Novel gene therapy for hemophilia A leads to sustained expression of clotting factor and reduced bleeding events.

News release: Novel gene therapy for hemophilia a leads to sustained expression of clotting factor and reduced bleeding events – Children’s Hospital of Philadelphia

Original Study: Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A – New England Journal of Medicine

Commentary on Twitter

3.5 years after therapy with factor VIII-containing adeno-associated virus, 16 of 18 men with hemophilia A had enough factor VIII expression that bleeding events were reduced to nearly zero and prophylaxis was discontinued. https://t.co/n3cPyjGDkn pic.twitter.com/vPyz0OmT1S

— NEJM (@NEJM) November 18, 2021