One of the company's approved drugs is betibeglogene autotemcel (Zynteglo), which treats transfusion-dependent beta thalassemia (TDT), a rare genetic blood disorder, and has been approved for use by the European Medicines Agency.[1] The company has been criticized for the $1.8 million cost of the drug, which is the second most expensive drug in the world.[2][3][4]
The company was founded as Genetix Pharmaceuticals in April 1992 by MIT faculty members Philippe Leboulch and Irving London.[6]
From 2001 to 2005, Walter Ogier was chief executive officer of Genetix.[7] 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 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.[8]
Also in September 2010, the company was renamed bluebird bio and Nick Leschly was named chief executive officer.[9]
In June 2014, the company acquired Precision Genome Engineering Inc. for up to $156 million.[12]
In November 2017, Celgene, now Bristol-Myers Squibb (BMS), announced a collaboration with bluebird bio regarding bb2121 Anti-BCMA chimeric antigen receptor T cell therapy.[13] In May 2020, the Food and Drug Administration (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.[14] In September 2020, the FDA accepted bluebird's marketing application for ide-cel in and established a PDUFA goal date of March 27, 2021.[15] 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.[16][17]
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.[27] Lyfgenia carries a boxed warning of potential risk for hematologic malignancy (blood cancer) in treated patients.[28] 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.[29] 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.[29]
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