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News & Press: ISTH News

SSC Subcommittee Seeks Feedback on Gene Therapy Working Group

Thursday, August 23, 2018   (0 Comments)
Posted by: Luke Blount
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The SSC Subcommittee on Factor VII, Factor IX and Rare Coagulation Disorders seeks comments regarding the Gene Therapy Working Group (GTWG). The GTWG is charged with addressing issues that could hamper the development of gene therapy for hemophilia. These include issues related to the following: i.preclinical models and data needed for initiating clinical trials; ii.assays for assessment of pre-existing immune response to adeno associated virus vector for selection of suitable subjects for trials and subsequent therapies; iii.definition and characterization of the final therapeutic product; iv.clinical trial design with particular reference to definition of efficacy criteria, reliable and consistent factor assays, the trial design and long term monitoring of safety.


The group, which includes scientists and regulators, wishes to engage with all stakeholders (sponsors, academics and advocacy groups) in the field and has reached out to them with meetings held at recent WFH and ISTH conferences. This note is intended to make more people aware of this working group and to get their comments through this questionnaire or by communicating with any of the working group members.


Please complete this questionnaire by the end of September 2018.

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