OC 08 - Von Willebrand Disease and Thrombotic Thrombocytopenic Purpura (TTP) Novel Treatments

Moderator(s)

• 

  Paula James, MD

  Professor
  Medicine
  Department of Medicine, Queen's University, Kingston, ON, Canada
  Kingston, Ontario, Canada
• 

  Dearbhla Doherty, MD (she/her/hers)

  Clinical Research Fellow
  School of Pharmacy and Biomolecular Sciences
  Irish Centre for Vascular Biology, School of Pharmacy and Biomolecular Sciences, Royal College of Surgeons in Ireland, Dublin, Ireland
  Dublin 2, Dublin, Ireland

Presentations:

• 13:00 – 13:15 ET

  GC1126A, a novel ADAMTS13 mutein that evades autoantibody as a superior therapy for acquired thrombotic thrombocytopenic purpura (aTTP)
  

  Presenting Author: Hyun-Ja Nam, PhD – GC Biopharma

  Co-Author: Heechun Kwak – GC Biopharma

  Co-Author: Gahee Choi – GC Biopharma

  Co-Author: Suyong Kim – GC Biopharma

  Co-Author: Ji-Min Park – GC Biopharma

  Co-Author: Young Eun Kwon – GC Biopharma

  Co-Author: Yongmin Lee – GC Biopharma

  Co-Author: Chae Mok Lee – GC Biopharma
• 13:15 – 13:30 ET

  High-Density Lipoprotein prevents Microvascular Thrombosis in two Mouse Models of Thrombotic Microangiopathy
  

  Presenting Author: Reheman Adili, MD – Bloodworks Research Institute

  Co-Author: Nicole Rhoads – Bloodworks Research Institute

  Co-Author: Jeff Harris – Bloodworks Research Institute

  Co-Author: Junmei Chen, PhD – Bloodworks Research Institute

  Co-Author: José López – Bloodworks Research Institute

  Co-Author: Dominic Chung – Bloodworks Research Institute
• 13:30 – 13:45 ET

  KB-V13A12, a novel nanobody-based therapeutic molecule for the treatment of von Willebrand disease
  

  Presenting Author: Ivan Peyron, PhD (he/him/his) – Laboratory for Hemostasis, Inflammation and Thrombosis, Unité Mixte de Recherche S1176, Institut National de la Santé et de la Recherche Médicale, Université Paris-Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Caterina Casari, PhD (she/her/hers) – Laboratory for Hemostasis, Inflammation and Thrombosis, Unité Mixte de Recherche S1176, Institut National de la Santé et de la Recherche Médicale, Université Paris-Saclay

  Co-Author: Olivier D. Christophe – Laboratory for Hemostasis, Inflammation and Thrombosis, Unité Mixte de Recherche S1176, Institut National de la Santé et de la Recherche Médicale, Université Paris-Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Peter J. Lenting, PhD – Laboratory for Hemostasis, Inflammation and Thrombosis, Unité Mixte de Recherche S1176, Institut National de la Santé et de la Recherche Médicale, Université Paris-Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Cécile V. Denis, PhD – Laboratory for Hemostasis, Inflammation and Thrombosis, Unité Mixte de Recherche S1176, Institut National de la Santé et de la Recherche Médicale, Université Paris-Saclay, Le Kremlin-Bicêtre, France
• 13:45 – 14:00 ET

  Aptamer BT200 prolongs VWF half-life by blocking interaction with macrophage scavenger receptor LRP1
  

  Presenting Author: Alain Chion, PhD – RCSI

  Co-Author: Sonia Aguila, PhD (she/her/hers) – Centro Regional de Hemodonación, IMIB-Pascual Parrilla, Murcia, Spain

  Co-Author: Judicael Fazavana – RCSI

  Co-Author: Ciara Byrne – RCSI

  Co-Author: Soracha Ward – RCSI

  Co-Author: Jamie O'Sullivan, PhD (she/her/hers) – Royal College of Surgeons in Ireland, Irish Centre for Vascular Biology, Dublin, Ireland

  Co-Author: Thomas McKinnon, BSc, PhD – Imperial College London

  Co-Author: Shuhao Zhu – Band Therapeutics

  Co-Author: Jim Gilbert – Band Therapeutics

  Co-Author: Bernd Jilma – department of Clinical Pharmacology

  Co-Author: James S. O'Donnell, MB, PhD – Irish Centre for Vascular Biology, Royal College of Surgeons in Ireland,Dublin, Ireland
• 14:00 – 14:15 ET

  Allele-selective inhibition of mutant von Willebrand factor with small interfering RNAs to ameliorate a von Willebrand disease type 2B phenotype in vivo in mice
  

  Presenting Author: Noa A. Linthorst, MSc – Department of Internal Medicine, Division of Thrombosis and Hemostasis, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands.

  Co-Author: Yvonne K. Jongejan, MSc – Department of Internal Medicine, Division of Thrombosis and Hemostasis, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands.

  Co-Author: Ruben Bierings, PhD – Department of Hematology, Erasmus MC, University Medical Center Rotterdam, the Netherlands

  Co-Author: James E. Dahlman – Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine, Atlanta, Georgia, United States.

  Co-Author: Cécile V. Denis, PhD – Laboratory for Hemostasis, Inflammation and Thrombosis, Unité Mixte de Recherche S1176, Institut National de la Santé et de la Recherche Médicale, Université Paris-Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Frank W.G. Leebeek, MD, PhD – Erasmus MC, University Medical Center Rotterdam, The Netherlands

  Co-Author: Elisa Schrader – Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine, Atlanta, Georgia, United States.

  Co-Author: Jan Voorberg – Department of Molecular Hematology, Sanquin Research and Landsteiner Laboratory

  Co-Author: Bart J.M. van Vlijmen – Department of Internal Medicine, Division of Thrombosis and Hemostasis, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands.

  Co-Author: Jeroen C.J Eikenboom, MD. PhD – Leiden University Medical Center