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Hemophilia Gene Therapy- Learning from Clinical Trials and Preparing for Clinical Practice
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When: Wednesday, August 5, 2020

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With the expected approval of the first gene therapy drugs for hemophilia in the next few months, it is important to clinicians are prepared for implementation in their respective hemophilia treatment centers (HTCs). During this 90-minute session, Drs. Steven Pipe and Guy Young will provide an overview of gene therapy for treatment of hemophilia along with an update on clinical trials on Hemophilia A and B. They will also discuss what success looks like and how HTCs should be preparing for the advent of gene therapy. The presentation will conclude with a live question and answer session between attendees and Drs. Pipe and Young. 

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7:00-7:05: Welcome and Introductions

7:05-7:20: Overview of Gene Therapy of Hemophilia: Vector and Transgene Optimization

7:20-7:35: Update on Clinical Trials of Gene Therapy in Hemophilia A and Hemophilia B

7:35-7:50: What does Success Look Like? Outcomes Behind Clinical Trial Endpoints 

7:50-8:10: What does Readiness Look Like? Peparing HTCs for Gene Therapy

8:10-8:30: Question and Answer Session


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

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