Company:CRISPR Therapeutics

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Short description: Swiss-American biotechnology company
CRISPR Therapeutics AG
FormerlyInception Genomics
TypePublic company
NASDAQ: CRSP
ISIN[https://handwiki.org/wiki/index.php?title=Toollabs:isin/&language=en&isin=CH0334081137 CH0334081137]
IndustryBiotechnology
Founded2013; 11 years ago (2013)
Founders
HeadquartersZug, Switzerland
Key people
Samarth Kulkarni
(CEO)
RevenueDecrease US$1.2 million (2022)
Decrease US$−673 million (2022)
Decrease US$−650 million (2022)
Total assetsDecrease US$2.24 billion (2022)
Total equityDecrease US$1.88 billion (2022)
Number of employees
458 (2022)
Websitecrisprtx.com
Footnotes / references
[1]

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 in December 2023.[5]

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 current CEO of the company is Samarth Kulkarni, PhD, who joined the company in 2015 as Chief Business Officer. Kulkarni became CEO in 2017.

CRISPR Therapeutics has formed various 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 potential treatments to emerge from the joint research program was CTX001 or exagamglogene autotemcel (commonly known as Exa-cel). Subsequently, CRISPR Therapeutics and Vertex have 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, and started to operate Casebia Therapeutics as a joint venture with Bayer, which is now operated under the management of CRISPR Therapeutics.[11] The company went public on the NASDAQ exchange in October of 2016.[12]

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

Products

CRISPR Therapeutics has several drugs in development for blood diseases, cancer, diabetes, and other severe diseases. These include the drug exa-cel for the treatment of the rare blood disorders Beta thalassemia and sickle cell disease, which is being developed jointly with Vertex Pharmaceuticals.[7] In May 2020, exa-cel received Orphan drug Designation from the U.S. Food and Drug Administration for transfusion-dependent beta thalassemia and from the European Medicines Agency for sickle cell disease and transfusion-dependent beta thalassemia.[16] Phase 3 clinical trial results support the safety and efficacy of this treatment.[17][18][19][20] The rolling Biologics License Applications (BLAs) were submitted to the U.S. Food and Drug Administration (FDA) for exa-cel for sickle cell disease and transfusion-dependent beta thalassemia. Additionally, EU and UK filings were completed in 2022, and these submissions were validated by European Medicines Agency and The Medicines and Healthcare products Regulatory Agency.[21]

See also

References

  1. ↑ "CRISPR Therapeutics AG 2022 Annual Report". U.S. Securities and Exchange Commission. 2023-02-21. https://www.sec.gov/ix?doc=/Archives/edgar/data/1674416/000095017023003441/crsp-20221231.htm. 
  2. ↑ "CRISPR Therapeutics, Vertex Start First Company-Backed Human CRISPR Trial". August 31, 2018. https://www.fiercebiotech.com/biotech/crispr-therapeutics-vertex-start-first-company-backed-human-crispr-trial. 
  3. ↑ "A Young Mississippi Woman's Journey Through A Pioneering Gene-Editing Experiment". December 25, 2019. https://www.npr.org/sections/health-shots/2019/12/25/784395525/a-young-mississippi-womans-journey-through-a-pioneering-gene-editing-experiment. 
  4. ↑ "2022 Category Winner for Innovation". 2022. https://ispe.org/facility-year-awards/winners/2022/innovation. 
  5. ↑ "F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPR". December 8, 2023. https://www.nytimes.com/2023/12/08/health/fda-sickle-cell-crispr.html?smid=nytcore-android-share. 
  6. ↑ Arney, Kat (22 June 2016). "Disease is in the sights of gene reprogrammers". Wired UK. https://www.wired.co.uk/article/gene-editing-crispr. 
  7. ↑ 7.0 7.1 "CRISPR Therapeutics: Ist diese Aktie nobelpreiswürdig?" (in de). https://www.godmode-trader.de/artikel/crispr-therapeutics-ist-diese-aktie-nobelpreiswuerdig,8772188?. 
  8. ↑ "CRISPR Partnerships Seek Win-Win Situations". March 3, 2022. https://www.genengnews.com/topics/genome-editing/crispr-partnerships-seek-win-win-situations/. 
  9. ↑ "Vertex Dives into DMD with Exonics Acquisition and CRISPR Therapeutics Partnership Expansion". June 7, 2019. https://www.biospace.com/article/vertex-dives-into-dmd-with-exonics-acquisition-and-expansion-of-partnership-with-crispr-therapeutics. 
  10. ↑ "Vertex doubles down on CRISPR Therapeutics and diabetes in new $100M deal". March 27, 2023. https://www.fiercebiotech.com/biotech/vertex-goes-back-crispr-well-paying-100m-upfront-exa-cel-partner-help-diabetes-work. 
  11. ↑ "Bayer backs off running Casebia JV as CRISPR Tx takes over management" (in en). https://www.fiercebiotech.com/biotech/bayer-backs-off-running-casebia-jv-as-crispr-tx-takes-over-management. 
  12. ↑ "Crispr's Nobel prize and IPOs both happened at lightning speed". October 7, 2020. https://qz.com/1914415/why-crisprs-nobels-and-ipos-both-happened-in-record-time. 
  13. ↑ "CRISPR and Capsida Partner in Gene Edited Therapies for ALS, Friedreich's Ataxia". June 15, 2021. https://www.biospace.com/article/crispr-and-capsida-partner-in-gene-edited-therapies-for-als-friedreich-s-ataxia. 
  14. ↑ "CRISPR Therapeutics inks deal with Nkarta on gene-edited cell therapies for cancer". July 5, 2021. https://www.thepharmaletter.com/article/crispr-therapeutics-inks-deal-with-nkarta-on-gene-edited-cell-therapies-for-cancer. 
  15. ↑ "CRISPR, Bayer JV tap CureVac for Cas9 mRNA constructs". November 13, 2017. https://www.fiercebiotech.com/biotech/crispr-bayer-jv-taps-curevac-for-cas9-mrna-constructs. 
  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. ↑ "Vertex, CRISPR strengthen case for pioneering gene-editing treatment". June 11, 2022. https://www.biopharmadive.com/news/vertex-crispr-gene-editing-sickle-beta-thalassemia-eha-data/625327/. 
  18. ↑ Frangoul, Haydar; Altshuler, David; Cappellini, M. Domenica; Chen, Yi-Shan; Domm, Jennifer; Eustace, Brenda K.; Foell, Juergen; de la Fuente, Josu et al. (2021-01-21). "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. 
  19. ↑ "Synthego | Full Stack Genome Engineering" (in en). https://www.synthego.com/blog/sickle-cell-ctx001. 
  20. ↑ "1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive" (in en). https://www.npr.org/sections/health-shots/2020/12/15/944184405/1st-patients-to-get-crispr-gene-editing-treatment-continue-to-thrive. 
  21. ↑ "First CRISPR therapy seeks landmark approval". April 3, 2023. https://www.nature.com/articles/d41573-023-00050-8. 



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