Genome editing systems, such as CRISPR/Cas9, can be delivered inside the body – by injection – to treat disease. This is called in vivo therapy…
or, the system can be used to correct a sample of a patient’s (or a donor’s) cells, which are then re-infused. This is an example of an ex vivo application called “engineered cell therapy.”
Our vision is to leverage CRISPR/Cas9 genome editing to create engineered cells that have profound therapeutic utility in immuno-oncology and autoimmune diseases. Engineered cells promise to transform clinical medicine, and we are focused on diseases with high unmet medical need where genome edited cell therapy offers a powerful and differentiated modality that can either disrupt a disease-causing genetic mutation, such as in cancer, or repair a faulty gene.
Modulating the immune system to eliminate cancerous tumors or to arrest autoimmune diseases
In oncology, we aim to discover and unlock the capabilities of the body’s immune system – selecting, preparing and directing these cells to destroy cancerous tumors. In autoimmunity, we strive to achieve the opposite – modulating the patient’s immune cells to prevent them from destroying self-tissues.
Engineered cells promise to transform clinical medicine.
Our wholly owned engineered cell therapy programs in immuno-oncology and autoimmune diseases will help broaden the application of CRISPR/Cas9.
Improving the way engineered cell therapies are administered
We also are working to improve and simplify the way engineered cell therapies are administered.
Today, cell therapies have shown great promise, but are restricted to very limited indications, such as certain leukemias and lymphomas, and the process for providing the therapy to each patient is very cumbersome and expensive. At the first visit to the clinic, blood is withdrawn from the patient and separated. The relevant immune cells are isolated and genetically engineered to target the cancer. These engineered cells are allowed to divide and grow to increase their number before patients return to the clinic to have their own modified cells reinfused. In addition, unfortunately, this process cannot be performed for all patients because some patients don’t have a healthy enough immune system to begin with.
Intellia envisions a better way. We believe that genome editing may be the key to providing solutions to these important limitations.
In the short term, we intend to use CRISPR/Cas9 to selectively and carefully edit the genes of selected immune cells, with the goal of improving their performance. These improvements are aimed at yielding immune cells that will more effectively identify and attack cancerous cells upon re-infusion.
Additionally, in our vision for the future, patients would only need a single visit to the clinic for administration of an off-the-shelf (i.e., allogeneic) cellular medicine derived from either a donor or cell bank. Applying CRISPR/Cas9 to human cells, we intend to pre-manufacture engineered cellular therapies that can be administered to patients based on their specific cancer mutations. These therapies would address a broader range of both hematological and solid cancers.