Novel gene therapy for hemophilia A leads to sustained expression of clotting factor and reduced bleeding events
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17 November 2021Novel gene therapy for hemophilia A leads to sustained expression of clotting factor and reduced bleeding events
A novel gene therapy for hemophilia A led to sustained expression of the clotting factor those patients lack, resulting in a reduction—or in some cases complete elimination—of painful and potentially life-threatening bleeding events, according to a new study led by researchers at Children’s Hospital of Philadelphia (CHOP). The results of the phase 1/2 trial, which were published today in the New England Journal of Medicine, are the first to demonstrate stable coagulation factor VIII in hemophilia A patients following gene therapy.
