bluebird bio announced that the FDA has granted Breakthrough Therapy designation to LentiGlobin BB305 Drug Product for the treatment of transfusion-dependent patients with beta-thalassemia major.

The Breakthrough Therapy designation is supported by data from the ongoing Phase 1/2 Northstar (HGB-204) and HGB-205 studies of LentiGlobin. In four of the eight patients, treatment resulted in sufficient hemoglobin production to reduce or eliminate the need for transfusion support among patients with beta-thalassemia major who would otherwise require chronic blood transfusions.

RELATED: Hematological Disorders Resource Center

LentiGlobin BB305 Drug Product is being developed to treat beta-thalassemia major and severe sickle cell disease by inserting a functional human beta-globin gene into the patient’s own hematopoietic stem cells ex vivo and then returning those modified cells to the patient through an autologous stem cell transplantation.

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