ARM Member Profiles

bluebird bio is developing potentially transformative gene therapies for severe genetic diseases.  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 gene therapy platform involves inserting the correct copy of a faulty gene into the patient’s own blood stem cells. bluebird bio’s approach represents the ultimate personalized therapy and a true paradigm shift in the treatment of severe genetic diseases by presenting a potential one-time transformative option for patients. bluebird bio has two clinical-stage programs in development for childhood cerebral adrenoleukodystrophy (CCALD) and betathalassemia/sickle cell disease.  Led by a world-class team, bluebird bio is privately held and backed by top-tier life sciences investors.