Bluebird bio

American biotechnology company

Logo of Bluebird Bio

Bluebird Bio, Inc. is an American biotechnology company that focuses on developing gene therapies for severe genetic diseases. The company is headquartered in Somerville, Massachusetts, and it is known for its innovative approaches to treating conditions such as beta thalassemia, cerebral adrenoleukodystrophy, and sickle cell disease.

History

Bluebird Bio was founded in 1992 under the name Genetix Pharmaceuticals. The company rebranded to Bluebird Bio in 2010, reflecting its focus on gene therapy. Over the years, Bluebird Bio has been at the forefront of developing gene therapy treatments, leveraging its proprietary lentiviral vector technology.

Technology

Bluebird Bio utilizes lentiviral vectors to deliver therapeutic genes into patients' cells. This technology allows for the stable integration of the therapeutic gene into the patient's genome, providing a long-term solution for genetic disorders. The company’s approach involves extracting a patient’s hematopoietic stem cells, modifying them with the therapeutic gene, and then reintroducing them into the patient’s body.

Products and Pipeline

Bluebird Bio has developed several gene therapy products, including:

• Zynteglo (betibeglogene autotemcel): A gene therapy for transfusion-dependent beta thalassemia. Zynteglo was approved by the European Medicines Agency in 2019.
• Skysona (elivaldogene autotemcel): A treatment for cerebral adrenoleukodystrophy, a rare neurodegenerative disease.
• LentiGlobin: An investigational therapy for sickle cell disease, designed to introduce a functional copy of the beta-globin gene into a patient’s stem cells.

Research and Development

Bluebird Bio is actively involved in research and development to expand its pipeline of gene therapies. The company collaborates with various academic and research institutions to advance its technology and explore new therapeutic areas.

Business Strategy

Bluebird Bio’s business strategy focuses on developing and commercializing gene therapies for severe genetic diseases. The company aims to provide transformative treatments that address the underlying causes of these conditions, offering patients potentially curative options.

Challenges and Controversies

Like many biotechnology companies, Bluebird Bio faces challenges related to the high cost of gene therapy development and the complexities of regulatory approval. The company has also encountered setbacks in clinical trials, which are inherent in the development of novel therapies.

Related pages

• Gene therapy
• Biotechnology
• Hematopoietic stem cell
• Lentiviral vector