Gene Therapy for Hemophilia A Shows Sustained Clotting Factor Expression and Reduced Bleeding
• A novel gene therapy, SPK-8011, demonstrates stable coagulation factor VIII expression in hemophilia A patients, offering a potential long-term treatment option.
• The phase 1/2 trial showed a 91.5% reduction in bleeding episodes among participants, with most maintaining FVIII expression for the duration of the study.
• The therapy involves a recombinant AAV vector engineered to produce FVIII in liver cells, providing a one-time, disease-altering approach.
• While most participants responded well, two experienced a loss of expression due to an immune response, highlighting the need for further research.
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