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bluebird bio

bluebird bio, Inc.
Company typePublic
Nasdaq: BLUE
Industry
FoundedApril 16, 1992; 32 years ago (1992-04-16)
Founders
  • Philippe Leboulch
  • Irving London
HeadquartersSomerville, Massachusetts, U.S.
Key people
  • Andrew Obenshain (President & CEO)
  • Chris Krawtschuk (CFO)
ProductsZynteglo, Skysona, Lyfgenia
RevenueIncrease US$3.66 million (2021)
Decrease US$−819 million (2021)
Total assetsDecrease US$594 million (2021)
Total equityDecrease US$374 million (2021)
Number of employees
281 (September 2024)
Websitewww.bluebirdbio.com Edit this at Wikidata
Footnotes / references
[1]

bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders.[1]

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.[2] The company has been criticized for the $1.8 million cost of the drug, which is the second most expensive drug in the world.[3][4][5]

The company is developing LentiGlobin[6] 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.[1]

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 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.[8]

Also in September 2010, the company was renamed bluebird bio and Nick Leschly was named chief executive officer.[9]

In June 2013, the company became a public company via an initial public offering, raising $116 million.[10]

In June 2014, the company acquired Precision Genome Engineering Inc. for up to $156 million.[11]

In November 2017, Celgene, now Bristol-Myers Squibb (BMS), announced a collaboration with bluebird bio regarding bb2121 Anti-BCMA CAR-T Cell Therapy.[12] 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.[13] In September 2020, the FDA accepted bluebird's marketing application for ide-cel in and established a PDUFA goal date of March 27, 2021.[14] 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.[15]

On October 8, 2021, bluebird bio, Inc. announced the impending spinoff of a new public company, 2seventy bio, which would be focused on oncology that officially launched on November 4th, 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.[16]

bluebird bio has faced two major rounds of layoffs, letting go of 30% and 25% of its workforce in 2022 and 2024, respectively.[17] As of September 2024, the company has an estimated 281 employees.[17]

Products

  • Zynteglo: In June 2019, the company received approval from the European Commission to market in the EU betibeglogene autotemcel (Zynteglo), a medication for the treatment of the beta thalassemia group of inherited blood disorders.[18] Bluebird received the FDA's approval for the treatment in August 2022.[19] The FDA granted Zynteglo orphan drug and breakthrough therapy designations for the treatment of TDT.[18][20][21]
  • Skysona: In July 2021 the company received approval from the European Commission to market elivaldogene autotemcel (Skysona) for the treatment of adrenoleukodystrophy.[22]
  • 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.[23] Lyfgenia carries a boxed warning of potential risk for hematologic malignancy (blood cancer) in treated patients.[24] 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.[25] 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.[26]

References

  1. ^ a b c "bluebird bio, Inc. 2021 Annual Report (Form 10-K)". U.S. Securities and Exchange Commission. March 4, 2022.
  2. ^ "Zynteglo". European Medicines Agency. 3 June 2019.
  3. ^ Lovelace, Berkeley (June 14, 2019). "Bluebird Bio CEO defends $1.8 million price for gene therapy — 'It's really thinking about it differently'". CNBC.
  4. ^ LaMattina, John (19 June 2019). "What Bluebird Bio Gets Wrong In Pricing For Its $1.8 Million Drug". Forbes.
  5. ^ Kolata, Gina (September 11, 2017). "New Gene-Therapy Treatments Will Carry Whopping Price Tags". The New York Times.
  6. ^ "Anemia Treatment Drugs: 2019 Global Market Study; Analyzed by Type of Anemia, Type of Drug, and Geography". GlobeNewswire (Press release). 2019-04-26. Retrieved 2023-08-02.
  7. ^ "ChartEXE: Market Data Aggregation Service". ChartEXE: Market Data Aggregation Service. Retrieved 2021-01-07.
  8. ^ Cavazzana-Calvo, Marina; Payen, Emmanuel (September 16, 2010). "Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia". Nature. 467 (7313): 318–322. doi:10.1038/nature09328. PMC 3355472. PMID 20844535.
  9. ^ "Genetix Pharmaceuticals Renamed bluebird bio; Announces Appointment of Nick Leschly as Chief Executive Officer" (Press release). bluebird bio. September 9, 2010.
  10. ^ Alspach, Kyle (June 25, 2013). "Bluebird bio IPO closes with $116M raised". American City Business Journals.
  11. ^ Resende, Patricia (July 1, 2014). "Bluebird bio in deal to buy Seattle biotech for up to $156M". American City Business Journals.
  12. ^ 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
  13. ^ 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
  14. ^ "FDA Accepts BLA for bluebird and BMS' Multiple Myeloma CAR-T Treatment". BioSpace. 22 September 2020. Retrieved 2021-01-07.
  15. ^ "Regeneron and bluebird bio Announce Collaboration to Discover, Develop and Commercialize New Cell Therapies for Cancer" (Press release). PR Newswire. August 6, 2018.
  16. ^ Business Wire:October 8th, 2021:Bluebird Bio Provides Update on Upcoming Planned Business Separation
  17. ^ a b www.bizjournals.com https://www.bizjournals.com/boston/news/2024/09/24/bluebird-bio-layoffs-breakeven.html. Retrieved 2024-09-24. {{cite web}}: Missing or empty |title= (help)
  18. ^ a b "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.
  19. ^ Roy, Mrinalika (17 August 2022). "Bluebird's $2.8 million gene therapy becomes most expensive drug after U.S. approval". Reuters.
  20. ^ Our Products, Company Website, accessdate: May 9, 2020
  21. ^ "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.
  22. ^ Fidler, Ben (21 July 2021). "Bluebird, with little fanfare, is first to bring a second gene therapy to market". Industry Dive. Retrieved 22 November 2021.
  23. ^ Leo, Leroy; Satija, Bhanvi; Satija, Bhanvi (2023-12-08). "US FDA approves two gene therapies for sickle cell disease". Reuters. Retrieved 2023-12-11.
  24. ^ Commissioner, Office of the (2023-12-08). "FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease". FDA. Retrieved 2024-01-12.
  25. ^ BioSpace (2024-01-05). "Bluebird secures second big outcomes-based coverage contract for Lyfgenia". PharmaLive. Retrieved 2024-01-12.
  26. ^ BioSpace (2024-01-05). "Bluebird secures second big outcomes-based coverage contract for Lyfgenia". PharmaLive. Retrieved 2024-01-12.
Quelle: https://en.wikipedia.org/wiki/Bluebird_Bio