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AUGUST 15, 2016

Gene Therapy for Hemophilia Gains Ground

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By Kate O’Rourke


After a checkered past, gene therapy for hemophilia A and B is finally scoring some clinically meaningful successes, according to leading researchers in the field. Hopes are high that the promising results, most recently seen in Phase I/II trials, will progress to Phase III trials and then the clinic setting.

“The current landscape of gene therapy for hemophilia A and B is quite promising,” said Lindsey George, MD, an attending physician in the Division of