Bluebird Bio Launches First Gene Therapy for Beta Thalassemia

A member of the American Society of Human Genetics, Neda Razavi is a former marketing and portfolio manager at Abbott Laboratories. Neda Razavi has launched products that unlock gene information to provide targeted therapy for various chronic and life-threatening conditions.

Beta thalassemia is a blood disorder characterized by low production of hemoglobin, a protein in red blood cells that helps carry oxygen to other cells throughout the body. It can result in anemia and abnormal formation of blood clots. Beta thalassemia occurs in three different types, which are beta thalassemia minor (beta thalassemia trait), beta thalassemia intermedia, and beta thalassemia major (Cooley’s anemia).

People with beta thalassemia minor usually have mild anemia that generally doesn’t require treatment. However, people with beta thalassemia intermedia or beta thalassemia major usually have moderately severe or severe anemia requiring treatment. Traditional treatment for beta thalassemia involves routine blood transfusions to help deliver healthy red blood cells and hemoglobin to patients.

Recently, biotech company bluebird bio, Inc. launched the first gene therapy for beta thalassemia. With this treatment, beta thalassemia patients no longer have to rely on periodic blood transfusions. Marketed under the name Zynteglo, this therapy was approved in June 2019 by the European Union for treatment of beta thalassemia in patients above 12 years old except those with a very severe form of transfusion-dependent beta thalassemia.