CRISPR Therapeutics
{{short description|Swiss-American biotechnology company}}
{{Use dmy dates|date=February 2021}}
{{Infobox company
| name = CRISPR Therapeutics AG
| logo = File:CRISPR Therapeutics logo.svg
| logo_size = 200px
| type = Public
| traded_as = {{NASDAQ|CRSP}}
| ISIN = {{ISIN|sl=n|pl=y|CH0334081137}}
| former_name = Inception Genomics
| predecessor =
| founders = {{Unbulleted list|Emmanuelle Charpentier| Shaun Foy| Rodger Novak}}
| foundation = {{Start date and age|2013}}
| location = Zug, Switzerland
| area_served =
| key_people = Samarth Kulkarni (CEO)
| industry = Biotechnology
| products =
| services =
| revenue = {{nowrap|{{decrease}} US$37.3 million (2024)}}
| operating_income = {{nowrap|{{decrease}} US$−467 million (2024)}}
| net_income = {{nowrap|{{decrease}} US$−366 million (2024)}}
| assets = {{increase}} US$2.24 billion (2024)
| equity = {{increase}} US$1.93 billion (2024)
| num_employees = 393 (2024)
| divisions =
| homepage = {{URL|crisprtx.com}}
| footnotes = {{cite web |url=https://www.sec.gov/ix?doc=/Archives/edgar/data/1674416/000095017025017899/crsp-20241231.htm |title=CRISPR Therapeutics AG 20243 Annual Report |date=2025-02-11 |publisher=U.S. Securities and Exchange Commission}}
}}
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.{{Cite web|title=CRISPR Therapeutics, Vertex Start First Company-Backed Human CRISPR Trial|url=https://www.fiercebiotech.com/biotech/crispr-therapeutics-vertex-start-first-company-backed-human-crispr-trial|website=FierceBiotech|date=August 31, 2018}}{{Cite web|title=A Young Mississippi Woman's Journey Through A Pioneering Gene-Editing Experiment|url=https://www.npr.org/sections/health-shots/2019/12/25/784395525/a-young-mississippi-womans-journey-through-a-pioneering-gene-editing-experiment|website=NPR|date=December 25, 2019}} 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.{{Cite web|title=2022 Category Winner for Innovation|url=https://ispe.org/facility-year-awards/winners/2022/innovation|website=ISPE|date=2022}} 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.{{Cite web|title=F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPR|url=https://www.nytimes.com/2023/12/08/health/fda-sickle-cell-crispr.html?smid=nytcore-android-share|website=New York Times|date=December 8, 2023}}
History
CRISPR Therapeutics was founded in 2013 by Emmanuelle Charpentier, Shaun Foy and Rodger Novak.{{cite magazine |last=Arney |first=Kat |date=22 June 2016 |title=Disease is in the sights of gene reprogrammers |url=https://www.wired.co.uk/article/gene-editing-crispr |magazine=Wired UK}} 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.{{Cite web|title=CRISPR Therapeutics: Ist diese Aktie nobelpreiswürdig?|url=https://www.godmode-trader.de/artikel/crispr-therapeutics-ist-diese-aktie-nobelpreiswuerdig,8772188?|access-date=8 January 2021|website=GodmodeTrader|language=de}} The CEO is Samarth Kulkarni, PhD, who joined the company in 2015 as chief business officer. Kulkarni became CEO in 2017.{{cn|date=February 2024}}
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.{{Cite web|title=CRISPR Partnerships Seek Win-Win Situations|url=https://www.genengnews.com/topics/genome-editing/crispr-partnerships-seek-win-win-situations/|website=Genetic Engineering & Biotechnology News|date=March 3, 2022}} 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.{{Cite web|title=Vertex Dives into DMD with Exonics Acquisition and CRISPR Therapeutics Partnership Expansion|url=https://www.biospace.com/article/vertex-dives-into-dmd-with-exonics-acquisition-and-expansion-of-partnership-with-crispr-therapeutics|website=Biospace|date=June 7, 2019}}{{Cite web|title=Vertex doubles down on CRISPR Therapeutics and diabetes in new $100M deal|url=https://www.fiercebiotech.com/biotech/vertex-goes-back-crispr-well-paying-100m-upfront-exa-cel-partner-help-diabetes-work|website=Fierce Biotech|date=March 27, 2023}} 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.