Welcome to our podcast series, today we interview Prof. Thierry VandenDriessche to discuss how to enhance the efficacy and safety of gene therapy for haemophilia A and B. Despite achieving multiyear therapeutic levels of factor VIII and factor IX in patients, challenges remain. Variability in expression, gradual decline of factor VIII levels, and frequent incidence of transaminitis present ongoing hurdles.

In this episode, we delve into these challenges and discuss strategies to overcome them, with a focus on the use of adeno-associated viral vectors (AAV). We also examine the advancements and limitations of integrating vector systems, such as lentiviral vectors and CRISPR-based gene editing, in preclinical studies. These strategies aim to maintain sustained clotting factor expression, even amidst target cell division.

🎥 Watch the full module on the EAHAD Academy: https://academy.eahad.org/eahad/2024/module-12-how-can-we-improve-the-efficacy-and-saf/419034