Program

Tuesday, September 5

  • General Session
    Registration
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    17:00
  • General Session
    Keynote: Modifying Genomic DNA and RNA
    Gene Yeo, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    18:00
  • Social Program
    Opening Reception
    The Green Room in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    19:30

Wednesday, September 6

  • General Session
    Therapeutic Targeting of RNA
    C. Frank Bennett, Ph.D., Beverly L. Davidson, Ph.D., Mario Gomes-Pereira, Ph.D., Adrian Krainer, Ph.D., Stuart W. Peltz, Ph.D., Maurice Swanson, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • 8:30 - 10:30
      ASOs Modification of RNA Splicing (Adrian Krainer, Ph.D., professor, Cold Spring Harbor Lab)
    • 8:30 - 10:30
      Mechanisms and Therapies in the CAG-repeat Diseases (Beverly L. Davidson, Ph.D., professor, University of Pennsylvania)
    • 8:30 - 10:30
      Small Molecule Compounds Modifying RNA (Stuart W. Peltz, Ph.D., CEO, PTC Therapeutics)
    • 8:30 - 10:30
      Optimization of RNase H-Dependent Antisense Drugs for the Treatment of Myotonic Dystrophy (C. Frank Bennett, Ph.D., senior vice president of research, Ionis Pharmaceuticals)
    10:30
  • 10:30
    Coffee Break - Green Room
  • General Session
    Epigenetic Mechanisms in Congenital Myotonic Dystrophy
    Christopher Pearson, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    11:30
  • General Session
    DM Session 1: Microsatellite Instability and Epigenetics
    John Cleary, Ph.D., Darren Monckton, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • This session covers all aspects of the mutations associated with DM, including population genetics, particularities of the mutations, epigenetic modifications and repeat instability mechanisms.
    12:30
  • Poster Session
    Flash Poster Session
    Christopher Pearson, Ph.D., Derick Wansink, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    13:00
  • Poster Session
    Poster Viewing
    The 4th Floor Bryan Education Studio in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    14:30
  • Lunch
    Lunch
    The Green Room in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    14:30
  • General Session
    DM Session 2: Disease Mechanisms DM1, DM2 and CDM
    Thomas Cooper, M.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • This session covers the research devoted to understanding DM disease mechanisms in various model systems, with the exception of repeat instability mechanisms covered in Session 1.
    17:00

Thursday, September 7

  • General Session
    Pathogenic Mechanisms of C9orf72 Repeat Expansion
    Laura Ranum, Ph.D., Benedikt Schoser, M.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    9:00
  • General Session
    DM Session 3: Cancer and Aging in DM
    Nicolas Sergeant, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • This session covers preclinical and clinical studies of the underlying mechanisms and progression of cancer and premature aging in DM.
    10:00
  • 10:00
    Coffee Break - Green Room
  • General Session
    DM Session 4: Clinical, Ethical and Social Issues, and Disease Burden
    Cynthia Gagnon, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • This session covers all clinical aspects of DM and its burden on patients and caregivers, as well as ethical and social issues in DM patients and families.
    12:00
  • Poster Session
    Flash Poster Session
    Guillaume Bassez, M.D., Bruno Eymard, M.D., Giovanni Meola, M.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    12:30
  • Poster Session
    Poster Viewing
    The 4th Floor Bryan Education Studio in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    14:00
  • Lunch
    Lunch
    The Green Room in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    14:00
  • General Session
    DM Session 5: Specific Disease Features: CNS, Cardiac and GI
    Chad Heatwole, M.D., Ami Mankodi, M.D., Nicolas Sergeant, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • This session covers organ-specific phenotypes of DM and practices for their management and treatment.
    16:30
  • General Session
    Organization and Outreach Update
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    17:00

Friday, September 8

  • General Session
    CNS and Sleep
    John Day, M.D., Ph.D., Emmanuel Mignot, M.D., Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    9:30
  • General Session
    DM Session 6: Cell Models for DM
    David Brook, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • This session covers in vitro model systems, their development and use in mechanistic studies or therapy development.

    10:30
  • 10:30
    Coffee Break - Green Room
  • General Session
    DM Session 7: Animal Models and Tissue-specific Mechanisms
    Tetsuo Ashizawa, M.D., Bé Wieringa, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • This session covers in vivo model systems, their development and use in mechanistic studies or therapy development.
    12:00
  • Poster Session
    Flash Poster Session
    Guillaume Bassez, M.D., Ralf Krahe, Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    12:30
  • Lunch
    Lunch
    The Green Room in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    13:30
  • Poster Session
    Poster Viewing
    The 4th Floor Bryan Education Studio in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    13:30
  • General Session
    Neuromuscular Clinical Research Experience
    Richard Finkel, M.D., Laurence Mignon, Ph.D., Francesco Muntoni, M.D., John Porter, Ph.D., Jacinda Sampson, M.D., Ph.D., S. H. Subramony, M.D., Charles Thornton, M.D., Baziel Van Engelen M.D., Ph.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • 16:30 - 17:30
      Trial Design and Clinical Trial Results for SMA (Richard Finkel, M.D.)
    • 16:30 - 17:30
      Infrastructure Considerations for DMD Clinical Trial Readiness (Francesco Muntoni, M.D., invited)
    • 16:30 - 17:30
      Myotonic Dystrophy Clinical Research Network and Natural History Studies (Charles Thornton, M.D.)
    • 16:30 - 17:30
      Infrastructure Considerations and Lessons from the OPTIMISTIC Study in DM (Baziel Van Engelen, M.D., Ph.D.)
    • 16:30 - 17:30
      Lessons from DMPK-Rx clinical trials in myotonic dystrophy (Laurence Mignon, Ph.D.)
    16:30
  • General Session
    DM Session 8: RNA Clinical Research Methods: Biomarkers, Outcome Measures, Registries, Trial Design and Therapeutics
    Jacinda Sampson, M.D., Ph.D., S. Subramony, M.D.
    The Herbst Theatre in the San Francisco War Memorial
    401 Van Ness Ave, San Francisco, CA 94102
    Click for Details
    • 16:30 - 17:30
      This session covers clinical trial readiness resources and tools used in the clinical development of new therapeutic strategies.
    17:30
  • Social Program
    Closing Dinner
    Sens Restaurant
    4 Embarcadero Center, San Francisco, CA 94111
    Click for Details
    • Note: There is a bridge walkway directly from the Hyatt Regency San Francisco to the Sens Restaurant in the Embarcadero Center. Attire: Business Casual.
    21:30

Saturday, September 9

  • General Session
    Drug Development Roundtable
    Hyatt Regency San Francisco
    5 Embarcadero Center, San Francisco, CA 94111
    Click for Details
    • The annual Drug Development Roundtable brings together leaders from academia, biotechnology and pharmaceutical industries to discuss opportunities, barriers and pre-competitive space collaborations to facilitate the pathway to drugs and biologics for DM.
    9:00
  • General Session
    Breakfast and MDF Research Update
    John Porter, Ph.D.
    Hyatt Regency San Francisco
    5 Embarcadero Center, San Francisco, CA 94111
    9:00
  • Social Program
    IDMC Awards
    Hyatt Regency San Francisco
    5 Embarcadero Center, San Francisco, CA 94111
    9:45
  • 9:45
    Coffee Break
  • General Session
    Bringing the Patient Voice to CNS-Targeting Drug Development in Myotonic Dystrophy
    Hyatt Regency San Francisco
    5 Embarcadero Center, San Francisco, CA 94111
    Click for Details
    • In response to requests from drug development professionals to help identify CNS endpoints appropriate for the regulatory review process, and to accelerate the development of CNS-targeting DM therapies, IDMC-11 and the Myotonic Dystrophy Foundation will jointly host a multi-panel discussion to bring patient perspectives on disease burden and clinically meaningful endpoints to efforts to develop endpoints for CNS-targeting DM therapies. The meeting format will mirror the very successful Patient-Focused Drug Development (PFDD) meeting hosted by MDF with the FDA in September 2016. Panels will be seated with people affected by DM, and DM caregivers. The session will be moderated by James Valentine, J.D., who also moderated the September 2016 PFDD meeting. The session will begin with a presentation on outcome measure development to help the lay audience understand the importance and role of endpoints in drug development and approval, and to frame the goals for the session. Panel presentations will be followed by moderated audience discussion and a presentation from an industry representative regarding possible future DM CNS endpoints, and CNS endpoints used in other neurological diseases that might inform upcoming drug development efforts in DM. A publication summarizing the workshop is planned.
    12:00
  • Lunch
    Bringing the Patient Voice to CNS - Facilitated Lunch Discussion and Report Out
    Hyatt Regency San Francisco
    5 Embarcadero Center, San Francisco, CA 94111
    13:00
  • General Session
    Grant Writing Training
    Thomas Cheever, Ph.D., Glen Nuckolls, Ph.D.
    Hyatt Regency San Francisco
    5 Embarcadero Center, San Francisco, CA 94111
    13:45
  • General Session
    Expanding the Pipeline: Industry Updates on Drug Development
    Matthew Disney, Ph.D., Joseph Horrigan, M.D., Laurence Mignon, Ph.D.
    The Ballroom of the Hyatt Regency San Francisco
    5 Embarcadero Center, San Francisco, CA 94111
    Click for Details
    • 15:30 - 17:00
      Representatives from companies and academia will provide updates on early stage and advanced drug development efforts in the DM field.
    17:00