CRISPR Therapeutics AG is turning a DNA modification tool into a drug development platform. This biotechnology company, based in Zug, Switzerland, and with a presence in the Boston area, focuses particularly on blood disorders and certain cancers. Its journey illustrates the transition of gene editing from the laboratory to clinical practice, with the industrial, medical and economic constraints involved in personalized cell therapy. Its corporate website is crisprtx.com.
A company born out of the CRISPR revolution
Founded in 2013, CRISPR Therapeutics counts Emmanuelle Charpentier, Rodger Novak and Shaun Foy among its co-founders. Charpentier played a central role in the work that made it possible to turn CRISPR-Cas9 into a programmable genome-editing tool, recognized with the 2020 Nobel Prize in Chemistry, shared with Jennifer Doudna. The principle involves guiding an enzyme to a targeted DNA sequence to make a modification there.
Listed on Nasdaq in 2016, the company has grown through a model combining in-house research, clinical trials and industry partnerships. Its collaboration with Vertex Pharmaceuticals marks a pivotal milestone: it enabled the development and subsequent commercialization of an initial therapeutic application of this technology.
From blood disorders to immunotherapy
This treatment, marketed under the name Casgevy, is based on exagamglogene autotemcel. Blood stem cells are collected from the patient, modified in the laboratory and then reinfused. The intervention aims to reactivate fetal hemoglobin production to offset the consequences of abnormalities affecting adult hemoglobin. It therefore does not directly correct the disease-causing mutation within the body.
Casgevy received its first authorization in the United Kingdom in November 2023. Authorizations followed in the United States and the European Union for certain people with severe sickle cell disease or transfusion-dependent beta-thalassemia, according to the indications and conditions set by each authority. Vertex leads commercialization under the partnership.
CRISPR Therapeutics also develops cellular immunotherapies, including allogeneic CAR-T cells designed to recognize disease-related targets. Unlike approaches manufactured from each patient’s own cells, these candidates use donor cells. The aim is to make treatments more readily available, particularly in oncology. These programs remain subject to demonstrating their safety and clinical efficacy.
What next?
The challenge now extends beyond scientific proof of concept. For therapies such as Casgevy, the treatment pathway involves cell collection, specialized manufacturing and conditioning chemotherapy before reinfusion. This process limits the number of centers able to treat patients and raises questions about access, funding and industrial capacity.
The trajectory of CRISPR Therapeutics will also depend on the long-term follow-up of treated patients and the success of its new programs. Expanding indications while simplifying administration is a major prospect, but each application will need to establish its own benefit-risk profile. The company is thus entering a phase in which clinical and industrial execution matters as much as mastery of gene editing.