Short Courses

Monday, March 16 | 3:00 – 6:00 pm

SC1: Circular RNAs as a New Therapeutic Modality - Detailed Agenda
Circular RNA (circRNA) is a type of single-stranded RNA which forms a continuous loop due to the covalent binding of its 3′ and 5′ ends. They are naturally formed in the cell and found to play a role in cancer, CNS, cardiovascular and other diseases. The closed loop structure makes it less susceptible to exonuclease-mediated degradation and presumably more stable than most linear RNAs, which is appealing from a therapeutic standpoint. Studies involving the formation and function of circRNAs are still preliminary, however, there is a lot of interest in exploring its role in disease and how they can they used. This course aims to provide an introduction to circRNA biology, their function and how they can be used as a new therapeutic moiety. The chemistry leading to design and manufacturing of circRNAs as potential therapeutics, and issues dealing with dosing, biodistribution, immunogenicity will also be discussed.

Samie Jaffrey, MD, PhD, Department of Pharmacology, Weill Medical College, Cornell University

Bojan Losic, PhD, Associate Professor, Department of Genetics and Genomic Sciences, Icahn Institute for Data Science and Genomic Technology, Icahn School of Medicine at Mount Sinai
Additional Instructors to be Announced

Monday, March 16 | 6:30 – 9:30 pm

SC2: Examining the Safety and Toxicity of Nucleic Acid Therapeutics - Detailed Agenda
Nucleic acid drugs continue to deliver on their promise to become a third therapeutic modality, in addition to small molecules and biologics. Several antisense oligonucleotide drugs have been on the market for some time, while the first RNAi approval was granted in 2018. In addition, numerous mRNA and CRISPR therapeutic programs have entered clinical stages. Despite the common “nucleic acid” component, the mechanisms of action and of non-specific effects differ for each of these drug types.

Jeffrey Foy, PhD, Senior Director, Toxicology, Dicerna Pharmaceuticals

Shawna Weis, PhD, Senior Director, Regulatory Toxicology, Wave Life Sciences

Xiao Shelley Hu, Senior Director, Head of DMPK and Clinical Pharmacology, Wave Life Sciences


Tuesday, March 17 | 6:30 – 9:30 pm

SC3: Oligonucleotides for Cancer Immunotherapy - Detailed Agenda
Oligonucleotide-based therapies are now gaining attention as an alternative to antibody and small molecule-based therapies for cancer immunotherapy. In cancers, where current treatment options are limited by efficacy and specificity, oligonucleotide-based drug modalities are offering a good alternative. This course will bring together experts who will share their perspectives on the opportunities and challenges underlying the generation of novel, more targeted and effective oligonucleotide-based drugs for cancer immunotherapy.

Shanthi Ganesh, PhD, Associate Director, Preclinical Oncology, Dicerna Pharmaceuticals, Inc.
Weston Daniel, PhD, Senior Director Program Management, Exicure, Inc.
Sushma Gurumurthy, PhD, Associate Director, Oncology Research, Moderna

Wednesday, March 18 | 5:00 – 8:00 pm

SC4: Gene Editing for Targeted Therapies - Detailed Agenda
While the challenges and risks associated with oligonucleotide therapies still remain, there is a new and better understanding of how DNA and RNA can be effectively manipulated and delivered. With the rise of gene editing tools and enhanced knowledge of targeted delivery, these therapeutic modalities are once again being embraced with renewed hope and enthusiasm. This course helps you understand how DNA and RNA editing – particularly the one enabled by the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas9 system – works, and how it can be used to help develop targeted therapies.

Clifford Steer, M.D., Professor of Medicine and, Genetics, Cell Biology, and Development, University of Minnesota Medical School
Branden Moriarity, PhD, Assistant Professor, Department of Pediatrics, University of Minnesota Medical School 

Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women’s Hospital

Jonathan Gootenberg, PhD, McGovern Fellow, McGovern Institute for Brain Research, MIT

Omar Abudayyeh, PhD, McGovern Fellow, McGovern Institute for Brain Research, MIT

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Oligonucleotide Discovery and Delivery