How Can CRISPR Be Used to Treat Cancer

CRISPR Beyond Genetic Diseases

When CRISPR-Cas9 was first found and subsequently earned its discoverers a Nobel Price in 2020, the obvious first application of this technology was gene therapy. This is because CRISPR allows for very precise and targeted gene editing, making possible the idea of correcting abnormal gene sequences in patients.

But another growing potential of CRISPR is in oncology (cancer treatment), an application with some of the largest number of ongoing gene editing clinical trials.

Source: ARK Invest

One of the first applications of CRISPR to cancer research is replacing previous genetic modification tools. CRISPR being cheaper, quicker, and easier opens the door to more researchers working with genetically modified animal models, like mice.

But CRISPR as a tool for researchers is only the beginning, and it could be fashioned into a weapon against cancers.

Curing Cancer With CRISPR?

The main obstacle on the way to cure cancer is that this is not just one disease. Each cancer is made of abnormal cells in its own unique way. This means that no chemical drug is likely to work on every cancer, as each will have a unique pattern of resistance and weaknesses.

One promising strategy is to take immune cells from the patient and modify them so they focus on killing cancerous cells. The cells are then multiplied in a lab and re-injected in the patient.

Source: Cancer.gov

These are the basics behind the promising CAR-T cancer therapies (Chimeric Antigen Receptor).

Other similar methods use another immune cell type than lymphocyte T, like lymphocyte NK (Natural Killer). In any case, CRISPR is also used to improve the cells so they are not rejected by the patient body, and with an increased efficiency at finding and destroying cancer.

The most advanced therapies look to engineer CAR-T or CAR-NK cells that can be produced in large batches and still accepted by the body of multiple patients.

CRISPR Companies Leading In Cancer Therapies

1. CRISPR Therapeutics

One of the leading companies in this sector is CRISPR Therapeutics, founded by one of the discoverers of CRISPR-CAs9.

Its CAR-T cells are modified not only to target cancer cells but also to reduce the risk of unwanted side effects (graft versus host disease) and to increase the survival time of the modified immune cell in the patient's body, giving it more time to attack cancer cells.

Source: CRISPR Therapeutics

The company currently has 7 candidates in the pipeline, of which 4 already in clinical trials.

CRISPR is also investing in a CAR-NK therapy, in partnership with Nkarta Therapeutics (NKTX), in the pre-clinical stage. CAR-NK therapies have the potential to be even less likely to trigger side effects and to be more effective against solid tumors (90% of cancer in adults).

2. Ginkgo Bioworks Holdings, Inc.

Synthetic biology company Gingko Bioworks announced in April 2023 a partnership with the Wisconsin Alumni Research Foundation (WARF) to jointly develop the new generation of CAR-T treatment. Researchers at the WARF were behind the first FDA-approved CAR-T therapy. Today, 6 CAR-T therapies have been approved, and intense research efforts are being made to adapt CAR-T therapies to solid tumors.

A key technology behind Bioworks’ ambitions in CAR-T therapies is its CAR-T screening platform. It allows it to “scan” hundreds of thousands of CAR-T designs, helping find quicker and cheaper what might work for solid tumors.

2. Editas Medicine, Inc.

Editas is another gene-editing biotech company with programs targeting cancer. It currently has 2 different strategies in oncology, both relying on genetically modifying immune T-cells.

Source: Editas

Its autologous CAR-T cell therapies are developed in partnership with Bristol Myers Squibb (BMY).

It also has a program running with Immatix (IMTX) on a proprietary Activated allogeneic cell therapy (ACT), a therapy modifying the tumor micro-chemical environment in order to reduce its resistance or growth.

Editas is focused on using a variant of CRISPR called AsCas12a. This is expected to create more precise and efficient gene editing.

(We explained in more detail the potential of Cas12a in a dedicated article, “What Is CRISPR-Cas12a2? & Why Does It Matter?”)

4. Caribou Biosciences

Caribou Biosciences is working on CAR-T therapies for blood cancers, with 2 out of 3 candidates in phase I of clinical trials.  It has a CAR-NK therapy platform in the pre-clinical stage to target solid tumors.

It also has a partnership with AbbVie for cancer cell therapies (CAR-T) at an undisclosed stage due to confidentiality agreements.

Source: Caribou

Caribou is also pushing the frontier of CRISPR technology with the invention of a DNA/RNA hybrid CRISPR system, ChRDNA.

This system should be able to achieve a strong reduction of off-target gene editing, a constant concern for all gene editing and CRISPR therapies. This, in turn, should reduce side effects potential dangers for the patients and, increase efficiency, and maybe allow for smaller doses.

5. Intellia Therapeutics, Inc.

Intellia is the first company to achieve successful systemic CRISPR gene editing in humans. This is especially useful for genetic diseases where ex-vivo, like CAR-T therapies, might not be an option. The company still favors an ex-vivo approach for its cancer therapies.

Source: Intellia

Intellia’s main focus is rare diseases, with a total of 8 candidates, of which 2 are in early clinical stage. In the cancer segment, one is at the pre-clinical stage, and another one is at the IND-enabling stage (Investigational New Drug).

While the Itellia cancer pipeline is interesting, investors will want to pay attention to the rare disease portfolio, likely the focus of markets for the foreseeable future.