CRISPR Therapeutics

CRISPR Therapeutics specializes in developing gene editing-based novel therapeutics for a variety of areas including immuno-oncology and hemoglobinopathies. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes, and focuses predominantly on treating somatic cells that do not transfer DNA to the next generations, preventing future generations from contracting a genetic disease. 

The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, the company had 18 drug candidates in its pipeline.

In a milestone event in November 2023, the company’s gene-editing therapy exagamglogene autotemcel, CASGEVY (exa-cel) , developed with Vertex Pharmaceuticals to treat sickle-cell disease and beta-thalassemia, received the world’s first authorization from the UK's Medicines and Healthcare products, Regulatory Agency (MHRA). In December 2023, it received FDA approval for patients aged 12 and older. The drug for beta-thalassemia patients is also nearing its market entry in the US, with the FDA’s decision expected on March 30, 2024. 

Key customers and partnerships

The company has secured strategic collaborations with several companies to co-develop and co-commercialize gene editing medicines that include Vertex Pharmaceuticals (2015) to develop a therapy (CTX001) for various indications including beta-thalassemia, sickle cell disease, and Duchenne muscular dystrophy; and ViaCyte (September 2018) to develop VCTX210, a regenerative medicine for diabetes.

Big pharma Bayer also signed an option agreement in 2019 with the company to co-develop and co-commercialize two candidates, after entirely handing over the joint venture Casebia Therapeutics to CRISPR.  

Funding and financials

Founded in 2013, CRISPR has been listed on the Nasdaq since 2016. For 2021, the company reported a revenue of USD 913.1 million earned via milestone payments, collaborations, and licensing.