Potential Treatments For Sickle Cell Disease

bluebird bio
bluebird bio

bluebird bio Inc. (BLUE) is developing a gene therapy by the name LentiGlobin BB305 for sickle cell disease.

LentiGlobin works by inserting a functional human beta-globin gene into a patient’s own hematopoietic stem cells outside the body (ex vivo) and then returning those modified cells to the patient through autologous stem cell transplantation.

A phase I/II study of LentiGlobin BB305 in subjects with either beta-thalassemia major or severe sickle cell disease, dubbed HGB 205, is being conducted in France.

A phase I study of LentiGlobin BB305 in adults with severe sickle cell disease, being conducted in the U.S., is known as HGB-206.

Data from the HGB-206 study is expected by the end of this year.

BLUE closed Thursday’s (Apr.5) trading at $170.15, down 1.70%.