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Bethesda North Marriott Hotel and Conference Center

2017 年 10 月 25 日 7:00 上午 - 2017 年 10 月 27 日 3:00 下午

5701 Marinelli Road, , North Bethesda, MD 20852 , USA

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Convening industry and health authorities to inform, educate, and share advancements in oligonucleotide-based therapeutic product development.

Session 10: Hot Topics in Oligonucleotide Therapeutics

Session Chair(s)

Arthur A. Levin, PhD

Arthur A. Levin, PhD

Distinguished Scientist

Avidity Biosciences, United States

As we learn more about RNA biology, its importance in the regulation of gene expression, and its role in disease processes, it is becoming clear there are many more ways to use oligonucleotides as therapeutic agents. With the identification of novel exploitable mechanism of RNA modulation, the range of diseases that can be targeted increases. This session will explore multiple novel ways to modulate RNA biology that may ultimately be used as a way to address disease processes and become the foundation for new therapeutic agents.

Learning Objective :
  • Integrate novel mecahanisms of RNA processing into new areas for therapeutic agents
  • Appreciate the complexities of RNA biology and how it can be used for therapeutic activity, and how manipulating RNA biology can produce phenotypic changes
  • Speaker(s)

    Shwu-Luan  Lee, PhD

    Session Co-Chair

    Shwu-Luan Lee, PhD

    FDA, United States

    Pharmacologist, Office of New Drug, CDER

    Huw M. Nash, PhD

    Antisense-Mediated Control of RNA Splicing to Treat Monogenic Diseases

    Huw M. Nash, PhD

    Stoke Therapeutics Inc., United States

    Chief Executive Officer

    Joshua  Rosenthal, PhD

    A to I Editing and Neuronal Plasticity

    Joshua Rosenthal, PhD

    Marine Biological Laboratory, United States

    Senior Scientist

    Maria  Montiel-Gonzalez, PhD

    A Site-Directed RNA Editing Strategy to Correct Genetic Mutations

    Maria Montiel-Gonzalez, PhD

    The Marine Biological Laboratory, University of Chicago, United States

    Post Doctoral Fellow

    Troels  Koch, PhD, MSc

    Stereodefined LNA Phosphorothioates: A New Perspective in RNA Therapeutics

    Troels Koch, PhD, MSc

    Roche Innovation Center Copenhagen, Denmark

    VP, Head of Research, RNA Therapeutics

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