Gene Therapy for Hemophilia

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 SocietyGuest Speakers: Dr Pratima Chowdary, U.K.; Dr Davide Matino, Canada; Brian O'Mahony, Ireland; David Page, CanadaThis series is a creation of The Canadian Hemophilia Society - to find out more go to www.hemophilia.ca/gene-therapySee Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

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 SocietyGuest Speakers: Daan Breederveld, Netherlands; Stephen Brown, Canada, John Curley, U.K.; Brian O'Mahony, IrelandThis series is a creation of The Canadian Hemophilia Society - to find out more go to www.hemophilia.ca/gene-therapySee Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

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 SocietyGuest Speakers: James Foley, Ireland; Brian O'Loughlin, Ireland; David Page, Canada; Joseph Walsh, IrelandThis series is a creation of The Canadian Hemophilia Society - to find out more go to www.hemophilia.ca/gene-therapySee Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

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 SocietyGuest Speakers: James Foley, Ireland; Brian O'Loughlin, Ireland; David Page, Canada; Joseph Walsh, IrelandThis series is a creation of The Canadian Hemophilia Society - to find out more go to www.hemophilia.ca/gene-therapySee Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

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.

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.

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

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

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

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

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

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

In this podcast, Dr. David Lillicrap presents the results of clinical trials in hemophilia A and hemophilia B gene therapy. He describes how high factor VIII and IX levels can rise, how predictable this response is from person to person, and how long the therapy may be effective. Dr. Lillicrap is a Professor in the Department of Pathology and Molecular Medicine at Queen’s University in Kingston, Ontario, and is world renowned for his research in hemophilia gene therapy.Canadian Hemophilia SocietySee Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

In this podcast, Brian O’Mahony explores why gene therapy for hemophilia A and Hemophilia B is not for all patients. He describes what makes a person eligible, and in some cases ineligible, to receive the treatment. 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 SocietySee Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

In this podcast, Dr. David Lillicrap describes in easy-to-understand language what gene therapy for hemophilia A and hemophilia B is, how it is administered and how it works. Dr. Lillicrap is a Professor in the Department of Pathology and Molecular Medicine at Queen’s University in Kingston, Ontario, and is world renowned for his research in hemophilia gene therapy.Canadian Hemophilia SocietySee Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

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.

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.

This is a series of podcasts featuring well-known patient and physician experts will provide important answers, for example: What are the clinical trial results in hemophilia GT? What is the shared decision-making process in hemophilia GT? What is it like to receive it? What other approaches to hemophilia GTs are down the road?David Page is your host and explores the possibilities with medical experts. See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.