Unmet needs in β-thalassemia and the evolving treatment landscape

•Currently available conventional treatment modalities in β-thalassemia have posed many challenges and limitations.•There is a need for therapies that reduce patient reliance on RBC transfusions and iron chelation therapy.•Many novel therapies for β-thalassemia are now being tested in clinical trials.•These can be classified into three major categories based on their efforts to address different features of the underlying pathophysiology of β-thalassemia: correction of the α/β globin chain imbalance, addressing ineffective erythropoiesis, and improving iron overload.