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Gene-Editing Therapies for HIV

Description

Human Immunodeficiency Virus uses surface proteins such as CCR5 to infect immune cells. People with a rare natural variant of the CCR5 gene exhibit resistance to HIV infection.

Our scientists developed a method for treating HIV using mRNA encoding gene-editing proteins to inactivate CCR5 and/or CXCR4 in hematopoietic cells.

Gene-Editing Therapies for HIV is protected by three U.S. patents (with additional patents pending in the U.S. and in other countries). Of note, certain granted patents include claims that are not limited by specific target sequence, mRNA sequence or chemistry, or type of gene-editing protein.


Example Applications

  • Replicate natural HIV resistance using site-specific genome engineering of a patient’s cells
  • Achieve high-efficiency delivery of mRNA encoding gene-editing proteins to hematopoietic cells ex vivo using electroporation
  • Target T cells and/or HSCs for persistent HIV immunity
  • Gene edit somatic cells to avoid the risk of germline transmission
  • Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity ex vivo gene editing

Representative Claim

U.S. Pat. No. 9,605,278
A therapeutic composition, comprising a gene-edited hematopoietic cell, wherein:

the gene-edited hematopoietic cell comprises:

an in vitro transcribed synthetic RNA molecule encoding a gene-editing protein, the gene-editing protein comprising a DNA-binding domain and a nuclease catalytic domain that causes a double-strand break in the DNA of the hematopoietic cell; and

a double-strand break in its DNA, the double-strand break being caused by the gene-editing protein and reducing the function of one or more of CCR5 and CXCR4.