[2][3] The company has approximately 500 employees and has offices in Zug, Switzerland, Boston, Massachusetts, San Francisco, California and London, United Kingdom.

[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.

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.

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.

[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.