CHOP-led Study Shows Novel Gene Therapy for Hemophilia A Leads to Sustained Expression of Clotting Factor and Reduced Bleeding Events

A novel gene therapy for hemophilia A, SPK-8011, demonstrated sustained FVIII expression, reducing bleeding episodes significantly. The phase 1/2 trial, led by CHOP, showed stable coagulation factor VIII levels in patients, with a 91.5% reduction in bleeding. Funded by Spark Therapeutics, the study supports liver-directed AAV gene therapy for long-term hemophilia A treatment.