According to a story from PR Newswire, the genomics medical company Sangamo Therapeutics, Inc., recently announced that the first patient had been dosed in its Phase 1/2 clinical trial which is testing SB-FIX, an experimental gene therapy in development as a treatment for hemophilia B. Sangamo is committed to the development of gene therapies for serious diseases.
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.
New Treatments Needed
Hemophilia B is characterized by a deficiency affecting clotting factor IX. The primary treatment for this disease is periodic infusions of the missing factor, which can restore normal clotting behavior. However, the risk of bleeding is never entirely resolved and the patient burden of periodic infusions is substantial.
SB-FIX has a different functional mechanism compared to other gene therapies, which often adeno-associated viruses (AAV) or retroviral based mechanisms to deliver the genetic material to the patient. While effective, this approach has some noticeable disadvantages, such as a lack of precision and the risk of immune system response. SB-FIX uses a unique zinc finger nuclease to insert a normal F9 gene company into liver cells. This could allow for this gene therapy to function as a curative, one-time treatment.
The clinical trial is currently recruiting more subjects for the study. SB-FIX has earned Fast Track and Orphan Drug designations from the US Food and Drug Administration (FDA).