SSC Working Group on Gene Therapy

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      The Working Group on Gene Therapy has been established to formalize the standardization of methods and management of gene therapy for hemophilia in the United States and Europe.

If you are interested in the subcommittee and want to follow its activity, please click "join group" above to sign up as a member (follower).


  • Valuation of existing data and identifying areas of collaborative research.
  • Establish interdisciplinary collaborations with the aim of standardizing diagnosis and management
  • Evaluation of antiboides to various AAV isotypes
  • Development of laboratory standards for the measurement of transgene activity
  • Assessment of adverse events and side effects of gene therapy; development of treatment strategies. 
  •           Provide recommendations for the optimal use of gene therapy of hemophilia.

If you are a member of the Society and would like to know how to participate in the work of this group, please join the group to receive updates on activity or submit an Expression of Interest Form to the Chairman or any of the Steering Committee. We would be pleased to learn of your interest.


  • Chair - Wolfgang Miesbach
  • Steering Committee - Pratima Chowdary
  • Steering Committee - Frank Leebeek
  • Steering Committee - Johnny Mahlangu
  • Steering Committee - Flora Peyvandi
  • Steering Committee - Steve Pipe
  • Steering Committee - Alok Srivastava
  • Steering Committee - Mike Makris
  • Steering Committee - Jan Voorberg
  • Steering Committee - Glenn Pierce
  • Steering Committee - Radoslaw Kaczmarek 


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International Society on Thrombosis & Haemostasis