bluebird bio (NASDAQ:BLUE) is a clinical-stage biotechnology company focused on developing transformative gene therapies for severe genetic diseases and cancer. The company leverages its proprietary lentiviral-based platform to deliver functional copies of genes into patients’ cells, aiming to address the underlying causes of monogenic disorders. bluebird bio’s lead programs include a treatment for transfusion-dependent β-thalassemia and a therapy for cerebral adrenoleukodystrophy, with additional candidates advancing in sickle cell disease, metachromatic leukodystrophy and oncology indications.
Founded in 1992 as Genetix Pharmaceuticals and rebranded in 2013, bluebird bio has established a global footprint with operations in Cambridge, Massachusetts, and European research facilities. Its multidisciplinary teams encompass gene therapy research, process development, clinical operations and regulatory affairs. The company’s manufacturing network supports both ex vivo autologous cell therapies and in vivo gene editing approaches, reflecting its commitment to scalable production and broad patient access.
Beyond its core pipeline, bluebird bio collaborates with academic institutions and industry partners to accelerate discovery and optimize clinical outcomes. These partnerships span translational research, vector engineering and long-term follow-up studies aimed at evaluating durability and safety. Through these efforts, the company seeks to expand its platform’s versatility across rare genetic diseases and selected oncology applications, including CAR-T cell therapies for hematologic malignancies.
bluebird bio is guided by a leadership team composed of seasoned executives and scientific experts with extensive experience in biotechnology and pharmaceutical sectors. Supported by a board of directors that includes innovators in gene therapy and drug development, the company is committed to advancing its mission of delivering potentially curative treatments to patients with high unmet medical needs.
