The Applications of Zynteglo in Treating Transfusion Dependent Beta-Thalassemia

Abstract

Beta-thalassemia is a hereditary blood disorder, with one of the most severe forms being transfusion-dependent thalassemia (TDT). Zynteglo is a cell-based gene therapy that seeks to provide an alternative for routine blood transfusions required in the treatment of TDT. The manufacturing process of Zynteglo begins with the collection and modification of a patient’s hematopoietic stem cells. Functional copies of the beta-globin gene are then introduced to restore normal hemoglobin production. In clinical trials, Zynteglo has demonstrated its effectiveness as a treatment for TDT, with an assessment report led by Northwestern University 78.6% of American patients achieving complete transfusion independence. However, staggering costs, accessibility issues, and safety concerns hinder the acceptance of the drug in major regions of the world. Future considerations must address the risks and barriers associated with the treatment process to foster greater accessibility.