bluebird bio, Inc., based in

Somerville, Massachusetts Somerville ( ) is a city located directly to the northwest of Boston, and north of Cambridge, in Middlesex County, Massachusetts, United States. As of the 2020 United States Census, the city had a total population of 81,045 people. With an area ...

, is a

biotechnology Biotechnology is the integration of natural sciences and engineering sciences in order to achieve the application of organisms, cells, parts thereof and molecular analogues for products and services. The term ''biotechnology'' was first used b ...

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 chromosomal abnormality. Although polygenic disorde ...

s. The company's only - in the

European Union The European Union (EU) is a supranational political and economic union of member states that are located primarily in Europe. The union has a total area of and an estimated total population of about 447million. The EU has often been ...

(EU) - approved drug is

betibeglogene autotemcel Betibeglogene autotemcel, sold under the brand name Zynteglo, is a medication for the treatment for beta thalassemia. It was developed by Bluebird Bio and was given breakthrough therapy designation by the U.S. Food and Drug Administration in Febr ...

(Zynteglo), which treats transfusion-dependent beta thalassemia (TDT), a rare genetic blood disorder, and has been approved for use by the European Medicines Agency. 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) is a group of blood disorders typically inherited from a person's parents. The most common type is known as sickle cell anaemia. It results in an abnormality in the oxygen-carrying protein haemoglobin found in red b ...

and cerebral

adrenoleukodystrophy Adrenoleukodystrophy (ALD) is a disease linked to the X chromosome. It is a result of fatty acid buildup caused by peroxisomal fatty acid beta oxidation which results in the accumulation of very long chain fatty acids in tissues throughout the b ...

. It is also developing

T cell A T cell is a type of lymphocyte. T cells are one of the important white blood cells 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 ...

product candidates to treat acute myeloid leukemia, Merkel-cell carcinoma, diffuse large B-cell lymphoma, and MAGEA4 solid tumors.

History

The company was founded as Genetix Pharmaceuticals in April 1992 by MIT faculty members Philippe Leboulch and Irving London. In 2001, Walter Ogier was appointed chief executive officer of Genetix Pharmaceuticals, and 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 the journal Nature by Drs. Marina Cavazzana-Calvo and Philippe Leboulch, scientific founder of Genetix Pharmaceuticals / bluebird bio. Stable (21 months) transfusion independence had been 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. Nature 467, 318–322 (2010) 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 stock which are intended to be freely traded on a stock exchange or in over-the-counter markets. A public (publicly traded) company can be listed on a stock exchange ( l ...

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 investme ...

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

Bristol-Myers Squibb The Bristol Myers Squibb Company (BMS) is an American multinational pharmaceutical company. Headquartered in New York City, BMS is one of the world's largest pharmaceutical companies and consistently ranks on the ''Fortune'' 500 list of the l ...

(BMS), announced a collaboration with bluebird bio regarding bb2121 Anti-BCMA CAR-T Cell Therapy. In May 2020, the

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food ...

(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 antibodies. Often, no symptoms are noticed initially. As it progresses, bone pain, ane ...

. In September 2020, the FDA accepted bluebird's marketing application for ide-cel in and established a PDUFA goal date of March 27, 2021. US approval of ide-cel by March 31, 2021, is one of the required remaining milestones of the contingent value rights (CVR) issued upon the close of Bristol Myers Squibb's purchase of Celgene in 2019. Ide-cel is a BCMA-directed genetically modified autologous CAR-T-cell immunotherapy. In August 2018, the company announced a collaboration with

Regeneron Pharmaceuticals Regeneron Pharmaceuticals, Inc. is an American biotechnology company headquartered in Westchester County, New York. The company was founded in 1988. Originally focused on neurotrophic factors and their regenerative capabilities, giving rise to ...

to discover, develop and commercialize new cell therapies for cancer. On October 8, 2021, bluebird bio, Inc. announced the impending spinoff of a new public company 2seventy bio focused on oncology. The spin-off is expected to be completed by early November 2021. bluebird bio's corporate filing announced they have appointed Najoh Tita-Reid and Sarah Glickman to the bluebird bio board of directors. Ms. Glickman will be a member of the board of directors of 2seventy bio and will step down from the bluebird bio board of directors when the new arrangement goes into effect.

Products

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

European Commission The European Commission (EC) is the executive of the European Union (EU). It operates as a cabinet government, with 27 members of the Commission (informally known as "Commissioners") headed by a President. It includes an administrative body ...

to market in the EU

(Zynteglo), a medication for the treatment of the beta thalassemia 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 pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment o ...

and

breakthrough therapy Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's " ...

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

to market

elivaldogene autotemcel Elivaldogene autotemcel, sold under the brand name Skysona, is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD). It was developed by Bluebird bio and was given breakthrough therapy designation by the U.S. Food and Drug Administ ...

(Skysona) for the treatment of

References

External links

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