Nationally and internationally renowned hemophilia treatment center City of Hope has opened a new gene therapy clinical trial for treatment of hemophilia B.
The pioneering therapy, conducted in collaboration with Sangamo Biosciences, offers a novel means of treating severe hemophilia B, possibly eliminating the need to infuse intravenously and frequently with costly factor products, which is the current gold standard of treatment for bleeding disorders.
“As it is a gene defect that causes a lack of factor IX production, leading to the diagnosis of severe hemophilia B, this new treatment is designed to deliver normal factor-producing genes intravenously to the liver via specially designed vectors, or carriers, thereby improving factor production and blood clotting,” says Nadia Ewing
, M.D., the director of City of Hope’s Hemophilia and Sickle Cell Program, and the principal investigator of the clinical trial. “In this way, the investigational product, called SB-FIX, aims to treat spontaneous bleeding episodes, as well as prevent recurrence of bleeding events.”
There have been three previous studies investigating the safety and efficacy of gene therapy for treatment of severe hemophilia B in 25 patients. The most recent study showed favorable results, with 40 percent of participants eliminating the need for their previous prophylactic infusion regimen.