Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of β-globin protein and resulting in chronic anemia, failure to thrive, jaundice, pallor, poor musculature, and skeletal deformities. No curative therapy exists for beta thalassemia other than an allogenic hematopoietic stem-cell transplantation (HSCT) with a matched (most often sibling) donor. Patients are currently managed with a combination of blood transfusions and iron chelators. The most severe thalassemia major patients require chronic blood transfusions to survive, substantially affecting their quality of life (QOL). A high unmet exists for therapies to reduce or eliminate the burden of transfusion. Drug developers have recognized the commercial opportunity in the beta thalassemia market and have focused on developing agents with novel mechanisms of action that target the underlying genetic defect and reduce the transfusion burden.
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