Beta thalassemia is a blood disease where the body cannot make enough healthy red blood cells because of a problem with a gene called β-globin. People with transfusion-dependent beta thalassemia (β-TDT) need regular blood transfusions to stay healthy, which can cause other problems like damage to the heart and liver.

HGI-001 is a new gene therapy that helps fix this problem by changing the genes in a patient’s own blood-making stem cells. Using a safe virus called a lentiviral vector, HGI-001 adds a working β-globin gene into the patient’s stem cells. These improved cells are put back into the body, where they start making healthy red blood cells. This helps the patient make their own blood cells and reduces or stops the need for blood transfusions.