This bleeding often leads to disability and premature death. In the case of hemophilia B, patients are missing protein factor IX, which is one of the essential proteins responsible for the clotting of blood. The common treatment for hemophilia employs repeated infusions of the missing clotting factor. This treatment is costly, time-consuming and imperfect since the administered clotting factor only remains in the body for a limited time. Damaging bleeds often occur between infusions and result in joint damage, chronic pain and disability.
Current therapies treat only the symptoms of hemophilia rather than the cause.
Commenting on the trial was Steven Humes, Director of Research for the National Hemophilia Foundation. B. We are committed to finding a cure for all bleeding disorders and this trial is an important step in support of that goal. The goal of gene therapy is to provide the body with the missing genetic information which will enable it to produce the missing clotting factor. Such a therapy may diminish or eliminate the life-endangering complications common to those suffering with hemophilia. Avigen hopes that Coagulin-B will ultimately provide enough clotting factor to prevent spontaneous bleeding.