OC 30 - Gene therapy - Long term follow up

Moderator(s)

• 

  Alok Srivastava

  Professor of Medicine
  Christian Medical College, Department of Haematology, Vellore, Tamil Nadu, India
  Vellore, Tamil Nadu, India
• 

  Natsaruth Songthawee, MD

  Assistant Professor
  Department of Pediatrics, Faculty of Medicine, Prince of Songkla University, Songkhla, Thailand
  Hat Yai, Songkhla, Thailand

Presentations:

• 09:30 – 09:45 ICT

  Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A
  

  Co-Author: Emily Symington – Cambridge University Hospitals NHS Foundation Trust

  Co-Author: Savita Rangarajan – Southampton University Hospital

  Co-Author: Will Lester – Univ Hospitals Birmingham NHS Foun Trust

  Presenting Author: Bella Madan, MD FRCP FRCPath – Guy's & St. Thomas' NHS Foundation Trust

  Co-Author: Glenn F. Pierce, MD PhD – World Federation of Hemophilia/Consultant

  Co-Author: Priyanka Raheja – Barts Health NHS Trust

  Co-Author: Carolyn M. Millar, MD PhD – Imperial College Healthcare NHS Trust & Imperial College London

  Co-Author: Dane Osmond – BioMarin Pharmaceuticals Inc.

  Co-Author: Mingjin Li – BioMarin Pharmaceuticals Inc.

  Co-Author: Tara M. Robinson – BioMarin Pharmaceuticals Inc.
• 09:45 – 10:00 ICT

  Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1
  

  Presenting Author: Andrew D. Leavitt, MD – Adult Hemophilia Treatment Center, University of California San Francisco

  Co-Author: Johnny Mahlangu, MBBCh, MMed – Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, and Charlotte Maxeke Johannesburg Academic Hospital

  Co-Author: Priyanka Raheja – Barts Health NHS Trust

  Co-Author: Emily Symington – Cambridge University Hospitals NHS Foundation Trust

  Co-Author: Doris V. Quon – Orthopaedic Hemophilia Treatment Center at Orthopaedic Institute for Children Los Angeles

  Co-Author: Adam Giermasz – Division of Hematology/Oncology, Hemophilia Treatment Center, University of California Davis

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

  Co-Author: Gillian Lowe – West Midlands Adult Haemophilia Comprehensive Care Centre, University Hospitals Birmingham NHS Foundation Trust

  Co-Author: Nigel S. Key – Division of Hematology, Department of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA

  Co-Author: Carolyn M. Millar, MD PhD – Imperial College Healthcare NHS Trust & Imperial College London

  Co-Author: Steven W. Pipe, MD – University of Michigan

  Co-Author: Bella Madan, MD FRCP FRCPath – Guy's & St. Thomas' NHS Foundation Trust

  Co-Author: Sheng-Chieh Chou, MD. – Department of Internal Medicine, National Taiwan University Hospital

  Co-Author: Robert Klamroth, MD – Vivantes Klinikum, Friedrichshain, Berlin, Germany

  Co-Author: Jane Mason – Queensland Haemophilia Centre, Cancer Care Services, Royal Brisbane and Women’s Hospital

  Co-Author: Hervé Chambost – AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN

  Co-Author: Flora Peyvandi, MD, PhD – Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Università degli Studi di Milano, Department of Pathophysiology and Transplantation

  Co-Author: Hua Yu – BioMarin Pharmaceutical Inc.

  Co-Author: Tara M. Robinson – BioMarin Pharmaceuticals Inc.

  Co-Author: Margareth C. Ozelo, MD, PhD – Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas
• 10:00 – 10:15 ICT

  Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec
  

  Presenting Author: Bella Madan, MD FRCP FRCPath – Guy's & St. Thomas' NHS Foundation Trust

  Co-Author: Margareth C. Ozelo, MD, PhD – Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas

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

  Co-Author: Sheng-Chieh Chou, MD. – Department of Internal Medicine, National Taiwan University Hospital

  Co-Author: Steven W. Pipe, MD – University of Michigan

  Co-Author: Andrew D. Leavitt, MD – Adult Hemophilia Treatment Center, University of California San Francisco

  Co-Author: Francisco P. Careta – Universidade Federal do Espírito Santo

  Co-Author: Patrick J. Lynch – Believe Ltd

  Co-Author: Simon Fletcher – Oxford University Hospitals NHS Trust

  Co-Author: Dor Goshen – Israeli Hemophilia Association

  Co-Author: Andres Ruiz – National Bleeding Disorders Foundation

  Co-Author: Ebony Dashiell-Aje – BioMarin Pharmaceutical Inc.

  Co-Author: Christine Rivat – BioMarin Pharmaceutical Inc.

  Co-Author: Johnny Mahlangu, MBBCh, MMed – Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, and Charlotte Maxeke Johannesburg Academic Hospital
• 10:15 – 10:30 ICT

  Sustained efficacy of gene transfer with BBM-H901 in Chinese Hemophilia B patients: results of long term follow up
  

  Presenting Author: Feng Xue, MD – State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin 300020, China

  Co-Author: xia wu – Belief BioMed, Xuhui District, Shanghai, China

  Co-Author: xiao xiao – Belief BioMed, Xuhui District, Shanghai, China

  Co-Author: Lei Zhang – State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin 300020, China
• 10:30 – 10:45 ICT

  Efficacy and safety of fidanacogene elaparvovec in adults with moderately severe or severe hemophilia B: updated results from the phase 3 BENEGENE-2 gene therapy trial
  

  Co-Author: Jiaan-Der Wang, PhD – Center for Rare Disease and Hemophilia, Department of Pediatrics, Taichung Veterans General Hospital

  Co-Author: Adam Cuker, MD, MS – Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

  Co-Author: Hazzaa Alzahrani – King Faisal Specialist Hospital and Research Centre Riyadh, Saudi Arabia

  Co-Author: Jan Astermark – Department of Translational Medicine, Lund University, Malmö, Sweden

  Co-Author: Laurent Frenzel – Institut Necker, Paris, France

  Co-Author: Olga Katsarou – Laiko Hospital, Blood Transfusion Centre, National Reference Centre for Congenital Bleeding Disorders, Athens, Greece

  Presenting Author: Kaan Kavakli, MD – Ege University Faculty of Medicine, Turkey

  Co-Author: Robert Klamroth, MD – Vivantes Klinikum, Friedrichshain, Berlin, Germany

  Co-Author: Davide Matino – Division of Hematology & Thromboembolism, Department of Medicine, McMaster University, Ontario, Canada

  Co-Author: Amy D Shapiro – Indianapolis, IN, USA

  Co-Author: Matco Kalac – Pfizer Inc.

  Co-Author: Joanne Fuiman – Pfizer Inc.

  Co-Author: John McKay – Pfizer Inc.

  Co-Author: Pengling Sun – Pfizer Inc.

  Co-Author: Jeremy Rupon – Pfizer Inc.

  Co-Author: Frank Plonski – Pfizer Inc