WEBINAR 2: [EN] Hemophilia Gene Therapy: One Step Back, Two Steps Forward
Gene Therapy for Hemophilia17 Dec 2024

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.


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