Bluebird bio's gene therapy plans hit a high note

Over the last week, Cambridge, MA-based bluebird bio has taken several key steps forward. The gene therapy pioneer named Alexandre LeBeaut as its new chief medical officer, signed up an impressive roster of experts for its scientific advisory board and inked a $4.2 million pact with the French Muscular Dystrophy Association to develop a treatment for beta-thalassemia and sickle cell anemia. For bluebird bio CEO Nick Leschly, the series of moves help represent a turning point for a biotech determined to prove that its novel approach can make a big difference in the treatment of rare genetic conditions--an increasingly popular field in biopharma in which rare diseases represent significant market opportunities. Story