A lower-dose gene therapy has been reported to reduce bleeding episodes in people with severe haemophilia A.
The therapy, GS001, is designed to deliver a functional copy of the F8 gene to liver cells using an adeno-associated viral vector. This enables the body to produce factor VIII, the clotting protein that is missing or reduced in people with haemophilia A.
Twelve men with severe haemophilia A received a single infusion of the therapy at one of two dose levels, both lower than those used in existing gene therapies for the condition.
Factor VIII levels increased within the first week after treatment and the number of bleeding episodes decreased.
The findings are published in Signal Transduction and Targeted Therapy.
Haemophilia News Today has more on the story.