Gene Therapy Advance in Hemophilia: Small Clinical Trial Using Gene for Hyperactive Clotting Factor Shows Impressive Success; Results Published in NEJM

A team of gene therapy researchers has reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered gene therapy treatment enabled adult participants to safely produce sustained levels of clotting factor that prevented debilitating bleeding episodes. Patients were able to terminate prophylactic treatments--the gene therapy nearly universally eliminated their need for intravenous infusions of manufactured clotting factor. "A one-time therapy sufficient to prevent bleeding without further medical intervention is the ideal treatment goal for patients with hemophilia," said lead investigator Lindsey A. George (photo), MD, a hematologist at Children's Hospital of Philadelphia (CHOP). "This cohort of 10 patients all safely experienced sustained clinical benefit after one infusion." Dr. George and colleagues published their findings in the December 7, 2017 issue of the New England Journal of Medicine (online December 6). The article is titled “Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.” The team previously reported preliminary results from the trial in December 2016 at the plenary session of the American Society of Hematology annual meeting. Dr. George is a member of CHOP's Division of Hematology as well as the Raymond G. Perelman Center for Cellular and Molecular Therapeutics. That center is part of CHOP's Roberts Collaborative for Genetics and Individualized Medicine. The phase 1/2 clinical trial, involving ten adult male participants, was sponsored by Spark Therapeutics of Philadelphia. The study's senior author is gene therapy pioneer Katherine High, MD, President and Head of Research and Development at Spark Therapeutics. Starting in the 1990s, Dr.
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