bluebird bio, Inc., based in

Somerville, Massachusetts Somerville ( ) is a city located directly to the northwest of Boston, and north of Cambridge, Massachusetts, Cambridge, in Middlesex County, Massachusetts, United States. As of the 2020 United States census, the city had a total population of 81, ...

, is a

biotechnology Biotechnology is a multidisciplinary field that involves the integration of natural sciences and Engineering Science, engineering sciences in order to achieve the application of organisms and parts thereof for products and services. Specialists ...

company that develops gene therapies for severe

genetic disorder A genetic disorder is a health problem caused by one or more abnormalities in the genome. It can be caused by a mutation in a single gene (monogenic) or multiple genes (polygenic) or by a chromosome abnormality. Although polygenic disorders ...

s. The company is owned by

investment fund An investment fund is a way of investment, investing money alongside other investors in order to benefit from the inherent advantages of working as part of a group such as reducing the risks of the investment by a significant percentage. These ad ...

s managed by

The Carlyle Group The Carlyle Group Inc. is an American multinational company with operations in private equity, alternative asset management and financial services. As of 2023, the company had $426 billion of assets under management. Carlyle specializes in ...

and SK Capital Partners. One of the company's approved drugs is betibeglogene autotemcel (Zynteglo), which treats transfusion-dependent

beta thalassemia Beta-thalassemia (β-thalassemia) is an genetic disorder, inherited hemoglobinopathy, blood disorder, a form of thalassemia resulting in variable outcomes ranging from clinically asymptomatic to severe anemia individuals. It is caused by reduce ...

(TDT), a rare genetic blood disorder, and has been approved for use by the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

. The company has been criticized for the $1.8 million cost of the drug, which is the second most expensive drug in the world. The company is developing LentiGlobin gene therapy for the treatment of

sickle cell disease Sickle cell disease (SCD), also simply called sickle cell, is a group of inherited Hemoglobinopathy, haemoglobin-related blood disorders. The most common type is known as sickle cell anemia. Sickle cell anemia results in an abnormality in the ...

and cerebral

adrenoleukodystrophy Adrenoleukodystrophy (ALD) is a genetic disorder, disease linked to the X chromosome. It is a result of fatty acid buildup caused by failure of peroxisome#Metabolic functions, peroxisomal fatty acid beta oxidation which results in the accumulation ...

. It is also developing

T cell T cells (also known as T lymphocytes) are an important part of the immune system and play a central role in the adaptive immune response. T cells can be distinguished from other lymphocytes by the presence of a T-cell receptor (TCR) on their cell ...

product candidates to treat

acute myeloid leukemia Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells, characterized by the rapid growth of abnormal cells that build up in the bone marrow and blood and interfere with haematopoiesis, normal blood cell production. Sympt ...

Merkel-cell carcinoma Merkel cell carcinoma (MCC) is a rare and aggressive skin cancer occurring in about three people per million members of the population. It is also known as cutaneous APUDoma, primary neuroendocrine carcinoma of the skin, primary small cell ca ...

diffuse large B-cell lymphoma Diffuse large B-cell lymphoma (DLBCL) is a cancer of B cells, a type of lymphocyte that is responsible for producing antibodies. It is the most common form of non-Hodgkin lymphoma among adults, with an annual incidence of 7–8 cases per 100,000 ...

, and MAGEA4 solid tumors.

History

The company was founded as Genetix Pharmaceuticals in April 1992 by MIT faculty members Philippe Leboulch and Irving London. From 2001 to 2005, Walter Ogier was chief executive officer of Genetix. At that time, the company was focused on the development of LentiglobinTM for the treatment of sickle cell disease and thalassemia major (beta-thalassemia), the two most globally prevalent severe human genetic diseases. In September 2010, preliminary results of clinical trials of LentiglobinTM at Hospital Necker in Paris, France, were published in ''

Nature Nature is an inherent character or constitution, particularly of the Ecosphere (planetary), ecosphere or the universe as a whole. In this general sense nature refers to the Scientific law, laws, elements and phenomenon, phenomena of the physic ...

