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Biotechs Partner on Gene-Edited Cancer Cell Therapies

Natural killer cell

Natural killer cell (NIAID, Flickr)

7 May 2021. Two biotechnology companies are collaborating on using the gene editing technique Crispr to create engineered immune-system cells to treat cancer. Nkarta Inc. in South San Francisco, California and Crispr Therapeutics in Zug, Switzerland aim to co-develop and commercialize gene-edited natural killer cells as cancer therapies.

Nkarta develops engineered natural killer cells from the immune system as off-the-shelf cancer therapies. Like their cousins B- and T-cells, natural killer cells are white blood cells, which act against cells infected by viruses and in tumors. They’re called “natural killers” for their innate ability to seek out and attack pathogens and tumors without priming or prior activation. Nkarta says it further engineers natural killer cells with receptors designed to identify and bind to specific molecular targets, the release cancer-killing cytokine enzymes. The company also adds interleukin-15, a cytokine that induces proliferation of natural killer cells.

To produce its cancer treatments, Nkarta takes natural killer cells from healthy donors, The donated natural killer cells are expanded and engineered to express chimeric antigen receptors or CARs and interleukin-15 on the cell membranes. The cells are then expanded further with interleukin-15 and frozen to preserve for future administration to patients. The company’s lead product, code-named NKX101, is in an early-stage clinical trial with acute myeloid leukemia and myelodysplastic syndrome patients, two forms of blood-related cancer. Another product code-named NKX019 is completing preclinical development as a treatment for B-cell malignancies.

Gene-edited natural killer cells

Crispr — short for clustered, regularly interspaced short palindromic repeats — makes it possible to edit genomes of organisms harnessing bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. At Crispr Therapeutics and in most other cases, the actual editing is done by Crispr-associated protein 9, or Cas9, enzyme that programs RNA to cut DNA at precise points in genomes, making it possible to delete, insert, or correct defects in human genomes.

For cell therapies, Crispr Therapeutics edits genes that improve the safety or efficacy of the treatments, either as immune system cells outside the body then re-infused to the patient, or packaged in nanoscale lipid particles and delivered systemically or into target organs. As reported in Science & Enterprise, Crispr Therapeutics and Vertex Pharmaceuticals are collaborating on a gene-edited treatment for the blood disorders sickle cell disease and beta thalassemia, now in mid-stage clinical trials.

In their project, Nkarta and Crispr Therapeutics will jointly develop two cancer treatments with natural killer cells engineered with CARs. One of those programs targets the CD70 tumor antigen, part of a pathway for regulating T-cells in the immune system and implicated in non-small cell lung cancer. The second program, for an undisclosed target, engineers both natural killer and T-cells to harness both innate and adaptive immunity. In the collaboration, Nkarta and Crispr Therapeutics are equally sharing all research and development costs, and any subsequent profits.

Nkarta is also licensing Crispr Therapeutics’ technology to edit five gene targets, applicable to any number of Nkarta’s natural killer cell products. Nkarta retains all worldwide rights for these targets, with Crispr Therapeutics eligible for undisclosed milestone payments and royalties on sales. The agreement has a three-year exclusivity period between the two companies for R&D and commercialization of off-the-shelf, gene-edited, donor-derived natural killer and combination natural killer/T-cells. Dollar amounts in the agreement were not disclosed.

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