CRISPR_Therapeutics

CRISPR Therapeutics

CRISPR Therapeutics

Swiss-American biotechnology company

CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases.[2][3] The company has approximately 500 employees and has offices in Zug, Switzerland, Boston, Massachusetts, San Francisco, California and London, United Kingdom. Its manufacturing facility in Framingham, Massachusetts won the Facilities of the Year Award (FOYA) award in 2022.[4] The company’s lead program, exagamglogene autotemcel, or exa-cel (formerly CTX001), was granted regulatory approval by the US Food and Drug Administration (FDA) in December 2023.[5]

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History

CRISPR Therapeutics was founded in 2013 by Emmanuelle Charpentier, Shaun Foy and Rodger Novak.[6] Charpentier later shared the Nobel Prize in Chemistry in 2020 with Jennifer Doudna. As part of a working group, she provided the first scientific documentation on the development and use of CRISPR gene editing. This allows DNA to be specifically modified and edited, which can be used to ameliorate diseases. CRISPR Therapeutics is applying this technology platform to research, develop and commercialize medicines for various diseases including sickle cell disease, beta thalassemia, various cancers, type 1 diabetes, and cardiovascular diseases.[7] The CEO is Samarth Kulkarni, PhD, who joined the company in 2015 as chief business officer. Kulkarni became CEO in 2017.[citation needed]

CRISPR Therapeutics has formed collaborations in support of its mission of developing medicines. Vertex Pharmaceuticals and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease.[8] One of the first treatments to emerge from the joint research program was CTX001 or exagamglogene autotemcel (commonly known as Exa-cel). Subsequently, CRISPR Therapeutics and Vertex expanded their collaboration to include diseases like Duchenne muscular dystrophy and type 1 diabetes.[9][10] In 2016, the company signed an agreement with Bayer AG to operate Casebia Therapeutics as a joint venture with Bayer under the management of CRISPR Therapeutics.[11] The company went public on the NASDAQ exchange in October 2016.[12]

CRISPR Therapeutics has established partnerships with additional companies such as Viacyte (part of Vertex), Nkarta, Capsida, Curevac, and others.[13][14][15]

Products

CRISPR Therapeutics has drugs approved and in development for blood diseases, cancer, diabetes, and other severe diseases.

Exa-cel

Exa-cel is a drug therapy for the treatment of the rare blood disorders beta thalassemia and sickle cell disease developed jointly with Vertex Pharmaceuticals,[7] that received FDA approval for clinical use in December 2023.[5] In May 2020, exa-cel had received orphan drug designation from the US Food and Drug Administration (FDA) for transfusion-dependent beta thalassemia and from the European Medicines Agency for sickle cell disease and transfusion-dependent beta thalassemia.[16] As of 2022 Phase 3 clinical trial results supported the safety and efficacy of this treatment.[17][18][19][20] The rolling Biologics License Applications (BLAs) were submitted to the FDA for exa-cel for sickle cell disease and transfusion-dependent beta thalassemia. EU and UK filings were completed in 2022, and the submissions were validated by European Medicines Agency and The Medicines and Healthcare products Regulatory Agency as of April 2023.[21]

See also

References

  1. [1]
    "CRISPR Therapeutics AG 2023 Annual Report". U.S. Securities and Exchange Commission. 21 February 2024.
  2. [2]
    "CRISPR Therapeutics, Vertex Start First Company-Backed Human CRISPR Trial". FierceBiotech. 31 August 2018.
  3. [3]
    "A Young Mississippi Woman's Journey Through A Pioneering Gene-Editing Experiment". NPR. 25 December 2019.
  4. [4]
    "2022 Category Winner for Innovation". ISPE. 2022.
  5. [5]
    "F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPR". New York Times. 8 December 2023.
  6. [6]
    Arney, Kat (22 June 2016). "Disease is in the sights of gene reprogrammers". Wired UK.
  7. [7]
    "CRISPR Therapeutics: Ist diese Aktie nobelpreiswürdig?". GodmodeTrader (in German). Retrieved 8 January 2021.
  8. [8]
    "CRISPR Partnerships Seek Win-Win Situations". Genetic Engineering & Biotechnology News. 3 March 2022.
  9. [9]
    "Vertex Dives into DMD with Exonics Acquisition and CRISPR Therapeutics Partnership Expansion". Biospace. 7 June 2019.
  10. [10]
    "Vertex doubles down on CRISPR Therapeutics and diabetes in new $100M deal". Fierce Biotech. 27 March 2023.
  11. [11]
    "Bayer backs off running Casebia JV as CRISPR Tx takes over management". FierceBiotech. Retrieved 8 January 2021.
  12. [12]
    "Crispr's Nobel prize and IPOs both happened at lightning speed". Quartz. 7 October 2020.
  13. [13]
    "CRISPR and Capsida Partner in Gene Edited Therapies for ALS, Friedreich's Ataxia". BioSpace. 15 June 2021.
  14. [14]
    "CRISPR Therapeutics inks deal with Nkarta on gene-edited cell therapies for cancer". The Pharma Letter. 5 July 2021.
  15. [15]
    "CRISPR, Bayer JV tap CureVac for Cas9 mRNA constructs". Fierce Biotech. 13 November 2017.
  16. [16]
    CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001™ for the Treatment of Severe Hemoglobinopathies, PM Vertex 11 May 2020; retrieved 11 May 2020
  17. [17]
    "Vertex, CRISPR strengthen case for pioneering gene-editing treatment". Biopharma Dive. 11 June 2022.
  18. [18]
    Frangoul, Haydar; Altshuler, David; Cappellini, M. Domenica; Chen, Yi-Shan; Domm, Jennifer; Eustace, Brenda K.; Foell, Juergen; de la Fuente, Josu; Grupp, Stephan; Handgretinger, Rupert; Ho, Tony W. (21 January 2021). "CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia". New England Journal of Medicine. 384 (3): 252–260. doi:10.1056/NEJMoa2031054. ISSN 0028-4793. PMID 33283989. S2CID 227521558.
  19. [19]
    "Synthego | Full Stack Genome Engineering". www.synthego.com. Retrieved 21 February 2021.
  20. [20]
    "1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive". NPR.org. Retrieved 21 February 2021.
  21. [21]
    Kingwell, Katie (3 April 2023). "First CRISPR therapy seeks landmark approval". Nature Reviews Drug Discovery. 22 (5): 339–341. doi:10.1038/d41573-023-00050-8. PMID 37012339.
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