'' by Marina Cavazzana and company founder Philippe Leboulch. Stable transfusion (21 months) independence was successfully achieved by a patient with severe beta-thalassemia who had been treated with Lentiglobin 2 years earlier. This represented the first-ever long term correction of a major human genetic disease by gene therapy. Also in September 2010, the company was renamed bluebird bio and Nick Leschly was named chief executive officer. In June 2013, the company became a

public company A public company is a company whose ownership is organized via shares of share capital, stock which are intended to be freely traded on a stock exchange or in over-the-counter (finance), over-the-counter markets. A public (publicly traded) co ...

via an

initial public offering An initial public offering (IPO) or stock launch is a public offering in which shares of a company are sold to institutional investors and usually also to retail (individual) investors. An IPO is typically underwritten by one or more investm ...

, raising $116 million. In June 2014, the company acquired Precision Genome Engineering Inc. for up to $156 million. In November 2017,

Celgene Celgene Corporation, headquartered in Summit, New Jersey, was a pharmaceutical company that produced cancer and immunology drugs. Its primary products were Revlimid (lenalidomide), which is used in the treatment of multiple myeloma (63% of 2018 r ...

, now

Bristol-Myers Squibb The Bristol-Myers Squibb Company, doing business as Bristol Myers Squibb (BMS), is an American multinational pharmaceutical company. Headquartered in Princeton, New Jersey, BMS is one of the world's largest pharmaceutical companies and consist ...

(BMS), announced a collaboration with bluebird bio regarding bb2121 Anti-BCMA chimeric antigen receptor T cell therapy. In May 2020, the

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) issued a refusal to file letter to BMS and bluebird bio's marketing application seeking approval of idecabtagene vicleucel (ide-cel) for patients with heavily pre-treated relapsed and refractory

multiple myeloma Multiple myeloma (MM), also known as plasma cell myeloma and simply myeloma, is a cancer of plasma cells, a type of white blood cell that normally produces antibody, antibodies. Often, no symptoms are noticed initially. As it progresses, bone ...

. In September 2020, the FDA accepted bluebird's marketing application for ide-cel in and established a PDUFA goal date of March 27, 2021. Ide-cel is a BCMA-directed genetically modified autologous CAR-T-cell immunotherapy. In August 2018, the company announced a collaboration with Regeneron Pharmaceuticals to discover, develop and commercialize new cell therapies for cancer. In November 2021, the company completed the

corporate spin-off A corporate spin-off, also known as a spin-out, starburst or hive-off, is a type of corporate action where a company "splits off" a section as a separate business or creates a second incarnation, even if the first is still active. It is distinct ...

of 2seventy bio, focused on

oncology Oncology is a branch of medicine that deals with the study, treatment, diagnosis, and prevention of cancer. A medical professional who practices oncology is an ''oncologist''. The name's Etymology, etymological origin is the Greek word ὄγ ...

. In 2022 and 2024, the company laid off 25-30% of its staff. In June 2025,

s managed by

and SK Capital Partners acquired the company and increased its funding.

Products

* Zynteglo: In June 2019, the company received approval from the

European Commission The European Commission (EC) is the primary Executive (government), executive arm of the European Union (EU). It operates as a cabinet government, with a number of European Commissioner, members of the Commission (directorial system, informall ...

to market in the EU betibeglogene autotemcel (Zynteglo), a medication for the treatment of the

group of inherited blood disorders. Bluebird received the FDA's approval for the treatment in August 2022. The FDA granted Zynteglo

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

and breakthrough therapy designations for the treatment of TDT. * Skysona: In July 2021 the company received approval from the

to market elivaldogene autotemcel (Skysona) for the treatment of

. * Lyfgenia: In December 2023, the company received FDA approval of lovo-cel for the treatment of sickle cell disease in patients 12 years and older who have a history of vaso-occlusive events, with an announced wholesale acquisition cost of $3.1 million. Lyfgenia carries a boxed warning of potential risk for hematologic malignancy (blood cancer) in treated patients. bluebird has introduced outcomes-based contracts for Lyfgenia; these contracts involve a three-year patient monitoring period following treatment, during which payers will not be required to pay the full price if a patient is hospitalized due to vasco-occlusive events related to sickle cell disease. Since Lyfgenia's approval, bluebird has successfully secured two significant reimbursement agreements with distinct insurance providers, collectively providing coverage for approximately 200 million individuals in the United States.

References

External links

* {{authority control 1992 establishments in Massachusetts 2013 initial public offerings 2025 mergers and acquisitions American companies established in 1992 Biotechnology companies of the United States Companies based in Cambridge, Massachusetts Companies formerly listed on the Nasdaq Pharmaceutical companies established in 1992