Cellectis has been granted a patent for a method of producing HIV-resistant primary hematopoietic cells. The method involves introducing a specific reagent, known as a TALE-nuclease, into primary hematopoietic cells to reduce the expression of CCR5, a protein involved in HIV infection. This technology could potentially lead to the production of safer cells for infusion into HIV patients. GlobalData’s report on Cellectis gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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According to GlobalData’s company profile on Cellectis, CAR-T cell based therapies was a key innovation area identified from patents. Cellectis's grant share as of September 2023 was 36%. Grant share is based on the ratio of number of grants to total number of patents.
Patent granted for method to produce hiv resistant cells
A recently granted patent (Publication Number: US11767512B2) describes a method for producing HIV resistant primary hematopoietic cells. The method involves providing a population of primary hematopoietic cells and introducing a nucleic acid encoding a sequence-specific reagent that targets a genomic sequence encoding the N-terminal hydrophilic external region of the CCR5 protein. This specific reagent is a TALE-nuclease that binds to a target sequence. The expression of the sequence-specific reagent reduces the expression of functional CCR5 by more than 50% in the resulting population of primary cells.
The patent claims specify different levels of reduction in the expression of functional CCR5, ranging from more than 50% to more than 70%. Importantly, this reduced expression of functional CCR5 is achieved without cleaving the CCR2 gene. The population of primary cells used in the method can include at least 50% CD34+ cells or at least 50% T-cells. Additionally, the method can involve targeted gene insertion into the genomic sequence encoding the N-terminal hydrophilic external region of the CCR5 protein. This targeted gene insertion can include an exogenous sequence encoding a drug-resistant protein, a protein that inhibits the CXCR4 pathway for broader HIV resistance, or a chimeric antigen receptor (CAR).
The patent also describes the use of TALE-nuclease monomers with specific polypeptide sequences and RVD sequences. These TALE-nuclease monomers can be expressed from mRNA transfected by electroporation. The population of primary hematopoietic cells used in the method can be derived from iPS cells or further dedifferentiated into iPS cells. The patent also claims a population of hematopoietic primary cells obtained using the described method.
Furthermore, the patent claims methods for treating HIV-positive patients or cancer patients by administering the population of primary cells obtained through the described method. The patent also includes claims for TALE-nuclease monomers designed to cleave the genomic sequence encoding the N-terminal hydrophilic external region of the CCR5 protein, as well as pairs of these TALE-nuclease monomers that bind to specific polynucleotide target sequences.
In summary, this granted patent describes a method for producing HIV resistant primary hematopoietic cells using a specific reagent and TALE-nuclease technology. The method allows for the reduction of functional CCR5 expression without cleaving the CCR2 gene and offers potential applications in HIV treatment and cancer treatment.
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GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies
