Expert’s Corner
Podcasts: Reflections and learnings from the early gene therapies for Hemophilia B
Four conversations
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The nurse perspective
Dr. Glenn Pierce speaks with nurse practitioner and gene therapy expert Greta Mulders about managing some of the first patients treated with gene therapy for hemophilia B. The discussion explores the emotional journey, patient relationships, and the evolving role of nurses in supporting patients and families through a completely new treatment paradigm.
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Real-world impact of early gene therapy
Dr. Glenn Pierce and researcher Dr. Radek Kaczmarek reflect on the scientific breakthroughs and real-world impact of early gene therapy developments for hemophilia B. They discuss the transition from early uncertainty to growing confidence in durability, safety, and long-term outcomes, alongside ongoing challenges in implementation and access.
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The science behind durable factor IX expression
Dr. Glenn Pierce and scientist Dr. Anne Galy explore the biological foundations behind successful gene therapy for hemophilia B. The episode examines AAV vector design, long-term factor IX expression, immune response, and the cellular mechanisms influencing treatment durability and therapeutic benefit.
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Early patient experiences with gene therapy
Dr. Glenn Pierce speaks with Dr. Karina Meijer about her involvement in the AMT-060 trial and the early clinical experience of discussing gene therapy with patients with hemophilia B. The episode explores patient expectations, long-term monitoring, and how these early experiences continue to shape conversations about gene therapy today.