About This Initiative

As gene therapy emerges as a potential new treatment approach for hemophilia, the International Society on Thrombosis and Haemostasis (ISTH) is proud to introduce Gene Therapy in Hemophilia: An ISTH Education Initiative.

In early 2019, ISTH organized a group of world-renowned experts from the global hemophilia community to develop a survey to identify unmet educational needs specific to gene therapy in hemophilia. The survey was distributed online to an international audience. The results demonstrated that many need more education on the fundamentals of gene therapy and a better understanding of gene therapy as a treatment approach for hemophilia A and B.

ISTH and the global hemophilia experts have developed educational resources to address these needs providing you a front seat to learn about gene therapy in hemophilia. Improve your understanding of the fundamentals and stay abreast of the latest clinical advances as it relates to gene therapy in hemophilia. Learn from leading global experts on this dynamic educational platform.


ISTH would like to thank BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc. for their support of this educational initiative

ISTH’s Educational Roadmap–Guiding Education for the Future


Steering Committee


Flora Peyvandi, MD, PhD (Co-Chair)


David Lillicrap, MD


Johnny Mahlangu, BSc, MBBCh, MMed, FCPath


Glenn F. Pierce, MD, PhD


Steven W. Pipe, MD


Alok Srivastava, MD, FRACP, FRCPA, FRCP


Thierry VandenDriessche, PhD

About ISTH


Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. ISTH is an international professional membership organization with more than 5,000 clinicians, researchers and educators working together to improve the lives of patients in more than 100 countries around the world. Among its highly regarded activities and initiatives are education and standardization programs, research activities, cell and gene therapy, yearly congresses, peer-reviewed publications, expert committees and awareness programs. Visit ISTH online at www.isth.org.


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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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