Gene Therapy for β-Thalassemia

Therapeutic options for severe β-thalassemia include red cell transfusions, chelation therapy for iron overload, and allogeneic hematopoietic stem cell transplantation for patients fortunate enough to have HLA-matched sibling donors.

Now, investigators have conducted an industry-sponsored, international, open-label, single-dose, phase I/II trial to evaluate the effect of infusing a functional β-globin gene in 22 β-thalassemia patients (ages, 12–35 years) with transfusion-dependent β⁰/β⁰- or β^ℇ/β⁰-thalassemia who lack HLA-matched sibling donors. Hematopoietic stem cells and progenitor cells harvested from patients were transduced with a LentiGlobin drug product (Bluebird Bio) and infused following myeloablative conditioning with busulfan.

All …