Advances in Gene Therapy for Hemophilia

Key Considerations:
Advances in Gene Therapy for Hemophilia

Recorded Live as a Friday Satellite Symposium preceding the 62nd ASH Annual Meeting and Exposition

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Presented by: Glenn F. Pierce, MD, PhD

Gene therapy holds much promise for individuals with a wide variety of conditions, including types of cancers, AIDS, diabetes, heart disease, and hemophilia. This innovative approach to treatment alters genes inside the body’s cells to stop the disease. 

Gene therapy attempts to replace or fix mutated genes and make diseased cells more evident to the immune system. Hemophilia A and B are inherited as part of an X-linked recessive pattern. Hemophilia genetics involve genes found in the X chromosome, and it takes just one faulty gene to lead to this bleeding disorder.

Cell and gene therapies are still under study, but the results are promising. Clinical trials have shown success, and the Food and Drug Administration (FDA) has approved one type of cell therapy. In our Key Considerations: Advances in Gene Therapy for Hemophilia led by Glenn F. Pierce, we discuss an overview of the disease, the current state of gene therapy, and more.


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

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