The International Society on Thrombosis and Haemostasis (ISTH) is pleased to announce the official launch of Gene Therapy in Hemophilia: An ISTH Education Initiative. This landmark launch, the first of its kind in hemophilia, will take place during the ISTH XXVII Congress held in Melbourne, Australia, July 6-10, 2019. 

As gene therapy emerges as a potential new treatment for patients with hemophilia, the ISTH recognizes an immediate need to educate clinicians, scientists and other interested healthcare professionals in the global hemophilia healthcare community. In early 2019, the ISTH organized the ISTH Gene Therapy for Hemophilia Steering Committee, a group of world-renowned experts, led by Flora Peyvandi, M.D., Ph.D., and David Lillicrap, M.D., to survey the global hemophilia healthcare community to identify unmet educational needs specific to gene therapy in hemophilia.

The ISTH Gene Therapy for Hemophilia Steering Committee utilized the results of the survey, with input from others, to design a dynamic educational roadmap to guide the evolution of the gene therapy education program. The aim in its initial stage is to raise awareness and to provide clinicians and scientists with a better understanding of the fundamentals of gene therapy, the treatment approach, research and clinical trials, safety and efficacy outcomes, how to identify patients who could benefit, and how to analyze implications of this new treatment approach alongside other available and emerging treatments for hemophilia.

The Gene Therapy in Hemophilia: An ISTH Education Initiative is supported by educational grants from BioMarin, Pfizer, Inc., Shire, Spark Therapeutics, and uniQure, Inc.