Highlights From the ISTH 2021 Congress
Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results From the Phase 3 GENEr8-1 Trial
M.C Ozelo1, J. Mahlangu2, KJ. Pasi3, A. Giermasz4, A.D Leavitt5, M. Laffan6, E. Symington7, D.V Quon8, J.-D. Wang9, K. Peerlinck11, S. Pipe11, B. Madan12, N.S Key13, G.F Pierce14, B. O’Mahony15,16, R. Kaczmarek17,18, A. Lawal19, M. Huang19, W.Y. Wong19, B. Kim19, GENEr8-1 Trial Group
1Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil
2Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa
3Barts and the London School of Medicine and Dentistry, London, United Kingdom
4Hemophilia Treatment Center, University of California Davis, Sacramento, United States
5University of California, San Francisco, United States
6Centre for Haematology, Imperial College London, London, United Kingdom
7Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom
8Orthopaedic Hemophilia Treatment Center, Los Angeles, United States
9Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan, Province of China
10Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium
11Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, United States
12Guy's & St. Thomas’ NHS Foundation Trust, London, United Kingdom
13UNC Blood Research Center, University of North Carolina, Chapel Hill, United States
14Independent Consultant, La Jolla, United States
15Irish Haemophilia Society, Dublin, Ireland
16Trinity College, Dublin, Ireland
17Department of Pediatrics, Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, Indianapolis, United States
18Laboratory of Glycobiology, Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Poland
19BioMarin Pharmaceutical Inc., Novato, United States
M.C Ozelo1, J. Mahlangu2, KJ. Pasi3, A. Giermasz4, A.D Leavitt5, M. Laffan6, E. Symington7, D.V Quon8, J.-D. Wang9, K. Peerlinck11, S. Pipe11, B. Madan12, N.S Key13, G.F Pierce14, B. O’Mahony15,16, R. Kaczmarek17,18, A. Lawal19, M. Huang19, W.Y. Wong19, B. Kim19, GENEr8-1 Trial Group
1Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil
2Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa
3Barts and the London School of Medicine and Dentistry, London, United Kingdom
4Hemophilia Treatment Center, University of California Davis, Sacramento, United States
5University of California, San Francisco, United States
6Centre for Haematology, Imperial College London, London, United Kingdom
7Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom
8Orthopaedic Hemophilia Treatment Center, Los Angeles, United States
9Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan, Province of China
10Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium
11Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, United States
12Guy's & St. Thomas’ NHS Foundation Trust, London, United Kingdom
13UNC Blood Research Center, University of North Carolina, Chapel Hill, United States
14Independent Consultant, La Jolla, United States
15Irish Haemophilia Society, Dublin, Ireland
16Trinity College, Dublin, Ireland
17Department of Pediatrics, Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, Indianapolis, United States
18Laboratory of Glycobiology, Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Poland
19BioMarin Pharmaceutical Inc., Novato, United States