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Recording

View the recording below:

2024 International Prophylaxis Study Group (IPSG) Virtual Webinar was held on the 17th of January.

Gene Therapy: The Ultimate Prophylaxis

Itinerary

  1. Introduction & Overview of the current status of gene therapy in hemophilia

    Co-Chairs: Guy Young, MD, USA and David Lillicrap, MD, CA

  2. Pre-infusion issues

    Robert Klamroth, MD

    Head, Department of Internal Medicine, Vascular Medicine and Coagulation Disorders; Director, Comprehensive Care Hemophilia Treatment Center and the Hemostasis and Thrombosis Unit at the Vivantes Klinikum Friedrichshain Berlin, Germany

    Head, Department of Internal Medicine, Vascular Medicine and Coagulation Disorders; Director, Comprehensive Care Hemophilia Treatment Center and the Hemostasis and Thrombosis Unit at the Vivantes Klinikum Friedrichshain

  3. Post-infusion issues

    Johnny Mahlangu, MD

    Professor and Head, Department of Molecular Medicine and Hematology; Clinical Hematologist, Charlotte Maxeke Johannesburg Academic Hospital Faculty of Health Sciences, the University of the Witwatersrand and National Health Laboratory Service, Johannesburg Academic Hospital Johannesburg, South Africa

    Professor and Head, Department of Molecular Medicine and Hematology; Clinical Hematologist, Charlotte Maxeke Johannesburg Academic Hospital Faculty of Health Sciences, the University of the Witwatersrand and National Health Laboratory Service, Johannesburg Academic Hospital

  4. The Future of Gene Therapy in Hemophilia

    Lindsey A. George, M.D.

    Assistant Professor of Pediatrics, University of Pennsylvania School of Medicine; Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia; Attending Physician, Hematology, Children's Hospital of Philadelphia Philadelphia, USA

    Assistant Professor of Pediatrics, University of Pennsylvania School of Medicine; Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia; Attending Physician, Hematology, Children's Hospital of Philadelphia

  5. Panel Discussion

    Panelists: Robert Klamroth, Johnny Mahlangu, Lindsey A. George, Guy Young, and David Lillicrap

  6. Closing Remarks

    Guy Young and David Lillicrap

Meet Your Speakers

Dr.Guy Young

Co-Chair

Guy Young, MD

Guy Young, MD, received his medical degree from the State University of New York at Stony Brook School of Medicine in Stony Brook, New York. He completed a residency in pediatrics at Schneider Children’s Hospital at Long Island Jewish Medical Center, Albert Einstein College of Medicine in New York, and a fellowship in pediatric hematology/oncology at Children’s National Medical Center, George Washington University School of Medicine in Washington, D.C.

Dr. Young is the Director of the Hemostasis and Thrombosis Center at Children’s Hospital Los Angeles and an Associate Professor of Pediatrics at the University of Southern California’s Keck School of Medicine.

His current research activities encompass clinical trials in hemophilia, the use of novel anticoagulants in children, and the development of the global hemostasis assays to monitor the effects of medications used to treat bleeding in hemophilia.

Dr. Young has published many journal articles, abstracts, and textbook chapters and has delivered lectures, workshops, and presentations at national and international meetings. He is a past Board member and past chair of the program committee for the American Society of Pediatric Hematology/Oncology, and also a past chair of the program committee for the Hemophilia and Thrombosis Research Society. He is the former chair of the Scientific and Standardization Committee on Factor VIII, Factor IX, and Rare Bleeding Disorders of the International Society on Thrombosis and Haemostasis and also serves on the Scientific Subcommittee on Hemostasis for the American Society of Hematology. He has been awarded the National Hemophilia Foundation Physician of the Year Award in 2013.

David Lillicrap, MD

Speaker

David Lillicrap, MD

David Lillicrap is a Professor in the Department of Pathology and Molecular Medicine at Queen’s University, Kingston, Canada. He is the recipient of a Senior Canada Research Chair in Molecular Hemostasis. Dr. Lillicrap is a past member of the World Federation of Hemophilia’s (WFH) Medical Advisory Board and Chair of the WFH’s Research Committee. He is a past Chairman of the International Society on Thrombosis and Haemostasis’ (ISTH) Scientific and Standardization Committee and prior member of the Council of ISTH. He has previously served as an Associate Editor for Blood, and recently completed a 5-year appointment as Co-Editor-in-Chief of the Journal of Thrombosis and Haemostasis.

Robert Klamroth, MD

Speaker

Robert Klamroth, MD

Priv. Doz. Dr. med. Robert Klamroth is Director and Head of Department for Internal medicine (2014-), Vascular medicine and Coagulation disorders in Berlin, Director of Haemophilia treatment centre for Children and Adults Hospital im Friedrichshain (2002-), Berlin and Adjunctive professor at Institute of Experimental Haematology and Transfusion Medicine (2017-) in Bonn, Germany.

Dr. Klamroth obtained his medical degree in 1991 at Free university Berlin. He holds specialist training degrees in Internal Medicine (2001), and Hemostaseology (Coagulation Disorders, 2007) and Angiology (2014).

In 2017 he completed his habilitation in Hemostaseology at University of Bonn and obtained title Privatdozent (Priv.Doz.).

Dr. Klamroth is a member of national and international societies in the field of angiology, thrombosis and haemostasis He is chairman of the Society for Thrombosis and Haemostasis Research (GTH) and the European Association for Haemophilia and Allied Disorders (EAHAD) Dr. Klamroth has authored or co-authored peer-reviewed numerous publications, review articles and book chapters in the field.

Dr Klamroth initiated, realised, and participated in numerous clinical trials (phases I-IV) for new products for haemophilia and new antithrombotic agents as investigator.

Johnny Mahlangu, MD

Speaker

Johnny Mahlangu, MD

Johnny Mahlangu is a Professor and Academic Head of the Department of Molecular Medicine and Haematology in the Faculty of Health Sciences of the University of the Witwatersrand and the National Health Laboratory Service. He is also a Consultant Clinical Haematologist at the Charlotte Maxeke Johannesburg Academic Hospital. Johnny received his undergraduate and postgraduate training in science and medicine at the University of the Witwatersrand with haematology specialist and clinical haematology sub-specialist qualifications through the Colleges of Medicine of South Africa. His main area of research is novel therapies in bleeding disorders, in which he has served as Principal Investigator for a large number of international multicentre studies. He has published many peer review journal articles and presented more than 500 oral talks and posters at national and international scientific meetings. His academic citizenship includes membership of many national and international Scientific Committees.

Lindsey George, MD

Speaker

Lindsey George, MD

Dr. Lindsey George is an Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and Director of Clinical In Vivo Gene Therapy and Attending Hematologist at the Children’s Hospital of Philadelphia. Her laboratory studies the molecular basis of coagulation with a focus on mechanisms that regulate factor VIII/VIIIa cofactor function to improve understanding of associated disease states of deficiency (hemophilia A) or excess function (thrombosis). Ongoing studies in her group aim to exploit their biochemical insights of FVIII function to develop novel gene-based therapies for hemophilia A. Dr. George has led early-phase, adeno-associated virus (AAV) mediated gene transfer trials for hemophilia A and B. Her Clinical In Vivo Gene Therapy group conducts and supports translational and clinical research in in vivo gene therapy as well as aims to implement licensed AAV vectors safely and efficiently across the Children’s Hospital of Philadelphia. Dr. George is a member of the Board of Directors of the American Society of Gene and Cell Therapy and participates in multiple and international professional societies for hemostasis and gene therapy research.