A Phase I/II Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-Thalassemia (TDT)
Thalassemia is an inherited blood disorder in which the genes that produce hemoglobin, the protein in red blood cells that carries oxygen from the lungs to all parts of the body, are broken. As a result, the red blood cells do not contain enough hemoglobin, causing anemia that can range from mild to life threatening.
Thalassemia can come in different forms depending on the genetic mutations causing it. One type of thalassemia is beta-thalassemia, which arises from mutations in or the loss of beta globin genes.
This thalassemia gene therapy clinical trial aims to assess the effectiveness of ST-400 on transfusion-dependent beta-thalassemia (TDT). ST-400 is a type of investigational therapy that consists of transfusing gene edited cells into a patient. In this study, the patient’s own blood stem cells are genetically modified in the laboratory using zinc finger nuclease (ZFN) genome editing technology created by Sangamo Therapeutics. Using this technology, the expression of the BCL11A gene, which normally shuts off the production of fetal hemoglobin shortly after birth, is turned down. After receiving conditioning chemotherapy to make room for the new cells in the bone marrow, the patient will receive a transfusion of edited blood stem cells.
Ultimately, this approach aims to boost the production of new enthrocytes, or red blood cells, with increased fetal hemoglobin (HbF), which can be substituted for either reduced or absent adult (defective) hemoglobin.