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    Oral Communication

    Hemophilia and Rare Bleeding Disorders
    Oral Communication Session

    OC 20 - Hemophilia Gene Therapy for Hemophilia A

    Sunday, June 25, 2023
    14:45 – 16:00 ET
    Room:517

    Session Available Online As: Live-Stream and On-Demand

    Presentations:

    • 14:45 – 15:00 ET
      Bleeding, FVIII activity, and safety 3 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

      Presenting Author: Johnny Mahlangu, MD (he/him/his) – Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa

      Co-Author: Annette von Drygalski, MD – University of California San Diego

      Co-Author: Susan Shapiro, MD, PhD (she/her/hers) – Oxford University Hospitals National Health Service Foundation Trust, Oxford, UK

      Co-Author: Sheng-Chieh Chou, MD. (he/him/his) – Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital

      Co-Author: Margareth C. Ozelo, MD, PhD (she/her/hers) – Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil

      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: Flora Peyvandi, MD, PhD (she/her/hers) – IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Italy

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

      Co-Author: Michael A. Laffan, MD – Centre for Haematology, Imperial College London, London, UK

      Co-Author: Amy L. Dunn – Oncology and Bone Marrow Transplant, Nationwide Children's Hospital and The Ohio State University College of Medicine, OH, USA

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

      Co-Author: Doris V. Quon, MD, PhD (she/her/hers) – Luskin Orthopaedic Institute for Children

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

      Co-Author: Johannes Oldenburg, MD (he/him/his) – University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany

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

      Co-Author: Mark T. Reding – Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, MN, USA

      Co-Author: Kala Jayaram – BioMarin Pharmaceutical Inc., Novato, CA, USA

      Co-Author: Hua Yu – BioMarin Pharmaceutical Inc., Novato, CA, USA

      Co-Author: Tara M. Robinson – BioMarin Pharmaceutical Inc., Novato, CA, USA

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

    • 15:00 – 15:15 ET
      Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 Trial

      Presenting Author: Mark W. Skinner, JD (he/him/his) – Institute for Policy Advancement Ltd.

      Co-Author: Er Chen – BioMarin Pharmaceuticals Inc.

      Co-Author: Quazi Ibrahim – Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON

      Co-Author: Alexandra Kucher – Patient Outcomes Research Group Ltd

      Co-Author: Federico Germini, MD, MSc, PhD – Department of Medicine, McMaster University, Hamilton, ON

      Co-Author: Milad Karimi – BioMarin Pharmaceuticals Inc.

      Co-Author: Elizabeth Clearfield – Institute for Policy Advancement Ltd.

      Co-Author: Brian O'Mahony – Irish Hemophilia Society

      Co-Author: Mohit Jain – BioMarin Pharmaceuticals Inc.

    • 15:15 – 15:30 ET
      Improved Joint Health After Gene Therapy with Dirloctocogene Samoparvovec (SPK-8011) in People with Hemophilia A

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

      Co-Author: Margaret V. Ragni, MD, MPH (she/her/hers) – Department of Medicine, University of Pittsburgh, Pittsburgh, PA, USA

      Co-Author: John E.J. Rasko – Department 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

      Co-Author: Kristina M. Haley – Department of Pediatrics, Oregon Health & Science University

      Co-Author: Stacy E. Croteau – Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children’s Hospital

      Co-Author: Jerome Teitel, MD – St Michael's Hospital Haemophilia Treatment Centre, University of Toronto

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

      Co-Author: Peter H. Cygan – Department of Medicine, Division of Blood and Vascular Disorders, Penn State Health Milton S. Hershey Medical Center

      Co-Author: Spencer Sullivan – Mississippi Center for Advanced Medicine

      Co-Author: Jill Moormeier – Department of Medicine, UMKC School of Medicine

      Co-Author: Savina Jaeger – Spark Therapeutics, Inc.

      Co-Author: Alex Long – Spark Therapeutics, Inc.

      Co-Author: Gallia Levy – Spark Therapeutics, Inc.

      Co-Author: Amy MacDougall – Spark Therapeutics, Inc.

      Co-Author: Kristen Jaworski – Spark Therapeutics, Inc.

      Co-Author: Tiffany Chang – Spark Therapeutics, Inc.

    • 15:30 – 15:45 ET
      The impact of gene therapy on the musculoskeletal health of patients with severe hemophilia A

      Presenting Author: Gabriela G. Yamaguti-Hayakawa, MD, PhD (she/her/hers) – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Janaina B. S. Ricciardi – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Jessica O. Frade-Guanaes, MsC – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Vanessa B. Faiotto – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Luiz G. R. R. De Lima – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Glenda Feldberg – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Emerson T. I. Sakuma – Department of Anesthesiology, Oncology and Radiology, School of Medical Sciences, University of Campinas, UNICAMP, Campinas, SP, Brazil

      Co-Author: Daniela M. De Paulo – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Andrea L. A. Sambo – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Mariana M. T. Hosokawa – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Valdirene C. F. Stahl – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Samuel S. Medina – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Marina P. P. Colella – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Nivia M. Foschi – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Carolina Costa-Lima, Master's degree and PHD student (she/her/hers) – Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil

      Co-Author: Margareth C. Ozelo, MD, PhD (she/her/hers) – Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil

    • 15:45 – 16:00 ET
      The WFH Gene Therapy Registry: A Collaborative Approach Towards a Global Resource for the Long-term Follow-up of People with Hemophilia Treated with Gene Therapy

      Presenting Author: Barbara A. Konkle, MD (she/her/hers) – University of Washington

      Co-Author: Donna Coffin, MSc Epid (she/her/hers) – World Federation of Hemophilia

      Co-Author: Mayss Naccache, MSc – World Federation of Hemophilia

      Co-Author: Toong Youttananukorn, PhD – World Federation of Hemophilia

      Co-Author: Cary Clark – International Society on Thrombosis and Haemostasis

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

      Co-Author: Alfonso Iorio – Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON

      Co-Author: Wolfgang Miesbach, MD – University Hospital Frankfurt, Frankfurt, Germany

      Co-Author: Declan Noone – European Haemophilia Consortium

      Co-Author: Flora Peyvandi, MD, PhD (she/her/hers) – IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Italy

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

      Co-Author: Michael Recht, MD, PhD, MBA (he/him/his) – National Hemophilia Foundation

      Co-Author: Mark W. Skinner, JD (he/him/his) – Institute for Policy Advancement Ltd.

      Co-Author: Leonard A. Valentino – Rush Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, Illinois, USA

      Co-Author: Johnny Mahlangu, MD (he/him/his) – Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa

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