FDA approved Zynteglo: 89% achieved transfusion independence.

Today represents a key milestone for beta-thalassemia patients battling hereditary chronic disease. This comes after the approval that was granted by the FDA approved to Zynteglo, also known as beti-cel.

Zynteglois developed by bluebird, is a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. ZYNTEGLO is expected to offer beta-thalassemia patients the possibility of freedom from chronic regular red blood cell transfusions and iron chelation.

ZYNTEGLO works by adding functional copies of a modified form of the beta-globin gene (βA-T87Q-globin gene) into a patient’s hematopoietic stem cells (HSCs) to allow them to make normal to near normal levels of total hemoglobin without regular RBC transfusions. The functional beta-globin gene is added into a patient’s cells outside of the body (ex-vivo) and then infused into the patient. Though ZYNTEGLO is designed to be administered to the patient once, the treatment process comprises several steps that may take place over several months. The approved gene therapy will be available exclusively at Qualified Treatment Centers only.

The effectiveness of ZYNTEGLO was established based on the achievement of transfusion independence, which is attained when the patient maintains a pre-determined hemoglobin level without needing any red blood cell transfusions for at least 12 months. Of 41 patients receiving Zynteglo, 89% achieved transfusion independence.