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公开(公告)号：US20190010514A1
公开(公告)日：2019-01-10
申请号：US16138908
申请日：2018-09-21
申请人： CELLECTIS
IPC分类号： C12N15/85 , C12N5/0783 , C07K14/74 , C12N15/90 , C07K14/705 , C12N15/113
摘要： The present invention pertains to engineered T-cells, method for their preparation and their use as medicament, particularly for immunotherapy. The engineered T-cells of the invention are characterized in that the expression of beta 2-microglobulin (B2M) and/or class II major histocompatibility complex transactivator (CIITA) is inhibited, e.g., by using rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding H2M and/or CIITA or by using nucleic acid molecules which inhibit the expression of B2M and/or CIITA. In order to further render the T-cell non-alloreactive, at least one gene encoding a component of the T-cell receptor is inactivated, e.g., by using a rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding said TCR component. In addition, expression of immunosuppressive polypeptide can be performed on those modified T-cells in order to prolong the survival of these modified T cells in host organism. Such modified T-cell is particularly suitable for allogeneic transplantations, especially because it reduces both the risk of rejection by the host's immune system and the risk of developing graft versus host disease. The invention opens the way to standard and affordable adoptive immunotherapy strategies using T-Cells for treating cancer, infections and auto-immune diseases.
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2.发明申请公开(公告)号：US20180291343A1
公开(公告)日：2018-10-11
申请号：US15556558
申请日：2016-03-11
申请人： Cellectis
IPC分类号： C12N5/0783 , C07K16/28 , C07K14/705 , C12N15/11 , C12N9/22 , C07K14/74 , C07K14/005 , A61K35/17 , C12N5/00
摘要： The present invention relates to methods for developing engineered immune cells such as T-cells for immunotherapy that have a higher potential of persistence and/or engraftment in host organism. IN particular, this method involves an inactivation of at least one gene involved in self/non self recognition, combined with a step of contact with at least one non-endogenous immunosuppressive polypeptide. The invention allows the possibility for a standard and affordable adoptive immunotherapy, whereby the risk of GvH is reduced.
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3.发明申请USE OF MEGANUCELASES FOR INDUCING HOMOLOGOUS RECOMBINATION EX VIVO AND IN TOTO IN VERTEBRATE SOMATIC TISSUES AND APPLICATION THEREOF 审中-公开用于诱导异体重组的血管内皮生长因子及其在皮肤组织中的应用公开(公告)号：US20160273003A1
公开(公告)日：2016-09-22
申请号：US15080232
申请日：2016-03-24
申请人： Cellectis S.A.
发明人： Philippe DUCHATEAU , Christophe PEREZ , Sylvia BRUNEAU , Jean-Pierre CABANIOLS , Julianne SMITH , Agnes GOUBLE
CPC分类号： C12N15/907 , A01K67/0275 , A01K67/0278 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2267/03 , A01K2267/0337 , A61K38/00 , A61K38/1709 , A61K38/465 , A61K48/00 , A61K48/0058 , C07K14/435 , C07K2319/81 , C12N7/00 , C12N9/22 , C12N15/1058 , C12N15/8509 , C12N15/86 , C12N15/90 , C12N2730/10143 , C12N2799/022 , C12N2800/80 , C12N2830/002 , C12N2830/55 , C12N2840/20 , C12N2840/44 , C12Y301/00 , C12Y301/21004
摘要： A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y and a second variant of I-CreI variant having the amino acid sequence of accession number pdb 1g9y in a single polypeptide.
摘要翻译： 单链归巢内切核酸酶，其包含具有登录号pdb 1g9y的氨基酸序列的I-CreI的第一变体和在单个多肽中具有登录号pdb 1g9y的氨基酸序列的I-CreI变体的第二变体。
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公开(公告)号：US20240026376A1
公开(公告)日：2024-01-25
申请号：US18480890
申请日：2023-10-04
申请人： CELLECTIS
IPC分类号： C12N15/85 , C07K14/74 , C12N5/0783 , C07K14/705 , C12N15/113 , C12N15/90
CPC分类号： C12N15/85 , C07K14/70539 , C12N5/0636 , C07K14/70503 , C12N15/1138 , C12N15/907 , C07K2317/24 , C07K2317/622
摘要： The present invention pertains to engineered T-cells, method for their preparation and their use as medicament, particularly for immunotherapy. The engineered T-cells of the invention are characterized in that the expression of beta 2-microglobulin (B2M) and/or class II major histocompatibility complex transactivator (CIITA) is inhibited, e.g., by using rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding B2M and/or CIITA, or by using nucleic acid molecules which inhibit the expression of B2M and/or CIITA. In order to further render the T-cell non-alloreactive, at least one gene encoding a component of the T-cell receptor is inactivated, e.g., by using a rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding said TCR component. In addition, expression of immunosuppressive polypeptide can be performed on those modified T-cells in order to prolong the survival of these modified T cells in host organism. Such modified T-cell is particularly suitable for allogeneic transplantations, especially because it reduces both the risk of rejection by the host's immune system and the risk of developing graft versus host disease. The invention opens the way to standard and affordable adoptive immunotherapy strategies using T-Cells for treating cancer, infections and auto-immune diseases.
