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.