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General Updates | Barriers and unanswered questions in the field of gene therapy for hemophilia
In this video, Radek Kaczmarek, PhD, Indiana University School of Medicine, Indianapolis, IN, provides insight into the barriers and unanswered questions that need to be addressed to facilitate the widespread implementation of gene therapy for the treatment of hemophilia. This interview took place virtually as part of the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH) 2025.
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Transcript
Variability and unpredictability of outcomes are important barriers in decision-making. Also, AAV delivery triggers long-lasting, robust, neutralizing anti-AAV antibody responses, which preclude redosing to correct suboptimal outcomes and will likely preclude administration of alternative gene therapies, at least those relying on AAV to deliver transgenes.
Now, the current gene therapies are not available to children who have the most to gain from early hemostatic correction and prevention of bleeding...
Variability and unpredictability of outcomes are important barriers in decision-making. Also, AAV delivery triggers long-lasting, robust, neutralizing anti-AAV antibody responses, which preclude redosing to correct suboptimal outcomes and will likely preclude administration of alternative gene therapies, at least those relying on AAV to deliver transgenes.
Now, the current gene therapies are not available to children who have the most to gain from early hemostatic correction and prevention of bleeding. And there are also institutional barriers, of course. Gene therapy is a new modality, and haemophilia treatment centers need to be able to perform the procedure, closely monitor the patient, and most importantly, secure reimbursement from payers. And gene therapies are high-priced drugs that have a high impact on healthcare budgets, especially if they were to be done on a one-off basis.
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Disclosures
Grant funding: Bayer; Speaker/Consultancy fees: Biomarin, Pfizer, Novo Nordisk, Spark, Bayer.
