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    DESIGN OF RARE-CUTTING ENDONUCLEASES FOR EFFICIENT AND SPECIFIC TARGETING DNA SEQUENCES COMPRISING HIGHLY REPETITIVE MOTIVES
    11.
    发明申请
    DESIGN OF RARE-CUTTING ENDONUCLEASES FOR EFFICIENT AND SPECIFIC TARGETING DNA SEQUENCES COMPRISING HIGHLY REPETITIVE MOTIVES 审中-公开
    用于有效和特异性靶向包含高度重复活性的DNA序列的切割内切酶的设计

    公开(公告)号：US20160273002A1

    公开(公告)日：2016-09-22

    申请号：US15031996

    申请日：2014-10-24

    Applicant: CELLECTIS

    Inventor： Philippe DUCHATEAU ,  Alexandre JUILLERAT

    IPC: C12N15/90 ,  C07K14/195 ,  C12N9/22

    CPC classification number: C12N15/907 ,  A61K38/00 ,  A61K48/00 ,  A61K48/005 ,  C07K14/195 ,  C07K2319/80 ,  C12N9/22 ,  C12Y301/00 ,  C12Y301/21004

    Abstract: The present invention is in the field of genetic editing tools and methods of genetic engineering. It relates to the engineering of rare-cutting endonucleases designed to contract highly repetitive motives in chromosomes, which are at the origin of certain genetic diseases, in particular the so-called “triplet repeat diseases”, such as the Huntington disease. The invention encompasses the method for contracting the repetitive motives, the rare-cutting endonucleases for use to contract repetitive motives in a gene subjected to repeat disorder, the polynucleotides and vectors encoding thereof as well as the resulting pharmaceutical compositions.

    Abstract translation: 本发明在遗传编辑工具和基因工程方法领域。 它涉及设计用于在染色体中收缩高度重复动机的稀有内切核酸酶的工程，这些动机是某些遗传疾病的起源，特别是所谓的“三重重复疾病”，如亨廷顿疾病。 本发明包括用于收缩重复动机的方法，用于在重复病症的基因中重复动作的稀有内切核酸酶，编码它们的多核苷酸和载体以及所得药物组合物。

    METHOD OF ENGINEERING MULTI-INPUT SIGNAL SENSITIVE T CELL FOR IMMUNOTHERAPY
    15.
    发明申请
    METHOD OF ENGINEERING MULTI-INPUT SIGNAL SENSITIVE T CELL FOR IMMUNOTHERAPY 审中-公开
    工程多用途信号敏感细胞免疫学方法

    公开(公告)号：US20170073423A1

    公开(公告)日：2017-03-16

    申请号：US15106783

    申请日：2014-12-19

    Applicant: CELLECTIS

    Inventor： Alexandre JUILLERAT ,  Claudia BERTONATI ,  Julien VALTON ,  Philippe DUCHATEAU ,  Laurent POIROT

    IPC: C07K16/30 ,  C07K16/28 ,  C07K14/47

    CPC classification number: C07K16/30 ,  C07K14/47 ,  C07K16/2863 ,  C07K16/468 ,  C07K2317/622 ,  C07K2319/03 ,  C07K2319/30 ,  C12N2510/02

    Abstract: The present invention relates to a method to engineer immune cell for immunotherapy. In particular said immune cells are engineered with chimeric antigen receptors, which be activated by the combination of hypoxia and ligand extracellular binding as input signals. The invention also relates to new designed chimeric antigen receptors which are able to redirect immune cell specificity and reactivity toward a selected target exploiting the ligand-binding domain properties and the hypoxia condition. The present invention also relates to cells obtained by the present method, in particular T-cells, comprising said chimeric antigen receptors for use in cancer treatments.

    Abstract translation: 本发明涉及免疫治疗免疫细胞的设计方法。 特别地，所述免疫细胞用嵌合抗原受体工程化，其通过缺氧和配体细胞外结合的组合被激活作为输入信号。 本发明还涉及新设计的嵌合抗原受体，其能够将免疫细胞特异性和反应性转向利用配体结合结构域性质和缺氧条件的选定靶标。 本发明还涉及通过本方法获得的细胞，特别是包含用于癌症治疗的所述嵌合抗原受体的T细胞。

    TAL- effector nuclease (TALEN) -MODIFIED ALLOGENIC CELLS SUITABLE FOR THERAPY
    17.
    发明申请
    TAL- effector nuclease (TALEN) -MODIFIED ALLOGENIC CELLS SUITABLE FOR THERAPY 有权

    公开(公告)号：US20230138915A1

    公开(公告)日：2023-05-04

    申请号：US16340412

    申请日：2017-10-19

    Applicant: CELLECTIS

    Inventor： Philippe DUCHATEAU ,  Brian BUSSER ,  Alexandre JUILLERAT ,  Anne-Sophie GAUTRON ,  Laurent POIROT

    IPC: C12N15/90 ,  C12N15/11 ,  C12N9/22 ,  C12N5/0783 ,  C07K14/725

    Abstract: The invention relates to the fields of immunotherapy, molecular biology and recombinant nucleic acid technology. In particular, the invention relates to a TALEN-modified human primary cell comprising in its genome, a modified human T cell receptor alpha gene with an insertion comprising at least, from 5′ to 3′, a polynucleotide encoding a self-cleaving peptide, a chimeric antigen receptor, wherein the cell has undetectable cell-surface expression of the endogenous alpha beta T cell receptor as compared to a TCR positive control cell and expresses a receptor to target a pathological cell, use of said cell for treating a disease, including cancer. The invention further relates to methods for producing such a TALEN-modified cell, and to means for detecting such an engineered human primary cell or other genetically modified human primary cell obtained using alternative and/or additional rare cutting endonucleases.

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