A better understanding of the pathophysiology of β-thalassemia has led to an increase in the life span of thalassemia patients and paved the way for new therapeutic strategies.

Gene therapy approaches using globin lentiviral vectors and genome-editing approaches to inhibit the BCL11A gene are currently under investigation.

Targeting ineffective erythropoiesis through the activin II receptor trap luspatercept has been shown to decrease the transfusion requirement in transfusion-dependent thalassemia.

Therapeutic strategies aimed at improving iron dysregulation such as minihepcidin and TMPRSS6 inhibitors are also showing promise, especially in non-transfusion-dependent thalassemia patients.