Gene Therapy for Hemophilia

2026 marks the 100th year anniversary of when Dr Eric Von Willebrand first described the condition in a five year old girl. With this anniversary, we at the Canadian Hemophilia Society feel it's the perfect time to focus on increasing awareness, improving diagnosis, and addressing the fact that VWD remains under diagnosed and/or misdiagnosed. With that in mind, we're joined in this first episode by Dr. Dawn Goodyear, clinical associate professor and co-director of the southern Alberta rare blood and bleeding disorders program. Dr. Goodyear aims to give listeners a "101" style crash course on the ins and outs of Von Willebrand disease, as well as all of the currently available treatments.

This series is a creation of The Canadian Hemophilia Society - find out more at www.hemophilia.ca/von-willebrand-disease/

See 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, 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.

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.

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.

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.

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.

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 Society

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