美国bluebird bio
单基因突变基因疗法

bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bio’s product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The company’s novel approach uses stem cells harvested from the patient’s bone marrow into which a healthy version of the disease causing gene is inserted. After being grown in culture, those cells are given back to the patient. bluebird bio’s approach represents a true paradigm shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and potentially presenting a one-time transformative therapy.

bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell anemia. Led by a world-class team, bluebird bio is privately-held and backed by top-tier life sciences investors, including Third Rock Ventures, TVM Capital, ARCH Venture Partners, Forbion Capital Partners, Easton Capital and Genzyme Ventures. Its operations are located in Cambridge, Mass., Paris, France and San Francisco, Calif.

Gene Therapy, The Time is Now
Over the last few years, a growing number of gene therapy trials have yielded increasingly promising results. bluebird bio’s proprietary lentiviral technology is designed to deliver corrective genes to the patient’s own bone marrow cells, providing a possible one-time effective therapy, a treatment approach that represents a true paradigm shift in the treatment of genetic diseases. bluebird bio has candidates in clinical development for the treatment of CCALD, a severe neurodegenerative disorder, beta-thalassemia, one of the most prevalent human genetic disorders, and sickle cell disease, a monogenic red blood cell disorder affecting over 10 million people worldwide. Both of these programs have shown promising early results in human trials to date with stabilization of their respective diseases. Results from the CCALD and Thalassemia programs were published in the November 2009 issue of and the September 2010 issue of , respectfully.