The first day of the GTRT started with a general update on the status of different gene therapy products, followed by a session which covered the importance of communicating science in an unbiased and balanced manner. The sessions that followed focused on safety, variability and durability for both factor VIII and factor IX gene therapies; regulatory perspectives from the United States and Europe; and informed consent and shared decision-making, including a presentation of the WFH Hemophilia Treatment Shared Decision-Making tool that is being developed now.
Day two focused on different aspects of accessibility, with speakers discussing how to expand eligibility beyond current limitations, and alternatives to adeno-associated virus (AAV) such as lentivirus, gene editing, and non-viral gene therapy. The final two sessions centered on the pricing and commercialisation of gene therapy in higher-middle income countries, and the specific challenges faced by lower-middle income countries.
The 5th WFH GTRT fostered important and informative conversation between all stakeholders as the global dialogue on gene therapy for hemophilia continues. To find out more about the WFH GTRT series, please click here.