RNA-Targeting Small Molecule Drugs

Cambridge Healthtech Institute’s 2nd Annual

RNA-Targeting Small Molecule Drugs

Designing and Optimizing Small Molecules to Modulate Diverse RNA Motifs and Pathways

May 18-19, 2021

Over the years there has been an increased understanding of the structure and function of diverse RNA moieties and their role in oncology, CNS, and other diseases. RNA has been a target for therapeutic intervention using other RNA entities like small interfering RNA (siRNA), antisense and aptamers. Only recently have scientists started exploring small molecules to target RNA, as they offer more structural diversity, oral bioavailability, and better drug-like properties. However, defining which RNA to target and how to target it is not easy, as some of the disease mechanisms and RNA ligand binding sites are not very well understood. Understanding the characteristics of disease-related RNA and finding the appropriate small molecules to create good RNA-ligand interactions to elicit the necessary physiological response, is no trivial task. Challenges also exist in terms of specificity, selectivity, and safety of these small molecules in vivo. The conference on RNA-Targeting Small Molecule Drugs will highlight some of the innovative approaches being pursued to find new small molecule drugs for targeting RNA for therapeutic intervention.


Coverage will likely include:


  • Tools for identifying the right RNA target for small molecule intervention
  • Determining the structural and physicochemical RNA-small molecule interactions for hit selection
  • Strategies for characterization and lead optimization of the RNA-targeting molecules
  • Bioanalytical/phenotypic screening for identifying and quantifying small molecule-RNA binding
  • Optimizing the efficacy and safety of drug candidates for clinical drug development
  • Tackling challenges with specificity, stability, biodistribution and toxicity      


If you would like to submit a proposal to give a presentation at this meeting, please click here.


The deadline for priority consideration is December 18, 2020.


All proposals are subject to review by session chairpersons and/or the Scientific Advisory Committee to ensure the overall quality of the conference program. Additionally, as per Cambridge Healthtech Institute policy, a select number of vendors and consultants who provide products and services will be offered opportunities for podium presentation slots based on a variety of Corporate Sponsorships.


We hope you can join us for this and the three concurrent day-and-a-half conferences during the first half of our 16th Annual Drug Discovery Chemistry event. Joining this virtual community in real-time allows you to ask questions of the speakers and engage with colleagues. Plus, you can return later to view the concurrent presentations and Q&A panels that you missed, via On-Demand access. 


For more details on the conference, please contact:

Tanuja Koppal, PhD

Senior Conference Director

Cambridge Healthtech Institute

Email: tkoppal@healthtech.com


For partnering and sponsorship information, please contact:

Rod Eymael

Manager, Business Development

Cambridge Healthtech Institute

Phone: (+1) 781-247-628

Email: reymael@healthtech.com