Location: Massachusetts, USA
Number of employees: 52
Year founded: 1993
840 Memorial Drive
Cambridge, MA 02139
Gene therapy holds the promise of transforming the way we treat many life-threatening diseases. bluebird bio is pioneering a way to correct aberrant sections of DNA that cause disease and are passed from generation to generation.
The company has identified a way to harness the natural ability of the human immunodeficiency virus (HIV), a lentivirus, to insert a modified gene into a patient’s own cells. bluebird uses its lentiviral vectors to transfer functional genes into a patient’s own stem cells, which are capable of changing into multiple cell types, providing the company with the opportunity of treating a wide range of genetic diseases.
bluebird is currently developing gene therapy products to treat three hereditary diseases where the genetic abnormality is known and found in a single gene: childhood cerebral adrenoleukodystrophy, a rare and often fatal neurological disorder affecting young boys; and beta-thalassemia major and severe sickle cell disease, which are blood disorders that often lead to severe anaemia and other potentially life-threatening symptoms.
In partnership with Celgene Corporation and Baylor University, bluebird is also working on using its lentiviral approach to modify a patient’s own T cells – a type of white blood cell – to fight their cancer.
If bluebird’s first gene therapy products successfully demonstrate clinical benefit with a positive safety profile, there is potential to apply the approach to a wide variety of diseases that have a genetic origin. Rather than addressing symptoms of a disease, this approach promises a one-time, transformational treatment of the underlying cause.