CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in

Zug Zug (Standard German: , Alemannic German: ; ; ; ; )Named in the 16th century. is the largest List of cities in Switzerland, town and capital of the Swiss canton of Zug. Zug is renowned as a hub for some of the wealthiest individuals in the wor ...

Switzerland Switzerland, officially the Swiss Confederation, is a landlocked country located in west-central Europe. It is bordered by Italy to the south, France to the west, Germany to the north, and Austria and Liechtenstein to the east. Switzerland ...

. The company does business as CRISPR Therapeutics, Inc. in the United States. It was one of the first companies formed to utilize the

CRISPR gene editing CRISPR gene editing (; pronounced like "crisper"; an abbreviation for "clustered regularly interspaced short palindromic repeats") is a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified. ...

platform to develop medicines for the treatment of various rare and common diseases. 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. The company’s lead program,

exagamglogene autotemcel Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease and transfusion-dependent beta thalassemia. It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The tr ...

, or exa-cel (formerly CTX001), was granted regulatory approval by the US Food and Drug Administration (FDA) in December 2023.

History

CRISPR Therapeutics was founded in 2013 by

Emmanuelle Charpentier Emmanuelle Marie Charpentier (; born 11 December 1968) is a French professor and researcher in microbiology, genetics, and biochemistry. As of 2015, she has been a director at the Max Planck Institute for Infection Biology in Berlin. In 2018, sh ...

, Shaun Foy and Rodger Novak. Charpentier later shared the

Nobel Prize in Chemistry The Nobel Prize in Chemistry () is awarded annually by the Royal Swedish Academy of Sciences to scientists in the various fields of chemistry. It is one of the five Nobel Prizes established by the will of Alfred Nobel in 1895, awarded for outst ...

in 2020 with

Jennifer Doudna Jennifer Anne Doudna (; born February 19, 1964) is an American biochemist who has pioneered work in CRISPR gene editing, and made other fundamental contributions in biochemistry and genetics. She received the 2020 Nobel Prize in Chemistry, wit ...

. As part of a working group, she provided the first scientific documentation on the development and use of

. 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. The CEO is Samarth Kulkarni, PhD, who joined the company in 2015 as chief business officer. Kulkarni became CEO in 2017. 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. 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 Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy predominantly affecting boys. The onset of muscle weakness typically begins around age four, with rapid progression. Initially, muscle loss occurs in the thighs and pe ...

and type 1 diabetes. In 2016, the company signed an agreement with

Bayer AG Bayer AG (English: , commonly pronounced ; ) is a German multinational pharmaceutical and biotechnology company and is one of the largest pharmaceutical companies and biomedical companies in the world. Headquartered in Leverkusen, Bayer's ...

to operate Casebia Therapeutics as a joint venture with Bayer under the management of CRISPR Therapeutics. The company went public on the NASDAQ exchange in October 2016. CRISPR Therapeutics has established partnerships with additional companies such as Viacyte (part of Vertex), Nkarta, Capsida, Curevac, and others.

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 Beta-thalassemia (β-thalassemia) is an genetic disorder, inherited hemoglobinopathy, blood disorder, a form of thalassemia resulting in variable outcomes ranging from clinically asymptomatic to severe anemia individuals. It is caused by reduce ...

and

sickle cell disease Sickle cell disease (SCD), also simply called sickle cell, is a group of inherited Hemoglobinopathy, haemoglobin-related blood disorders. The most common type is known as sickle cell anemia. Sickle cell anemia results in an abnormality in the ...

developed jointly with

Vertex Pharmaceuticals Vertex Pharmaceuticals Incorporated is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maint ...

, that received FDA approval for clinical use in December 2023. In May 2020, exa-cel had received

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

designation from the US Food and Drug Administration (FDA) for transfusion-dependent beta thalassemia and from the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

for sickle cell disease and transfusion-dependent beta thalassemia. Phase 3 clinical trial results supported the safety and efficacy of this treatment. The rolling

Biologics License Application A biologics license application (BLA) is defined by the U.S. Food and Drug Administration (FDA) as follows: The biologics license application is a request for permission to introduce, or deliver for introduction, a biologic product into inters ...

s (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 .

References

External links

* {{Official website, https://crisprtx.com/ Companies listed on the Nasdaq Swiss companies established in 2013 Biotechnology companies of Switzerland Companies based in Zug Genomics companies Biotechnology companies established in 2013 2013 establishments in Switzerland 2016 initial public offerings

History

Products

Exa-cel

See also

References

External links