Category: Drug Development
Phone: +1 (617) 491-5601
Fax: + (617) 576-2421
Location: Cambridge, MA
Therapeutic Areas: Blood Disorder, Cancer/Oncology, Central nervous system, Immune System
bluebird bio is developing potentially transformative, one-time gene therapies for severe genetic and orphan diseases. At the heart of bluebird bio’s product creation efforts is its broadly applicable gene therapy platform for the development of new treatments for diseases with few or no clinical options. bluebird bio has two clinical stage product candidates in development, one for childhood cerebral adrenoleukodystrophy (CCALD) and one for beta-thalassemia major/sickle cell disease, and an early stage oncology program in partnership with Celgene Corporation.
Our gene therapy process involves extracting certain cell types from a patient, inserting genetic material into the patient’s own cells and then re-infusing the gene-modified cells back into the patient. For beta-thalassemia, sickle cell disease and adrenoleukodystrophy, we insert a functional copy of the target gene into the patient’s hematopoietic stem cells (HSCs), enabling patients to produce proteins that are otherwise missing or dysfunctional as a result of their hereditary disease. In oncology, the gene therapy process targets a different cell type, called T-cells. In this case, genetic sequences are inserted into a patient’s own T-cells to program the T cells to specifically recognize and attack cancer cells. bluebird bio’s approach represents a highly personalized therapy and a potential paradigm shift in the treatment of severe genetic and orphan diseases.
Led by a world-class team, bluebird bio’s operations are located in Cambridge, Mass., San Francisco, CA and Paris, France. For more information, please visit www.bluebirdbio.com.