Sangamo Gene Therapy for Thalassemia (ST-400-01)

Individuals with beta thalassemia that is treated with regular transfusions, and who are between the ages of 18 and 40 years old, may be able to participate. A number of screening tests will be done to determine if subjects can participate. These include review of your past medical history, blood and urine tests, bone marrow aspirate, DEXA scan, heart ultrasound, electrocardiogram, magnetic resonance imaging (MRI) of the heart and liver, and liver biopsy. If eligible, blood stem cells will be collected by apheresis. These stem cells will then be modified using gene editing to increase fetal hemoglobin production. You will then be hospitalized and chemotherapy will be given to empty out the bone marrow. The stem cells, with the edited gene, will then be given back to you. You will be followed for side effects and to see if the cells with the edited gene help you make more fetal hemoglobin.