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Short Talks - Industry Bleeding - No CME
Moderators
Julie Jaffray, Johnathan Morris
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Presentations
10:30 IMPROVED JOINT HEALTH AFTER GENE THERAPY WITH DIRLOCTOCOGENE SAMOPARVOVEC (SPK-8011) IN PEOPLE WITH HEMOPHILIA A
Benjamin Samelson-Jones1, Peter Cygan2, Stacy Croteau3, Huyen Tran4, Margaret Ragni5, Jerome Teitel6, John Rasko 7, Spencer Sullivan 8, Jill Moormeier9, Kristina Haley10, Kristen Jaworski11, Amy MacDougall11, Alexander Long 11, Savina Jaeger 11, Tiffany Chang11, Gallia Levy11. 1Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia, Philadelphia, PA, USA 2Department of Medicine, Division of Blood and Vascular Disorders, Penn State Health Milton S. Hershey Medical Center, Hershey, PA, USA 3Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children’s Hospital, Boston, MA, USA 4Australian Centre for Blood Diseases, Monash University, Melbourne, Australia 5Department of Medicine, University of Pittsburgh, Pittsburgh, PA, USA 6St Michael's Hospital Haemophilia Treatment Centre, University of Toronto, Toronto, ON, Canada 7Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney, Camperdown, Australia 8Mississippi Center for Advanced Medicine, Madison, MS, USA 9Department of Medicine, UMKC School of Medicine, Kansas City, MO, USA 10Department of Pediatrics, Oregon Health & Science University, Portland, OR, USA 11Spark Therapeutics, Inc., Philadelphia, PA, USA
10:45 PROPHYLACTIC TREATMENT OF ACQUIRED VON WILLEBRAND DISEASE IN NON-HUMAN PRIMATES WITH VGA039, AN ANTI-PROTEIN S MONOCLONAL ANTIBODY
Benjamin Kim, Ian Huck, Lilley Leong, Tony Byun, Aline He, Abel Silva, Joanne Chan, Young Cho, Stephen J. Moore, Graham Parry, Sandip Panicker. Vega Therapeutics, Inc., South San Francisco, CA, USA
11:00 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY
Paul Coppo1, Parth Patwari2, Björn Mellgård2, Hong Li2, Marie Scully3, Masanori Matsumoto4, Jerzy Windyga5, Thomas L Ortel6, Spero Cataland7, Paul Knöbl8, Ziqiang Yu9, Ana Antun10, Sami Ibrahimi11, Doug Criger2, Linda T Wang2. 1APHP.6–Reference Center for Thrombotic Microangiopathies (CNR-MAT), Hôpital St Antoine, Paris, France 2Takeda Development Center Americas, Inc., Cambridge, MA, USA 3University College London Hospitals NHS Foundation Trust, London, United Kingdom 4Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Nara, Japan 5Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland 6Duke University, Durham, NC, USA 7Department of Internal Medicine, Ohio State University, Columbus, OH, USA 8Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna, Austria 9National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China 10Department of Hematology and Medical Oncology, School of Medicine, Emory University, Atlanta, GA, USA 11Department of Internal Medicine, Section of Hematology and Oncology, University of Oklahoma Health Science Center, Oklahoma City, OK, USA
11:15 INITIAL REPORT OF PART B PHASE 1/2 EFFICACY AND SAFETY RESULTS FOR BRUTON TYROSINE KINASE INHIBITOR RILZABRUTINIB IN PATIENTS WITH RELAPSED IMMUNE THROMBOCYTOPENIA
Nichola Cooper1, A. J. Gerard Jansen2, Jiri Mayer3, Michael D Tarantino4, Remco Diab5, Brad Ward6, Ahmed Daak6, David J Kuter7. 1Hammersmith Hospital, London, United Kingdom 2Erasmus MC, University Medical Center, Rotterdam, Netherlands 3Masaryk University Hospital, Brno, Czech Republic 4The Bleeding and Clotting Disorders Institute, University of Illinois College of Medicine-Peoria, Peoria, IL, USA 5Sanofi, Rotkreuz, LU, Switzerland 6Sanofi, Cambridge, MA, USA 7Hematology Division, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA