Wednesday, March 27 | 5:30 – 8:30 pm

CRISPR-BASED GENE EDITING FOR TARGETED THERAPIES

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.

5:30 Welcome Remarks and Speaker Introductions

5:40 The Cure to Human (and Non-Human) Disease Through Gene Editing

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

  • Evaluating target-based gene editing in the realm of personalized medicine
  • Identifying the appropriate clinical targets
  • Optimizing the strategies required to cure diseases
  • Establishing the safety parameters for gene editing
  • Identifying the translational aspects of preclinical safety assessments
  • Understanding the ethical issues involved in gene editing for human clinical disease

6:25 Dinner and Dessert Break 

6:45 Modeling CRISPR-directed Human Gene Editing In vitro Using a Mammalian Cell-free Extract

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

  • Defining reaction parameters that influence the balance between precise and imprecise homology directed repair
  • Understanding how target site selection can modulate the efficiency of precise and imprecise homology directed repair

7:30 CRISPR/Cas9 Genome Editing Tools: From Mouse Modeling to Potential Therapies

Ciro Bonetti, Ph.D., Scientist, Regeneron Pharmaceuticals 

  • Germline and Somatic gene modifications to generate mouse models to study disease
  • New tools to improve precise genome editing  
  • A new era for gene therapy (challenges and potential applications)

8:15 Q&A with attendees

8:30 End of Course

Speaker Biographies

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

Clifford J. Steer is a Professor of Medicine and Genetics, Cell Biology, and Development and a senior investigator at the University of Minnesota, Minneapolis, MN.  He has been active in the field of liver research for more than four decades.  In that capacity, he has been a long-standing member of several National Institutes of Health Study Sections.  He has been co-editor of a major scientific journal in liver diseases and presently serves on the editorial boards of three journals.  He has published his work in top-ranking scientific journals and numerous textbooks.  Steer’s areas of research over the last decade have focused on gene therapy, liver regeneration, neurodegenerative disorders and microRNA regulation of gene function.  He has published over 290 scientific articles; and has organized and chaired many national and international scientific conferences.  He was elected into the American Society for Clinical Investigation in 1991; and in 2014 was made an inaugural Fellow of the American Association for the Study of Liver Diseases.  His work has been written up in newspapers around the world, Time magazine, and in 1998 his work was featured in the Village Voice.

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

Widely recognized as a pioneer in the breakthrough technology, gene editing, Dr. Eric Kmiec is the Founder and Director of the Gene Editing Institute at the Helen F. Graham Cancer Center & Research Institute at Christiana Healthcare System with faculty appointments at the University of Delaware, the Wistar Institute and Georgetown University Medical School. His laboratory has been supported by the National Institute of Health in the form of R01 and R21 grants respectively for over 22 years. He has also received funding from the High Q Foundation, Hereditary Disease Foundation, the American Cancer Society, the March of Dimes, Pfizer, Bio-Rad, Tapestry Biotherapeutics and a variety of biotechnology companies. He serves on the editorial boards of many journals, has authored over 150 peer-reviewed journal publications as primary or senior author, numerous review articles and edited several books on gene therapy. Dr. Kmiec has 15 issued patents with 30 applications and has founded two biotechnology companies (Kimeragen, now Cibus in San Diego California, and Orphagenix LLC). He is currently a Senior Consultant and member of the Scientific Advisory Board of the gene editing company, ETAGEN, located in Cambridge, Massachusetts. From 1999-2008, Dr. Kmiec was a full, tenured Professor of Biology at the University of Delaware and Director of the Applied Genomics Laboratory at the Delaware Biotechnology Institute.

Ciro Bonetti, Ph.D., Scientist, Regeneron Pharmaceuticals

Ciro Bonetti is currently a Scientist at Regeneron Pharmaceuticals® in New York. He earned his PhD in Medical Genetics at TIGEM, Telethon Institute of Genetics and Medicine in Italy, where he applied and developed gene therapy approaches for recessive and dominant retinal congenital disorders. As American-Italian Cancer Foundation fellow, he moved for his postdoc to Memorial Sloan-Kettering Cancer Center in New York. During this time, he became an expert in a variety of genome editing tools including CRISPR/Cas9, which he has used to understand how non-coding genes affect normal physiology and impact human disease. He also adapted these tools to generate precise somatic mutations in terminally differentiated cells. This will impact not only our ability to create more precise animal models of human disease but will also pave the way to novel therapeutic opportunities.

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Oligonucleotide Discovery and Delivery