CRISPR Therapeutics
A Swiss-American biotechnology company developing medicines based on CRISPR/Cas9 gene editing.
Last updated August 21, 2026
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Overview
CRISPR Therapeutics AG is a Swiss-American biotechnology company focused on discovering, developing, and commercializing medicines based on CRISPR/Cas9 gene editing. The company was founded in 2013 by Emmanuelle Charpentier, Rodger Novak, and Shaun Foy, during the early commercial development of CRISPR as a programmable method for modifying DNA. Charpentier later shared the 2020 Nobel Prize in Chemistry with Jennifer Doudna for foundational work on the development of CRISPR-Cas9 genome editing. CRISPR Therapeutics was established to translate that scientific platform into treatments for serious diseases with genetic or cellular causes. The company is headquartered in Zug, Switzerland, and operates through a Swiss corporate structure and the United States-based CRISPR Therapeutics, Inc. It has offices and operations in Zug, Boston, San Francisco, and London. Its activities span research, clinical development, regulatory affairs, manufacturing, and commercial preparation. The company has worked on programs involving blood disorders, cancer, diabetes, and other severe diseases, using both ex vivo and in vivo approaches to gene editing and cell therapy. Its most advanced program is exagamglogene autotemcel, commonly called exa-cel and formerly known as CTX001. The therapy is an ex vivo gene-edited cell treatment developed with Vertex Pharmaceuticals for sickle cell disease and transfusion-dependent beta thalassemia. Patient stem cells are collected, edited outside the body, and returned after conditioning treatment. The program received regulatory approval from the United States Food and Drug Administration in December 2023 and has also been subject to regulatory reviews and approvals in other jurisdictions. The product is marketed as Casgevy. CRISPR Therapeutics has pursued its platform through internal research and collaborations. Its 2015 strategic collaboration with Vertex became the foundation for the exa-cel program and was later expanded to additional disease areas, including type 1 diabetes and Duchenne muscular dystrophy research. In 2016, CRISPR Therapeutics entered into an arrangement with Bayer to operate Casebia Therapeutics, a joint venture intended to develop CRISPR-based treatments. The company has also disclosed relationships with organizations including Viacyte, Nkarta, Capsida, and CureVac. CRISPR Therapeutics became a publicly traded company on the Nasdaq exchange in October 2016 under the symbol CRSP. Samarth Kulkarni joined in 2015 as chief business officer and became chief executive officer in 2017. The company's manufacturing facility in Framingham, Massachusetts, received a Facilities of the Year Award in 2022. With the approval of Casgevy, CRISPR Therapeutics moved from a development-stage gene-editing specialist toward a commercial biotechnology company, while continuing to invest in a broader pipeline of gene-edited cell therapies and other genetic medicines.
History
CRISPR Therapeutics emerged during the rapid transition of CRISPR/Cas9 from an academic gene-editing method into a potential therapeutic platform. The company was founded in 2013 by Emmanuelle Charpentier, Rodger Novak, and Shaun Foy. Charpentier's scientific work on CRISPR-Cas9 helped establish the basis for targeted modification of DNA, while the company was organized to pursue commercial medicines built around the technology. Charpentier subsequently shared the 2020 Nobel Prize in Chemistry with Jennifer Doudna for foundational contributions to CRISPR genome editing. From its beginning, CRISPR Therapeutics concentrated on diseases in which changing the activity of a gene or engineering a patient's cells could address an underlying biological cause. Its areas of interest have included sickle cell disease, beta thalassemia, oncology, type 1 diabetes, cardiovascular disease, and other serious conditions. The company has used an ex vivo model for some of its most advanced programs: cells are taken from a patient, edited in a controlled manufacturing process, and administered back to that patient after preparative treatment. A major early development was the 2015 strategic research collaboration with Vertex Pharmaceuticals. The collaboration focused on applying CRISPR/Cas9 to discover and develop treatments for diseases with genetic causes. Its leading result was CTX001, later called exagamglogene autotemcel or exa-cel, an edited-cell therapy for sickle cell disease and transfusion-dependent beta thalassemia. The companies later expanded their work to additional areas, including type 1 diabetes and Duchenne muscular dystrophy. In 2016, CRISPR Therapeutics entered a joint-venture arrangement with Bayer to operate Casebia Therapeutics. The venture was intended to develop CRISPR-based treatments and broaden the company's industrial and research base. That same year, CRISPR Therapeutics completed its public-market listing on Nasdaq, giving it access to public biotechnology capital and establishing the CRSP ticker. Samarth Kulkarni joined the company in 2015 as chief business officer and became chief executive officer in 2017. Under his leadership, the company continued advancing its clinical pipeline and building the capabilities required for gene-edited cell therapy development and manufacturing. The company maintains operations in Zug, Boston, San Francisco, and London, and its Framingham manufacturing facility was recognized with a Facilities of the Year Award in 2022. The exa-cel program reached a decisive regulatory milestone in December 2023, when the U.S. Food and Drug Administration approved it for sickle cell disease. The program had previously received orphan-drug designations and had undergone regulatory submissions in the United States, the European Union, and the United Kingdom. Its approval transformed CRISPR Therapeutics from a company principally associated with experimental gene editing into a biotechnology company with a commercial product, while its broader pipeline remained focused on translating gene editing and cell engineering into treatments for serious diseases.
