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    GENE THERAPY FOR THE TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY (SCID) RELATED TO RAG1
    1.
    发明公开
    GENE THERAPY FOR THE TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY (SCID) RELATED TO RAG1 审中-公开

    公开(公告)号：US20240318154A1

    公开(公告)日：2024-09-26

    申请号：US18561938

    申请日：2022-05-20

    申请人： CELLECTIS ,  Albert-Ludwigs-Universitat Freiburg

    发明人： Toni CATHOMEN ,  Tatjana CORNU ,  Julia KLERMUND ,  Manuel RHIEL ,  Julia ROSITZKA ,  Philippe DUCHATEAU ,  Alexandre JUILLERAT

    IPC分类号： C12N9/22 ,  A61K35/28 ,  C07K14/47 ,  C12N5/0789 ,  C12N15/90

    CPC分类号： C12N9/22 ,  A61K35/28 ,  C07K14/4705 ,  C12N5/0647 ,  C12N15/907 ,  C12N2510/00

    摘要： The present invention generally relates to the field of genome engineering (gene editing), and more specifically to gene therapy for the treatment of Severe Combined Immunodeficiency (SCID) related to RAG1. Particularly, the present invention pertains to the treatment of RAG1 deficiency in long-term repopulating hematopoietic stem cells (HSCs). The present invention provides means and methods for genetically modifying HSCs involving gene editing reagents, such as TALE-nucleases, that specifically target a non-functional endogenous RAG1 gene, comprising at least one mutation causing Severe Combined Immunodeficiency (SCID), thereby allowing the restoration of the normal cellular phenotype. The present invention also provides engineered RAG1-edited HSCs comprising an exogenous sequence comprising a nucleic acid sequence encoding a functional RAG1 protein which is integrated in said HSCs' genome into a non-functional RAG1 endogenous locus, resulting in the expression of a functional RAG1 polypeptide. The present invention further provides populations of cells comprising said engineered HSCs, pharmaceutical compositions comprising said engineered HSCs or populations of cells, as well as their use in gene therapy for the treatment of Severe Combined Immunodeficiency (SCID) related to RAG1.

    CELL DEATH INDUCING CHIMERIC ANTIGEN RECEPTORS
    3.
    发明申请
    CELL DEATH INDUCING CHIMERIC ANTIGEN RECEPTORS 审中-公开

    公开(公告)号：US20190338015A1

    公开(公告)日：2019-11-07

    申请号：US16342139

    申请日：2017-10-19

    申请人： CELLECTIS

    发明人： Alexandre JUILLERAT ,  Philippe DUCHATEAU ,  Laurent POIROT ,  Murielle DERRIEN ,  Donna Marie STONE ,  Javier Fernando CHAPARRO RIGGERS

    IPC分类号： C07K14/705 ,  C12N5/0783 ,  A61K35/17 ,  C07K14/725 ,  C07K16/28

    摘要： The invention relates to cell death inducing chimeric antigen receptors (D-CAR). In particular, the present invention relates to cell death inducing chimeric antigen receptors which comprise at least one death domain in their endodomain, including cell death inducing chimeric antigen receptors comprising within their death domains modifications which attenuate the self-association and/or binding to pro-apoptotic or pro-necrotic adaptor proteins, such as FADD or TRADD. Moreover, the present invention relates to an engineered immune cell expressing at its surface a cell death inducing CAR of the present invention and, optionally, an activating chimeric antigen receptor, wherein the extracellular ligand-binding domains of the cell death inducing CAR and the activating CAR bind to different antigens. The engineered immune cell may furthermore comprise at least one edited (e.g., inactivated) gene selected from TCR genes, immune check point genes, genes involved in drug resistance, and combinations thereof.

    METHOD OF ENGINEERING MULTI-INPUT SIGNAL SENSITIVE T CELL FOR IMMUNOTHERAPY
    7.
    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

    申请人： CELLECTIS

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

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

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

    摘要： 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.

    摘要翻译： 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。