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 own bone marrow into which a healthy version of the disease causing gene is inserted. 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 using a patient's own stem cells. bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia, and plans to initiate a program in sickle cell disease.

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