- 2023
U.S. approval of Casgevy for sickle cell disease
[1]The FDA approved exagamglogene autotemcel, marketed as Casgevy, for sickle cell disease.
- 2022
Framingham facility receives industry award
[1]The company's Framingham, Massachusetts manufacturing facility received a Facilities of the Year Award.
- 2020
Co-founder receives Nobel recognition
[1]Emmanuelle Charpentier shared the Nobel Prize in Chemistry for foundational work on CRISPR-Cas9 genome editing.
- 2017
Kulkarni becomes chief executive officer
[1]Samarth Kulkarni became chief executive officer.
- 2016
Casebia joint venture established
[1]CRISPR Therapeutics entered an agreement with Bayer to operate Casebia Therapeutics as a CRISPR-focused joint venture.
- 2016
Nasdaq listing
[1]The company became publicly traded on Nasdaq under the ticker CRSP.
- 2015
Vertex research collaboration begins
[1]CRISPR Therapeutics and Vertex Pharmaceuticals established a strategic collaboration to discover and develop CRISPR-based treatments for genetic diseases.
- 2015
Samarth Kulkarni joins the company
[1]Samarth Kulkarni joined CRISPR Therapeutics as chief business officer.
- 2013
Company founded
[1]Emmanuelle Charpentier, Rodger Novak, and Shaun Foy founded CRISPR Therapeutics to develop medicines using CRISPR/Cas9 gene editing.
Products and positioning
A pioneer in translating CRISPR/Cas9 genome editing into potentially transformative medicines, with particular emphasis on severe genetic diseases and engineered cell therapies.
Gene-edited cell therapy · 2023
Casgevy
Casgevy is the commercial name for exagamglogene autotemcel, also known as exa-cel and formerly CTX001. Developed with Vertex Pharmaceuticals, it is an ex vivo CRISPR-edited autologous hematopoietic stem-cell therapy. A patient's cells are collected and edited before being returned following conditioning treatment. The program targets severe inherited blood disorders, including sickle cell disease and transfusion-dependent beta thalassemia, and received U.S. FDA approval in December 2023.
Investigational and subsequently approved gene-edited cell therapy
CTX001 / exa-cel
CTX001 was the development name for the program that became exagamglogene autotemcel, or exa-cel, and was commercialized as Casgevy. It originated from the CRISPR Therapeutics-Vertex collaboration and was designed to modify patient-derived blood stem cells to address the biology underlying sickle cell disease and transfusion-dependent beta thalassemia.
Drug-discovery and gene-editing platform · 2013
CRISPR/Cas9 therapeutic platform
CRISPR Therapeutics' core platform applies programmable CRISPR/Cas9 genome editing to the discovery and development of medicines. The company has used the platform across blood disorders, cancer, diabetes, cardiovascular disease, and other severe conditions, with programs spanning edited cell therapies and additional therapeutic approaches.
Joint-venture research programs · 2016
Casebia Therapeutics programs
Casebia Therapeutics was established through an arrangement between CRISPR Therapeutics and Bayer to pursue CRISPR-based treatments. It represented an early industrial collaboration intended to extend gene-editing research into therapeutic programs under CRISPR Therapeutics' management.
Brand decisions
2023
Regulatory approval of CasgevyProduct launchBackground
The exa-cel program had advanced through clinical development and regulatory submissions for serious inherited blood disorders.
Action taken
The U.S. FDA approved exagamglogene autotemcel, marketed as Casgevy, for sickle cell disease.
Aftermath
CRISPR Therapeutics entered the commercial medicine market and became one of the first companies to secure approval for a CRISPR-based therapy.
2016
Casebia Therapeutics joint venture with BayerM&ABackground
The company pursued a corporate structure that could support broader CRISPR drug discovery and development.
Action taken
CRISPR Therapeutics entered an agreement with Bayer to operate Casebia Therapeutics as a joint venture.
Aftermath
Casebia became part of the company's early collaboration and platform-development history.
2016
Public-market listingOtherBackground
As a development-stage biotechnology company, CRISPR Therapeutics required access to public capital to fund research and clinical development.
Action taken
The company listed its shares on Nasdaq under the symbol CRSP.
Aftermath
The listing established CRISPR Therapeutics as a publicly traded gene-editing biotechnology company.
2015
Strategic collaboration with Vertex PharmaceuticalsStrategyBackground
CRISPR Therapeutics sought a development and commercialization partner with capabilities in clinical development and rare-disease medicines.
Action taken
The companies formed a research collaboration focused on CRISPR/Cas9 treatments for genetic diseases, later producing the CTX001/exa-cel program.
Aftermath
The collaboration became the foundation for Casgevy, the company's first approved medicine, and was expanded to additional disease areas.
Recent events
- 2023
CRISPR Therapeutics and Vertex receive U.S. approval for Casgevy[1]
Product launch · Regulation
The FDA approved exagamglogene autotemcel, marketed as Casgevy, for an indication involving sickle cell disease. The approval made it the company's first approved medicine in the United States.
- 2022
CRISPR Therapeutics manufacturing facility receives Facilities of the Year Award[1]
Other
The company's Framingham, Massachusetts manufacturing facility received a Facilities of the Year Award.
- 2016
CRISPR Therapeutics lists on Nasdaq[1]
Other
CRISPR Therapeutics became a publicly traded company on the Nasdaq exchange under the ticker CRSP.
Sources3 sources
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