Le webinaire de la Société canadienne de l'hémophilie a abordé la thérapie génique pour les hémophiles. Dr. Jean St. Louis a expliqué les différences entre les injections de facteurs et la thérapie génique, en utilisant des vecteurs AAV pour introduire des gènes dans les cellules du foie. Il a détaillé les thérapies géniques approuvées par Santé Canada : MGX et Bec Vibes, soulignant leurs différences de dose et de taux d'échec. Les données à long terme montrent une réduction significative des saignements et une stabilité des niveaux de facteurs. Dr. David Page a discuté des défis de remboursement et de l'accès au traitement au Québec.

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The Canadian Hemophilia Society's December 4, 2024 webinar on hemophilia gene therapy featured the latest research and access updates. Dr. Roy Khalifé described how AAV vector gene therapy works in hemophilia A and B. He reported the latest clinical trial results for of Roctavian (hemophilia A) and Hemgenix and Beqvez (hemophilia B), noting annualized bleeding rates and levels of factor VIII and IX expression. Safety concerns included liver enzyme elevations and the need for short-term corticosteroids. David Page addressed regulatory hurdles, noting Health Canada's approvals of Hemgenix and Beqvez, CDA's positive reimbursement recommendations, but INESSS' negative reimbursement recommendations for Quebec. The discussion emphasized the need for long-term monitoring and equitable access across Canada.

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In this podcast, Elizabeth Vansant, Mark Pervan, Murray Milne and Mathieu Jackson, all individuals with severe hemophilia B, are asked to debate the question: “If gene therapy for hemophilia B were available in Canada tomorrow, would you want to receive it?”

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In this podcast, Cleaven Pagani, Rick Waines, Paul Wilton and Emil Wijnker, all with severe hemophilia A and also members of the Canadian Hemophilia Society Blood Safety and Supply Committee, are asked to debate the question: “If gene therapy for hemophilia A were available in Canada tomorrow, would you want to receive it?”

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The first generation of gene therapy for hemophilia, called gene insertion or gene addition, is now a reality, but it is not for everyone. Dr. Glenn Pierce explores other approaches to gene therapy in the research pipeline that in the future may be more efficacious for a longer time for more people. Glenn is a long-time researcher in coagulation products for hemophilia and is currently the Medical Vice President of the World Federation of Hemophilia.

Canadian Hemophilia Society

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In this podcast, Dr. Jerry Teitel explores how gene therapy for hemophilia A and hemophilia B might be delivered to patients across Canada in an equitable way, even though the administration will occur in only some sites. He describes the follow-up that is required in the critical months following administration to get the best results possible. Dr. Teitel is a Professor of Medicine at the University of Toronto, and Medical Director of the Hemophilia Treatment Program at St. Michael’s Hospital in Toronto, Ontario.

Canadian Hemophilia Society

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Gene therapy—a high-cost, one-time, potentially life-changing treatment—presents unique challenges for governments that make decisions on funding innovative treatments. Dr. Durhane Wong-Rieger presents creative ideas on new ways to think about how gene therapy for hemophilia A and B could be funded in Canada. Durhane is a health coach, frequent lecturer and author. She is the President and CEO of the Canadian Organization for Rare Diseases and chair of Rare Diseases International.

Canadian Hemophilia Society

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In this podcast, Dr. Jerry Teitel describes the process of shared decision-making prior to gene therapy in hemophilia A and hemophilia B, and how the nature of gene therapy itself makes shared decision-making so important. Dr. Teitel is a Professor of Medicine at the University of Toronto, and Medical Director of the Hemophilia Treatment Program at St. Michael’s Hospital in Toronto, Ontario.

Canadian Hemophilia Society

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In this podcast, Dr. Glenn Pierce explores what we know we don’t know and what we don’t even know we don’t know about the brand-new treatment which is gene therapy for hemophilia A and B. Glenn is a long-time researcher in coagulation products for hemophilia and is currently the Medical Vice President of the World Federation of Hemophilia.

Canadian Hemophilia Society

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In this podcast, Brian O’Mahony explores the patient experience of gene therapy for hemophilia, from the shared decision-making process prior to gene therapy, to the administration itself, to the intense follow-up in the first months and finally, the long-term follow-up. Brian is Chief Executive Officer of the Irish Haemophilia Society and a former president of the World Federation of Hemophilia and the European Haemophilia Consortium.

Canadian Hemophilia Society

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