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Bethesda North Marriott Hotel and Conference Center

25 oct 2017 7:00 a.m. - 27 oct 2017 3:00 p.m.

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 3B: Understanding the Effects of 2’-MOE ASO Treatment on PLT Count in Non-Human Primates and Humans

Session Chair(s)

Scott  Henry, PhD

Scott Henry, PhD

Senior Vice President, Nonclinical Development

Ionis Pharmaceuticals, Inc., United States

This session will examine the overall experience with this class of oligonucleotides on PLT count in both monkeys and humans using a database comprised of multiple compounds. Comparisons will be made between monkeys and humans, include special patient populations that have recently reported servere thrombocytopenia. Background information on PLT biology and the current status investigation into the mechanism of potential PLT changes will be presented.

Learning Objective :
  • Understand the broad scope of PLT biology
  • Assess the experience on PLT changes observed in monkeys and humans treated with 2’-MOE ASO
  • Understand current hypothesized mechanisms for PLT changes observed in monkeys
  • Speaker(s)

    Ronald  Wange, PhD

    Session Co-Chair

    Ronald Wange, PhD

    FDA, United States

    Associate Director for Pharm/Tox OND

    Joseph  E. Italiano, PhD

    PLT Biology 101: Platelets and the Immune Continuum

    Joseph E. Italiano, PhD

    Brigham and Women’s Hospital, United States

    Director Cellular and Molecular Pathology, Division of Hematology

    Scott  Henry, PhD

    Characterizing the Nature and the Mechanism of PLT Changes Observed in Monkeys Treated with 2’ MOE ASO

    Scott Henry, PhD

    Ionis Pharmaceuticals, Inc., United States

    Senior Vice President, Nonclinical Development

    Richard Stephen Geary, PhD

    Human PLT Database for 2’-MOE ASO and Recent Expereince in FCS and TTR Phase 3 Trials

    Richard Stephen Geary, PhD

    Ionis Pharmaceuticals, Inc., United States

    Senior Vice President, Drug Development

      Panel Discussion

    Panel Discussion

    All Session Speakers, United States

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