Bluebird bio is building a gene-therapy platform that inserts a functional copy of a disease-causing gene into a patient’s own hematopoietic stem cells using lentiviral vectors, eliminating the complications of donor transplants. Its lead clinical asset, LentiGlobin, is in an ongoing Phase 1/2 study for beta-thalassemia and sickle cell anemia and has shown the potential to end lifelong transfusions according to data published in Nature. The company also has a later-stage program for childhood cerebral adrenoleukodystrophy. Operations are split between Cambridge, MA and Paris, and the firm is privately held with backing from Third Rock Ventures, TVM Capital, Forbion Capital Partners, Easton Capital and Genzyme Ventures. Beyond internal R&D, bluebird bio collaborates with Généthon to advance lentiviral manufacturing. Its strategy centers on delivering one-time, curative treatments for rare blood and neurological disorders, and the latest non-dilutive funding strengthens its clinical and manufacturing capabilities. No commercial revenue has been disclosed in the article.

Total raised

$101M

Funding rounds

4

Latest round

Grant

Latest activity

Oct 2012