One-Dose Gene Therapy Produces Clotting Factor, Safely Stops Bleeding in Hemophilia B Patients

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.

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