{{Cite web|title=Bayer backs off running Casebia JV as CRISPR Tx takes over management|url=https://www.fiercebiotech.com/biotech/bayer-backs-off-running-casebia-jv-as-crispr-tx-takes-over-management|access-date=8 January 2021|website=FierceBiotech|language=en}} The company went public on the NASDAQ exchange in October 2016.{{Cite web|title=Crispr's Nobel prize and IPOs both happened at lightning speed|url=https://qz.com/1914415/why-crisprs-nobels-and-ipos-both-happened-in-record-time|website=Quartz|date=October 7, 2020}}
CRISPR Therapeutics has established partnerships with additional companies such as Viacyte (part of Vertex), Nkarta, Capsida, Curevac, and others.{{Cite web|title=CRISPR and Capsida Partner in Gene Edited Therapies for ALS, Friedreich's Ataxia|url=https://www.biospace.com/article/crispr-and-capsida-partner-in-gene-edited-therapies-for-als-friedreich-s-ataxia|website=BioSpace|date=June 15, 2021}}{{Cite web|title=CRISPR Therapeutics inks deal with Nkarta on gene-edited cell therapies for cancer|url=https://www.thepharmaletter.com/article/crispr-therapeutics-inks-deal-with-nkarta-on-gene-edited-cell-therapies-for-cancer|website=The Pharma Letter|date=July 5, 2021}}{{Cite web|title=CRISPR, Bayer JV tap CureVac for Cas9 mRNA constructs|url=https://www.fiercebiotech.com/biotech/crispr-bayer-jv-taps-curevac-for-cas9-mrna-constructs|website=Fierce Biotech|date=November 13, 2017}}
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, that received FDA approval for clinical use in December 2023. 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.[https://investors.vrtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-pharmaceuticals-announce-fda 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 {{asof|2022}} Phase 3 clinical trial results supported the safety and efficacy of this treatment.{{Cite web|title=Vertex, CRISPR strengthen case for pioneering gene-editing treatment|url=https://www.biopharmadive.com/news/vertex-crispr-gene-editing-sickle-beta-thalassemia-eha-data/625327/|website=Biopharma Dive|date=June 11, 2022}}{{Cite journal|last1=Frangoul|first1=Haydar|last2=Altshuler|first2=David|last3=Cappellini|first3=M. Domenica|last4=Chen|first4=Yi-Shan|last5=Domm|first5=Jennifer|last6=Eustace|first6=Brenda K.|last7=Foell|first7=Juergen|last8=de la Fuente|first8=Josu|last9=Grupp|first9=Stephan|last10=Handgretinger|first10=Rupert|last11=Ho|first11=Tony W.|date=2021-01-21|title=CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia|journal=New England Journal of Medicine|volume=384|issue=3|pages=252–260|doi=10.1056/NEJMoa2031054|issn=0028-4793|pmid=33283989|s2cid=227521558 |doi-access=free}}{{Cite web|title=Synthego {{!}} Full Stack Genome Engineering|url=https://www.synthego.com/blog/sickle-cell-ctx001|access-date=2021-02-21|website=www.synthego.com|language=en}}{{Cite web|title=1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive|url=https://www.npr.org/sections/health-shots/2020/12/15/944184405/1st-patients-to-get-crispr-gene-editing-treatment-continue-to-thrive|access-date=2021-02-21|website=NPR.org|language=en}} 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 {{asof|April 2023|lc=y}}.{{Cite journal|title=First CRISPR therapy seeks landmark approval|url=https://www.nature.com/articles/d41573-023-00050-8|journal=Nature Reviews Drug Discovery|date=April 3, 2023|doi=10.1038/d41573-023-00050-8 |last1=Kingwell |first1=Katie |volume=22 |issue=5 |pages=339–341 |pmid=37012339 }}