Bluebird Bio’s gene therapy for blood disorders yields some impressive results ― but also raises questions
Bluebird Bio's trial of a gene therapy for two inherited blood disorders yielded some impressive results Friday ― but also raised key questions.
The field of gene therapy is making significant strides towards the day when a one-time treatment ― inserting a healthy gene into patients ― could cure a host of inherited, often fatal diseases. But it’s been a rocky road. And new clinical data presented this morning by Bluebird Bio make clear that success is far from guaranteed. Bluebird is testing a gene therapy for two inherited blood disorders ― and the data managed both to impress and to leave important questions unanswered.
On the impressive side: A patient with the inherited blood disease beta-thalassemia, which causes severe anemia, was able to
