Gene-Editing Checkpoint Molecule Genes for the Treatment of Cancer

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Checkpoint molecule genes act to prevent the immune system from attacking normal cells. In many cancers, the expression of checkpoint molecule genes by cancer cells prevents their destruction by the immune system (a “cold” tumor).

Our scientists developed a method for treating cancer by inactivating checkpoint molecule genes in cancer cells using mRNA encoding gene-editing proteins to unmask the cancer cells to the immune system, and thus turn a “cold” tumor “hot”.

Gene-Editing Checkpoint Molecule Genes for the Treatment of Cancer is protected by seven U.S. patents, as well as patents in China and Australia (with additional patents pending in the U.S. and in other countries). Of note, certain granted patents include claims that are not limited by type of cancer, specific checkpoint molecule gene, mRNA sequence or chemistry, or type of gene-editing protein.

Example Applications

  • Inactivate checkpoint molecule genes in tumor cells to unmask them to the immune system
  • Treat metastatic disease by training the immune system on unmasked tumor cells
  • Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity inactivation of target checkpoint molecule genes
  • Combine with Factor’s ToRNAdo™ Nucleic-Acid Delivery System for high-efficiency delivery to tumor cells in vivo
  • Inactivate multiple checkpoint molecule genes to unmask multi-pathway-resistant tumor cells

Representative Data

Figure 1. Gene editing the TRAC gene and the checkpoint molecule gene PD1 in human iPS cells.

Representative Claim

U.S. Pat. No. 10,363,321

An in vivo method for treating cancer in a subject comprising:

administering to the subject by intratumoral injection a non-viral, cell-free composition comprising a synthetic messenger RNA (mRNA) encoding a gene-editing protein capable of creating a single-strand or double-strand break in an immune checkpoint molecule gene, and

causing a single-strand or double-strand break in the DNA of a tumor cell, the single-strand or double-strand break being localized to an immune checkpoint molecule gene in the tumor cell in the subject, resulting in the stimulation or enhancement of an immune response in the subject;

thereby treating cancer in the subject.