Formerly known as Genetix Pharmaceuticals, Inc., and having now outgrown SBIR eligibility, bluebird bio Inc. (NASDAQ:BLUE) is usefully understood as a development stage biotechnology company developing transformative gene therapies for various inherited blood diseases, including bubble boy disease, SCD, and immune deficiency or marrow failure syndromes. With faciliites in Cambridge MA and Seattle, WA, the firm's disease targets include childhood cerebral adrenoleukodystrophy, which can cause buildup of fatty acids throughout the body; beta-thalassemia, a blood disorder that reduces production of hemoglobin; and sickel-cell diseases. The firm's core technology includes proprietary retroviral packaging cell line proven safe at the manufacturing level and in the clinic, as well as powerful gene expression enhancer elements. Other clinical research involved a phase I/II trial in cancer patients at Columbia-Presbyterian Hospital in New York where the multi-drug resistance gene is transduced ex vivo into hematopoietic stem cells. Those cells are then infused back into the patient with the hope of enabling transduced cells to withstand the toxicity of chemotherapy; similar trials have been approved to begin soon at other medical centers in the U.S. and Canada. bluebird bio has established a laboratory in Cambridge to pursue additional methods for permanent gene transduction, in conjunction with a focus on hematopoiesis and functional genomics. The company was honored in 2014 to be selected to attend and participate in the famous, highly exclusive Davos World Economic Forum.
