How and to Whom?
15:00 – 17:00

One of the most fascinating discoveries in molecular oncology has been that cancer represents a disease in which genetic alterations in protein-coding, but also in non-coding genes complement each other. MicroRNAs (miRNAs) are a type of non-coding RNA (ncRNA) transcripts that can regulate gene expression primarily by disrupting messenger RNA (mRNA) translation and/or stability, or alternatively by modulating the transcription of target mRNAs. For the last decade, miRNAs have shown to be pivotal characters of every single one of the cancer hallmarks. Profiling studies have proven the significance of identifying over-expressed miRNAs (oncomiRs) causative of the activation of oncogenic pathways that lead to malignancy. Due to their crucial role in cancer, it has become a challenge to develop efficient miRNA-inhibiting strategies such as antagomiRs, locked nucleic acids or antisense oligonucleotides. However, to this date, the accessible delivery agents and their pharmacokinetic/pharmacodynamic properties are not ideal. Thus, there is an urgent, unmet need to develop miRNA-based inhibitory therapeutics. Herein we will discuss the advantages and challenges of a novel therapeutic strategy: the use of small molecule inhibitors to target specific miRNAs (SMIRs), as well as the new concept of small molecules "hijacking" by oncogenic miRNAs.

Key questions the workshop aims to address:

  • Why develop SMIRs against oncogenic miRNAs?
  • What is the specific spectrum of patients to be targeted?
  • What is the small molecules "hijacking" by oncogenic miRNAs?
  • What are the best options to develop such SMIRs?

Is RNA therapeutics in cancer still a viable option?

George Calin

Workshop Leaders

George A. Calin
Professor, Department of Experimental Therapeutics
The University of Texas MD Anderson Cancer Center

Anna M. Krichevsky

Anna M. Krichevsky
Associate professor of neurology
Brigham and Women’s Hospital
Dana-Farber/Harvard Cancer Center;
HMS Initiative for RNA Medicine
Harvard Medical School 




Gabriele Varani
Professor of Chemistry
University of Washington