Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose. In this phase 1–2 trial, an investigational adeno-associated viral (AAV) vector (SPK-8011) was infused for hepatocyte expression of factor VIII in 18 men with hemophilia A. Results showed sustained factor VIII expression in 16 of 18 participants, permitting discontinuation of prophylaxis and a significant reduction in bleeding episodes. No major safety concerns were reported.

The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose. In this phase 1–2 trial, an investigational adeno-associated viral (AAV) vector (SPK-8011) was infused for hepatocyte expression of factor VIII in 18 men with hemophilia A. Results showed sustained factor VIII expression in 16 of 18 participants, permitting discontinuation of prophylaxis and a significant reduction in bleeding episodes. No major safety concerns were reported.