Take The Lead

A Cell Therapy Study for People Living with Beta Thalassemia


See if you Qualify

To learn more about this research study, click here


Living with Beta Thalassemia?

Beta thalassemia is a rare inherited blood disorder caused by a deficiency in the adult type of hemoglobin in red blood cells. Hemoglobin carries oxygen to all tissues of the body, including the heart, brain, lungs, and kidneys. In beta thalassemia patients, the deficiency is caused by a mutation, or change, in the gene encoding beta globin, a vital part of the oxygen-carrying hemoglobin complex in red blood cells. In severe forms of beta thalassemia, the mutations impair the production of healthy red blood cells and can lead to life-threatening anemia, enlarged spleen, liver and heart problems, and exceptionally weak bones. As a result, patients with severe forms of beta thalassemia need routine blood transfusions for the rest of their lives.  Because chronic blood transfusions can lead to toxicities from accumulating iron within the body, these patients also require life-long iron chelation.

Talk to your health care provider about the Thales Research Study.

The Thales Research Study is testing an investigational cell therapy for people with beta thalassemia who need routine blood transfusions. The therapy consists of a one-time infusion of ST-400 — manufactured from a person's own blood stem cells, which are removed and then genetically edited in a lab to produce blood with a higher oxygen-carrying capacity. Doctors are studying ST-400 to see if it can increase the body's production of fetal hemoglobin to make up for the defective adult-type hemoglobin.

You may qualify for the Thales Research Study if you:

  • Are 18 to 40 years old
  • Have been clinically diagnosed with beta thalassemia
  • Receive regular blood transfusions
  • Are in good general health

People who qualify will receive:

  • Investigational cell therapy (ST-400) for beta thalassemia
  • Research study-related medical care
  • Reimbursement for travel

To learn more about the Thales Research Study, please contact clinicaltrials@sangamo.com