Novel Therapies for Bleeding Disorders

Innovation is accelerating—not only in communications, computer technology, and artificial intelligence, but also in medical science. For people living with inherited bleeding disorders, this acceleration is transforming what is possible. Over the past decade, treatment advances have reshaped care. Extended half-life factor VIII and IX concentrates have reduced infusion frequency. Factor VIII mimetics such as emicizumab have introduced entirely new mechanisms of action. The first gene therapies for hemophilia A and B are now a reality. Rebalancing agents have entered the therapeutic landscape. And this is only the beginning. A growing pipeline of innovative therapies—subcutaneous treatments, non-factor therapies, gene editing approaches, rebalancing strategies, and more—are in various stages of development. These advances extend beyond hemophilia to include von Willebrand disease and rare bleeding disorders such as Glanzmann Thrombasthenia and others that historically received far less research attention. The pace of discovery shows no signs of slowing. With innovation comes choice. No longer are patients limited to nearly identical products. Today’s therapies differ significantly in how they work, how they are administered, their duration of effect, monitoring requirements, potential risks, and long-term considerations. As options expand, treatment decisions will become increasingly complex. Novel Therapies for Bleeding Disorders is a podcast series designed to help patients, families, and caregivers navigate this rapidly evolving landscape. Through clear, accessible conversations with leading clinicians, researchers and patient advocates, the series aims, among other things, to: break down complex science into understandable language;explain how emerging therapies work;discuss benefits, risks, and uncertainties;explore real-world considerations such as access, cost, monitoring, and long-term safety.The series will focus on therapies that may become available in Canada within the next few years for hemophilia A and B, von Willebrand disease, and rare bleeding disorders. It will also look further ahead to treatments in earlier stages of development that may transform care in the future. Our goal is to support informed, shared decision-making between patients and health care providers. By improving understanding of new and emerging therapies, we aim to empower listeners to ask the right questions, weigh their options, and make choices aligned with their medical needs, lifestyle, and personal values. Because innovation is not just about science—it is about improving lives. TRAITEMENTS NOVATEURS POUR LES TROUBLES DE LA COAGULATION L’innovation s’accélère — non seulement dans les domaines des communications, des technologies informatiques et de l’intelligence artificielle, mais aussi en sciences médicales. Pour les personnes vivant avec un trouble héréditaire de la coagulation, cette accélération transforme profondément les possibilités de traitement. Au cours de la dernière décennie, les avancées thérapeutiques ont redéfini les normes de soins. Les concentrés de facteurs VIII et IX à demi-vie prolongée ont permis de réduire la fréquence des perfusions. Les mimétiques du facteur VIII, comme l’émicizumab, ont introduit des mécanismes d’action entièrement nouveaux. Les premières thérapies géniques pour l’hémophilie A et B sont désormais une réalité. Les agents de rééquilibrage ont fait leur entrée dans l’arsenal thérapeutique. Et ce n’est qu’un début. Un nombre croissant de traitements innovants — traitements sous-cutanées, approches sans facteur, thérapies géniques, stratégies de rééquilibrage et autres technologies émergentes — sont à différents stades de développement. Ces progrès ne concernent pas uniquement l’hémophilie, mais aussi la maladie de von Willebrand et des troubles rares de la coagulation tels que la thrombasthénie de Glanzmann et