fb-pixelBluebird says gene therapy successful in sickle-cell patient - The Boston Globe Skip to main content

Bluebird says gene therapy successful in sickle-cell patient

Cambridge-based bluebird bio Inc. said Thursday that a patient with sickle cell disease was successfully treated with its gene therapy.

Bluebird's therapy, called LentiGlobin BB305, is being tested in blood disorders including beta-thalassemia and sickle cell disease. A patient's blood-forming stem cells are removed and a functioning human beta-globin gene is inserted. Then the cells are reinfused into the patient's bloodstream.

The patient's T87Q hemoglobin represented 24 percent of total hemoglobin after 4.5 months, bluebird said in a statement. T87Q is a type of hemoglobin with anti-sickling properties created by bluebird's product. Investors expected T87Q to compose 10 to 30 percent of total hemoglobin, said Jim Birchenough, an analyst at BMO Capital Markets Corp., in a note to clients on Wednesday.


The company's stock closed Thursday at $172.94, up 4.4 percent. The shares rose sixfold in the 12 months through Wednesday.

The shares had jumped 9 percent on Tuesday when the company said it had worked out an accelerated approval pathway with European and US regulators to get LentiGlobin BB305 to market for beta-thalassemia patients. A JPMorgan Chase & Co. analyst, Cory Kasimov, said the therapy could become available a couple of years earlier than expected under bluebird's announced regulatory plans.

Sickle cell disease and beta-thalassemia are both genetic disorders caused by mutations in the beta-globin gene. The conditions can results in severe anemia, leading patients to need chronic blood transfusions. About 90,000 to 100,000 people in the United States are affected, according to the Centers for Disease Control and Prevention. About 40,000 children worldwide are born with a serious form of beta-thalassemia each year, according to bluebird.