Short Courses*

Monday, March 25 6:30 – 9:30 pm

SC1: 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, besides small molecules and biologics. Several antisense oligonucleotide drugs have been on the market for some time, while the first RNAi approval was granted last year (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.

Topics to be discussed include:

• Different types of nucleic acid-based drugs
• Mechanisms of actions and non-specific effects
• Current approaches to address non-specific and potentially toxic effects

Aimed at both novice and advanced nucleic drug developers, the course will:

• Introduce and explain the differences between various types of nucleic acids drugs
• Summarize our current understanding of the origins of non-specific and potentially toxic effects
• Provide certain directions as to how to minimize the potential toxic effects of nucleic acids drugs

Instructors:

Muthiah (Mano) Manoharan, PhD, Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals

Dmitry Samarsky, PhD, CTO, Sirnaomics

Richard S. Geary, PhD, Senior Vice President (SVP), Development, Ionis Pharmaceuticals

Thomas Madden, PhD, President & CEO, Acuitas Therapeutics

Jeffrey Foy, Senior Director, Toxicology, Dicerna Pharmaceuticals

Tuesday, March 26 6:30 – 9:30 pm

SC2: Oligonucleotide-Based Drugs 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 better 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 therapies for cancer immunotherapy. Some topics that will be discussed include:

• Modulating the tumor microenvironment using novel approaches and drug modalities
• Emerging immuno-oncology targets (TLRs, STING, RIG-1 and more) for intervention using oligos
• Preclinical and clinical evaluation of new immunomodulatory pathways and nucleic acids
• Synthesis and delivery of oligonucleotides (siRNA, antisense, aptamers and others) for cancer immunotherapy

Instructors:

Shanthi Ganesh, PhD, Associate Director, Preclinical Oncology, Dicerna Pharmaceuticals, Inc.

A. Robert MacLeod, PhD, Vice President, Oncology Research and Development, Franchise Head Oncology, Ionis Pharmaceuticals, Inc.

Weston Daniel, PhD, Senior Director Program Management, Exicure, Inc.

Wednesday, March 27 5:30 – 8:30 pm

SC3: CRISPR-Based 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 genes 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 gene 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 with good efficacy and delivery.

Instructors:

Clifford Steer, MD, Professor of Medicine and Genetics, Cell Biology and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School

Ciro Bonetti, PhD, Scientist, Regeneron Pharmaceuticals

Eric Kmiec, PhD, Director, Gene Editing Program and Senior Research Scientist, Center for Translational Cancer Research, Helen F. Graham Cancer Center & Research Institute, Christiana Care Health System

* Separate registration required.