OC 50 - Non-Replacement Therapies III

Moderator(s)

• 

  Saicharan Akula

  PhD student
  Department of health sciences,
  universita del piemonte orientale
  Novara, Piemonte, Italy
• 

  Jerzy Windyga, MD, PhD

  Professor
  Hemostasis Disorders and Internal Medicine
  Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
  Warsaw, Mazowieckie, Poland

Presentations:

• 10:45 AM – 11:00 AM

  Transplantation of fetal liver and adult bone marrow cells for phenotypic correction of hemophilia A.
  

  Presenting Author: Saicharan Akula – universita del piemonte orientale

  Co-Author: Simone merlin – universita del piemonte orientale

  Co-Author: Alessia Cottonaro – universita del piemonte orientale

  Co-Author: Antonia Follenzi, MD, PhD – University of Piemonte Orientale, Novara, Italy

  Co-Author: Maria Jose sanchez – Centro Andaluz de Biología del Desarrollo (CABD)

  Co-Author: ester borroni – universita del piemonte orientale

  Co-Author: vakhtang Kalandadze – universita del piemonte orientale

  Co-Author: Tamara garcia leal – Centro Andaluz de Biología del Desarrollo (CABD)

  Co-Author: Luis J. Serrano ramos – Complutense University of Madrid

  Co-Author: antonio liras – Complutense University of Madrid

  Co-Author: silvia buzzi – universita del piemonte orientale
• 11:00 AM – 11:15 AM

  Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin: Analysis of Antithrombin Levels and Thrombin Generation from a Phase 3 Study in People with Hemophilia A or B Without inhibitors
  

  Presenting Author: Steven W. Pipe, MD – University of Michigan, Ann Arbor, MI, USA

  Co-Author: Alok Srivastava, MD, FRACP, FRCPA, FRCP – Christian Medical College, Vellore, India

  Co-Author: Robert Klamroth, MD, PhD – Vivantes Klinikum im Friedrichshain, Clinic for Internal Medicine Angiology and Haemostaseology

  Co-Author: Gili Kenet, MD – The National Hemophilia Center, and Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Hashomer, Tel Aviv University, Tel Aviv, Israel

  Co-Author: Huyen Tran – Australian Centre for Blood Diseases, Monash University

  Co-Author: Lila-Sabrina Fetita – Sanofi Genzyme, Paris, France

  Co-Author: Zhiying Qiu – Sanofi, Bridgewater, NJ, USA

  Co-Author: Shauna Andersson – Sanofi

  Co-Author: Baisong Mei – Sanofi, Cambridge, MA, USA

  Co-Author: Savita Rangarajan, MD, FRCP, FRCPath – Faculty of Medicine, University of Southampton, Southampton, UK
• 11:15 AM – 11:30 AM

  Improved Procoagulant Activity of Hemophilia B Causing Dysfunctional Factor IX Variants with Factor VIII Mimetics
  

  Co-Author: Julia Chau – The Children's Hospital of Philadelphia

  Co-Author: Anna R. Sternberg, PhD – Children's Hospital of Philadelphia

  Co-Author: Allyson Pishko – University of Pennsylvania

  Co-Author: Lindsey A. George – University of Pennsylvania

  Presenting Author: Benjamin J. Samelson-Jones, PhD – Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia
• 11:30 AM – 11:45 AM

  Differences in bleeding profiles and venous clot structures between emicizumab and factor VIII-treated mice
  

  Presenting Author: Thibaud SEFIANE – UMR_S 1176 INSERM, Le Kremlin-Bicêtre, France

  Co-Author: Vincent Muczynski – INSERM U1176

  Co-Author: Xavier Heiligenstein – Cryocapcell

  Co-Author: Olivier Christophe – INSERM U1176

  Co-Author: Cecile Denis – INSERM U1176

  Co-Author: Caterina Casari, PhD – Inserm U1176 - HITh

  Co-Author: Peter J. Lenting, PhD – INSERM U1176
• 11:45 AM – 12:00 PM

  Mim8 is associated with improved thrombin generation vs. emicizumab in patients with haemophilia A, with and without inhibitors
  

  Presenting Author: Jerzy Windyga, MD, PhD – Institute of Hematology and Transfusion Medicine

  Co-Author: Pratima Chowdary, MD – Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital

  Co-Author: Francisco Lopez Jaime, MD – Hospital Universitario Regional de Málaga

  Co-Author: Christian Winther Eskelund – Novo Nordisk A/S

  Co-Author: Pernille Juul Jørgensen – Novo Nordisk A/S

  Co-Author: Mads Kreilgaard – Novo Nordisk A/S

  Co-Author: Johnny Mahlangu, MD, MMed – University of the Witwatersrand and NHLS