bluebird bio

bluebird bio, Inc., based in Cambridge, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders and cancer.[2]

bluebird bio, Inc.
TypePublic company
NASDAQ: BLUE
IndustryBiotechnology
Pharmaceutical industry
FoundedApril 16, 1992 (1992-04-16)
FoundersPhilippe Leboulch and Irving London
HeadquartersCambridge, Massachusetts, U.S.
Key people
Nick Leschly, President & CEO
Chip Baird, CFO
ProductsZynteglo
Revenue $220 million (2020)[1]
-$789 million (2019)
Total assets $1.727 billion (2019)
Total equity $1.284 billion (2019)
Number of employees
1,090 (2019)
Websitewww.bluebirdbio.com
Footnotes / references
[2]

The company's only - in the European Union (EU) - approved drug 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.[3] The company has been criticized for the $1.8 million cost of the drug, which is the second most expensive drug in the world.[4][5][6]

The company is developing LentiGlobin gene therapy for the treatment of sickle cell disease and cerebral adrenoleukodystrophy. It is also developing T cell product candidates to treat acute myeloid leukemia, Merkel-cell carcinoma, diffuse large B-cell lymphoma, and MAGEA4 solid tumors.[2]

History

The company was founded as Genetix Pharmaceuticals in April 1992 by MIT faculty members Philippe Leboulch and Irving London.[7]

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 Cavazzano-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.[8]

In June 2013, the company became a public company via an initial public offering, raising $116 million.[9]

In June 2014, the company acquired Precision Genome Engineering Inc. for up to $156 million.[10]

In November 2017, Celgene, now Bristol-Myers Squibb (BMS), announced a collaboration with bluebird bio regarding bb2121 Anti-BCMA CAR-T Cell Therapy.[11] In May 2020 the 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.[12] In September 2020, the FDA accepted bluebird’s marketing application for ide-cel in and established a PDUFA goal date of March 27, 2021.[13] 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 to discover, develop and commercialize new cell therapies for cancer.[14]

Products

References
  1. http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-reports-second-quarter-2020-financial-results-and
  2. "bluebird bio, Inc. 2019 Form 10-K Annual Report". U.S. Securities and Exchange Commission.
  3. "Zynteglo". European Medicines Agency.
  4. Lovelace, Berkeley (June 14, 2019). "Bluebird Bio CEO defends $1.8 million price for gene therapy — 'It's really thinking about it differently'". CNBC.
  5. LaMattina, John (19 June 2019). "What Bluebird Bio Gets Wrong In Pricing For Its $1.8 Million Drug". Forbes.
  6. Kolata, Gina (September 11, 2017). "New Gene-Therapy Treatments Will Carry Whopping Price Tags". The New York Times.
  7. "ChartEXE: Market Data Aggregation Service". ChartEXE: Market Data Aggregation Service. Retrieved 2021-01-07.
  8. "Genetix Pharmaceuticals Renamed bluebird bio; Announces Appointment of Nick Leschly as Chief Executive Officer" (Press release). bluebird bio. September 9, 2010.
  9. Alspach, Kyle (June 25, 2013). "Bluebird bio IPO closes with $116M raised". American City Business Journals.
  10. Resende, Patricia (July 1, 2014). "Bluebird bio in deal to buy Seattle biotech for up to $156M". American City Business Journals.
  11. Celgene Corporation and bluebird bio Announce bb2121 Anti-BCMA CAR-T Cell Therapy Has Been Granted Breakthrough Therapy Designation from FDA and Prime Eligibility from EMA for Relapsed and Refractory Multiple Myeloma, PM Celgene November 16, 2017, retrieved May 14, 2020
  12. Bristol Myers Squibb and bluebird bio Provide Regulatory Update on Idecabtagene Vicleucel (ide-cel, bb2121) for the Treatment of Patients with Multiple Myeloma, PM BMS May 13, 2020, retrieved May 14, 2020
  13. "FDA Accepts BLA for bluebird and BMS' Multiple Myeloma CAR-T Treatment". BioSpace. Retrieved 2021-01-07.
  14. "Regeneron and bluebird bio Announce Collaboration to Discover, Develop and Commercialize New Cell Therapies for Cancer" (Press release). PR Newswire. August 6, 2018.
  15. "bluebird bio Announces EU Conditional Marketing Authorization for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype" (Press release). Business Wire. June 3, 2019.
  16. Our Products, Company Website, accessdate: May 9, 2020
  17. "Autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human BA-T87Q-globin gene Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 18 March 2013. Retrieved 8 June 2020.
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