- A 13-year-old French boy with severe sickle cell disease is the first patient to be treated with Bluebird Bio's Lentiglobin BB305 product, and he appears to be responding well to the treatment, according to the company.
- Sickle cell disease (SCD) is caused by a mutated gene, leading to abnormal red blood-cell function. As a result, patients suffer from anemia, painful obstruction of blood vessels, and sometimes, premature death.
- Bluebird Bio's therapy is based on extracting blood stem cells and then adding a functional gene back into the stem cells and reinjecting them into the patient.
In terms of therapy for SCD, the Bluebird Bio research team, headed up by Marina Cavazzana at Paris Descartes University, is tracking two main endpoints—the level of anti-sickling hemoglobin in the blood and the amount of time a patient can survive wihtout needing a life-sustaining blood transfusion. Researchers have deemed this case successful so far, becaue the young patient has not needed a blood transfusion for more than three months and his body was producing 45% anti-sickling hemoglobin at six months.
Another application of this therapy is for beta-thalassemia, a blood disorder related to SCD. Thus far, researchers have also had success treating this disorder, with two patients, who remained transfusion-independent at 16 and 14 month, respectively.
The success of these ongoing trials will determine whether or not Bluebird's pioneering gene therapy will eventually win approvals in the U.S. and E.U. markets. The company struck a tentative deal with the regulatory agencies mapping out a timeline and set of endpoints that could comprise the basis of a regulatory filing.