- The FDA designates bluebird bio's (NASDAQ:BLUE) LentiGlobin BB305 a Breakthrough Therapy for the treatment of transfusion-dependent patients with beta-thalassemia major. The product candidate works by inserting a functional beta-globin gene into the patient's own hematopoietic stem cells ex vivo. The modified cells are then returned to the patient via an autologous stem cell transplantation. It is currently in Phase 2 development.
- Breakthrough Therapy status allows a more rapid review of the New Drug Application (NDA) or Biologics License Application (BLA), including allowing the submission after a Phase 2 trial, in certain cases.