According to a story from hemophilia.org, the drug developer uniQure recently announced that the first patient had been dosed in the company’s Phase IIb clinical trial. This trial is testing uniQure’s investigational product AMT-061, a gene therapy treatment for patients with severe hemophilia B. This gene therapy has the potential to revolutionize treatment for hemophilia B.
Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The severity of symptoms can vary widely. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures. There are multiple types of hemophilia, with the most common types being type A and type B, which are distinguished by having deficiencies in different clotting factors. Treatment involves replacing the missing clotting factor. Drugs that thin the blood should be avoided. To learn more about hemophilia, click here.
AMT-061 represents a major advance in gene therapy development for uniQure. This prototype contains the “Padua” variant of factor IX. Factor IX is the clotting factor that is deficient or absent in patients with hemophilia B. The Padua variant magnifies factor activity by as much as eight or nine times in comparison to the type used in the company’s previous gene therapy, AMT-060.
The clinical trial will consist of just three patients who will receive an intravenous infusion of the experimental gene therapy. These patients will then be subsequently monitored for a period of six to eight weeks to track the activity of factor IX. Enrollment for the subsequent Phase III trial is also ongoing, and is this trial is expected to consist of 50 patients with severe hemophilia B.
If AMT-061 works as intended, this therapy will only have to be administered once per patient. That could make a world of difference for hemophilia patients with severe disease who are not seeing the results they need with current treatments.