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 survey results will be presented in a poster session in Melbourne on July 7, titled, "Gene Therapy Knowledge and Perceptions: Results of an International ISTH Survey." The detailed roadmap developed for education around gene therapy will be presented during the Product Theater Sessions on July 7 at 12:15 p.m.

"Launching the roadmap in Melbourne is an exciting opportunity to kick off this global education initiative. It is an important first step in educating clinicians and researchers about the science and potential role of gene therapy for patients with hemophilia," said ISTH President Claire McLintock, M.D. "Our leadership in the steering committee and feedback from the greater thrombosis and hemostasis community has allowed us to understand the current needs and create meaningful education in gene therapy for the global hemophilia community."

"With the treatment landscape in hemophilia rapidly evolving and clinicians around the world being challenged to keep current with the latest scientific developments and clinical advancements, developing state-of-the-art clinical practice guidelines and education programs is critical to ensure best patient care," said Flora Peyvandi, M.D., Ph.D., Co-Chair of the ISTH Gene Therapy for Hemophilia Steering Committee.

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

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