Gene Therapy for Hemophilia

Canadian Hemophilia Society
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Gene therapy has remained a constant prospect in the sphere of hemophilia treatment. For over two decades and the question remained: Is this just a dream, or could it one day become a reality? Hemophilia gene therapy has finally been approved. David Page talks to hemophilia specialists that will dive deep into understanding how the treatment works, its significance in advancing treatment, and how a one time treatment could change a severe condition to a milder one.Though not a definitive cure, gene therapy for hemophilia has transformed the dream of a cure into a reality for the future. David and his guests will provide exclusive insights into one of the most significant medical breakthroughs of our era.

  1. Shared Decision-Making

    FEB 11

    Shared Decision-Making

    Over the last several years, gene therapy for hemophilia B has become a reality. Some received it through clinical trials, and others received it after marketing authorizations and reimbursement approvals in their countries. Each of them had a difficult, life-changing decision to make. They needed to fully understand the potential benefits and risks, and the physicians responsible for their treatment needed to be assured that they were providing fully informed consent to the therapy. This process is called shared decision-making. In this episode of our second podcast series, GENE THERAPY FOR HEMOPHILIA – FROM DREAM TO REALITY, four people, two physicians and two people with hemophilia, were invited to share their insight on this topic. Host: Sarah Ford, CEO, Canadian Hemophilia Society Guest Speakers: Dr Pratima Chowdary, U.K.; Dr Davide Matino, Canada; Brian O'Mahony, Ireland; David Page, Canada This series is a creation of The Canadian Hemophilia Society - to find out more go to www.hemophilia.ca/gene-therapy See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

    28 min
  2. Four People Describe Their Experiences with Hemophilia B Gene Therapy

    FEB 11

    Four People Describe Their Experiences with Hemophilia B Gene Therapy

    Over the last several years, gene therapy for hemophilia B has become a reality. Some received it through clinical trials. Others received it after marketing authorizations and reimbursement approvals in their countries. In this episode of our second podcast series, GENE THERAPY FOR HEMOPHILIA – FROM DREAM TO REALITY, four of these people were asked “Are you happy you made the decision to receive gene therapy? Why or why not?” Host: David Page, Canadian Hemophilia Society Guest Speakers: Daan Breederveld, Netherlands; Stephen Brown, Canada, John Curley, U.K.; Brian O'Mahony, Ireland This series is a creation of The Canadian Hemophilia Society - to find out more go to www.hemophilia.ca/gene-therapy See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

    35 min
  3. If Gene Therapy for Hemophilia B Were Available Tomorrow, Would You Want to Receive It?

    JAN 28

    If Gene Therapy for Hemophilia B Were Available Tomorrow, Would You Want to Receive It?

    For the last 40 years, we have witnessed tremendous advances in care and treatment for people with hemophilia. Gene therapy has been a dream for most of those 40 years, and the last two to three years, two gene therapies for hemophilia B have been approved by regulatory bodies. But are they for everybody? In this episode of our second podcast series, GENE THERAPY FOR HEMOPHILIA – FROM DREAM TO REALITY, we will ask four people with severe hemophilia B to answer this question, “If gene therapy for hemophilia B were available tomorrow, where you live, would you want to receive it?” Host: Brian O’Mahony, Chief Executive, Irish Haemophilia Society Guest Speakers: James Foley, Ireland; Brian O'Loughlin, Ireland; David Page, Canada; Joseph Walsh, Ireland This series is a creation of The Canadian Hemophilia Society - to find out more go to www.hemophilia.ca/gene-therapy See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

    24 min
  4. Overview of Gene Therapy Approaches

    JAN 28

    Overview of Gene Therapy Approaches

    In this first episode of our second podcast series, GENE THERAPY FOR HEMOPHILIA – FROM DREAM TO REALITY, Dr. David Lillicrap describes what gene therapy is in 2026 and hopefully how it works. Dr. Lillicrap is a Professor in the Department of Pathology and Molecular Medicine at Queen’s University in Kingston, Ontario, and chair in Canadian Molecular Hemostasis Research. Host: Brian O’Mahony, Chief Executive, Irish Haemophilia Society Guest Speakers: James Foley, Ireland; Brian O'Loughlin, Ireland; David Page, Canada; Joseph Walsh, Ireland This series is a creation of The Canadian Hemophilia Society - to find out more go to www.hemophilia.ca/gene-therapy See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

    28 min
  5. WEBINAR 2: [FR] Un Pas en Arrière, Deux Pas en Avant

    02/19/2025 · BONUS

    WEBINAR 2: [FR] Un Pas en Arrière, Deux Pas en Avant

    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. See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

    1h 3m
  6. WEBINAR 2: [EN] Hemophilia Gene Therapy: One Step Back, Two Steps Forward

    12/17/2024 · BONUS

    WEBINAR 2: [EN] Hemophilia Gene Therapy: One Step Back, Two Steps Forward

    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. See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

    1h 1m
  7. DEBATE: If gene therapy for hemophilia B were available in Canada tomorrow, would you want to receive it?

    06/05/2024

    DEBATE: If gene therapy for hemophilia B were available in Canada tomorrow, would you want to receive it?

    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?” See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

    31 min
  8. DEBATE: If gene therapy for hemophilia A were available in Canada tomorrow, Would you want to receive it?

    03/28/2024

    DEBATE: If gene therapy for hemophilia A were available in Canada tomorrow, Would you want to receive it?

    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?” See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

    17 min
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Trailer

About

Gene therapy has remained a constant prospect in the sphere of hemophilia treatment. For over two decades and the question remained: Is this just a dream, or could it one day become a reality? Hemophilia gene therapy has finally been approved. David Page talks to hemophilia specialists that will dive deep into understanding how the treatment works, its significance in advancing treatment, and how a one time treatment could change a severe condition to a milder one.Though not a definitive cure, gene therapy for hemophilia has transformed the dream of a cure into a reality for the future. David and his guests will provide exclusive insights into one of the most significant medical breakthroughs of our era.

Information

  • Creator
    Canadian Hemophilia Society
  • Years Active
    2023 - 2026
  • Episodes
    18
  • Rating
    Clean
  • Copyright
    © 2023-2026
  • Show Website
    Gene Therapy for Hemophilia