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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

    Applicant: CELLECTIS ,  Albert-Ludwigs-Universitat Freiburg

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

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

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

    Abstract: 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

    Applicant: CELLECTIS

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

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

    Abstract: 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.

    CAS9 NUCLEASE PLATFORM FOR MICROALGAE GENOME ENGINEERING
    8.
    发明申请
    CAS9 NUCLEASE PLATFORM FOR MICROALGAE GENOME ENGINEERING 审中-公开
    用于微藻基因工程的CAS9核平台

    公开(公告)号：US20160304893A1

    公开(公告)日：2016-10-20

    申请号：US15103773

    申请日：2014-12-12

    Applicant: CELLECTIS

    Inventor： Fayza DABOUSSI ,  Marine BEURDELEY ,  Alexandre JUILLERAT

    IPC: C12N15/82 ,  C12N9/22

    CPC classification number: C12N15/8213 ,  C12N9/22

    Abstract: The present invention relates to a method of genome engineering in microalgae using the Cas9/CRISPR system. In particular, the present invention relates to methods of delivering RNA guides via cell penetrating peptides in microalgae, preferably in stable integrated Cas9 microalgae. The present invention also relates to kits and isolated cells comprising Cas9, split Cas9 or guide RNA and Cas9-fused cell-penetrating peptides. The present invention also relates to isolated cells obtained by the methods of the invention.

    Abstract translation: 本发明涉及使用Cas9 / CRISPR系统的微藻中的基因组工程方法。 特别地，本发明涉及通过微藻中的细胞穿透肽递送RNA引导物的方法，优选在稳定的综合Cas9微藻中。 本发明还涉及包含Cas9，裂解Cas9或引导RNA和Cas9融合的细胞穿透肽的试剂盒和分离的细胞。 本发明还涉及通过本发明的方法获得的分离的细胞。

    DESIGN OF RARE-CUTTING ENDONUCLEASES FOR EFFICIENT AND SPECIFIC TARGETING DNA SEQUENCES COMPRISING HIGHLY REPETITIVE MOTIVES
    9.
    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。

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