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5.发明申请公开(公告)号：US20170016025A1
公开(公告)日：2017-01-19
申请号：US15123974
申请日：2015-03-11
申请人： CELLECTIS
IPC分类号： C12N15/85 , C07K14/74 , C07K14/705 , C12N15/90 , C12N15/113
CPC分类号： C12N15/85 , C07K14/70503 , C07K14/70539 , C07K2317/24 , C07K2317/622 , C12N5/0636 , C12N15/1138 , C12N15/907
摘要： The present invention pertains to engineered T-cells, method for their preparation and their use as medicament, particularly for immunotherapy. The engineered T-cells of the invention are characterized in that the expression of beta 2-microglobulin (B2M) and/or class II major histocompatibility complex transactivator (CIITA) is inhibited, e.g., by using rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding B2M and/or CIITA, or by using nucleic acid molecules which inhibit the expression of B2M and/or CIITA. In order to further render the T-cell non-alloreactive, at least one gene encoding a component of the T-cell receptor is inactivated, e.g., by using a rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding said TCR component. In addition, expression of immunosuppressive polypeptide can be performed on those modified T-cells in order to prolong the survival of these modified T cells in host organism. Such modified T-cell is particularly suitable for allogeneic transplantations, especially because it reduces both the risk of rejection by the host's immune system and the risk of developing graft versus host disease. The invention opens the way to standard and affordable adoptive immunotherapy strategies using T-Cells for treating cancer, infections and auto-immune diseases.
摘要翻译： 本发明涉及工程化T细胞，其制备方法及其作为药物的用途，特别是用于免疫治疗。 本发明的工程化T细胞的特征在于，β2-微球蛋白（B2M）和/或II类主要组织相容性复合反式激活因子（CIITA）的表达受到抑制，例如通过使用能够选择性失活的稀有内切核酸酶 DNA切割编码B2M和/或CIITA的基因，或通过使用抑制B2M和/或CIITA表达的核酸分子。 为了进一步使T细胞非同种异体反应，至少一种编码T细胞受体成分的基因被灭活，例如通过使用能够通过DNA切割选择性失活的稀有切割内切核酸酶来编码所述TCR的基因 零件。 此外，可以对这些修饰的T细胞进行免疫抑制多肽的表达，以延长宿主生物体中这些修饰的T细胞的存活。 这种修饰的T细胞特别适用于同种异体移植，特别是因为它降低宿主免疫系统的排斥风险和发生移植物抗宿主病的风险。 本发明开启了使用T细胞治疗癌症，感染和自身免疫疾病的标准和负担得起的过继免疫治疗策略的方法。
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公开(公告)号：US20230287454A1
公开(公告)日：2023-09-14
申请号：US18303080
申请日：2023-04-19
申请人： CELLECTIS
IPC分类号： C12N15/86 , C12N5/0783 , C12N9/22 , C12N13/00 , C12N15/113 , C12N15/90
CPC分类号： C12N15/86 , C12N5/0636 , C12N9/22 , C12N13/00 , C12N15/113 , C12N15/902 , C12N2310/20 , C12N2510/00 , C12N2800/80
摘要： The invention pertains to the field of adaptive cell immunotherapy. It aims at reducing the occurrence of translocations and cell deaths when several specific endonuclease reagents are used altogether to genetically modify primary immune cells at different genetic loci. The method of the invention allows to yield safer immune primary cells harboring several genetic modifications, such as triple or quadruple gene inactivated cells, from populations or sub-populations of cells originating from a single donor or patient, for their subsequent use in therapeutic treatments.
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公开(公告)号：US20230087122A1
公开(公告)日：2023-03-23
申请号：US17817877
申请日：2022-08-05
申请人： CELLECTIS
IPC分类号： C12N15/86 , C12N5/0783 , C12N9/22 , C12N13/00 , C12N15/113 , C12N15/90
摘要： The invention pertains to the field of adaptive cell immunotherapy. It aims at reducing the occurrence of translocations and cell deaths when several specific endonuclease reagents are used altogether to genetically modify primary immune cells at different genetic loci. The method of the invention allows to yield safer immune primary cells harboring several genetic modifications, such as triple or quadruple gene inactivated cells, from populations or sub-populations of cells originating from a single donor or patient, for their subsequent use in therapeutic treatments.
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公开(公告)号：US20200208174A1
公开(公告)日：2020-07-02
申请号：US16314697
申请日：2017-06-30
申请人： CELLECTIS
IPC分类号： C12N15/86 , C12N13/00 , C12N5/0783 , C12N9/22 , C12N15/113 , C12N15/90
摘要： The invention pertains to the field of adaptive cell immunotherapy. It aims at reducing the occurrence of translocations and cell deaths when several specific endonuclease reagents are used altogether to genetically modify primary immune cells at different genetic loci. The method of the invention allows to yield safer immune primary cells harboring several genetic modifications, such as triple or quadruple gene inactivated cells, from populations or sub-populations of cells originating from a single donor or patient,for their subsequent use in therapeutic treatments.
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9.发明申请公开(公告)号：US20180171298A1
公开(公告)日：2018-06-21
申请号：US15578946
申请日：2016-06-30
申请人： Cellectis
IPC分类号： C12N5/0783 , C12N15/63 , A61K35/17 , A61K45/06 , C12N9/22 , C07K14/47 , C07K14/725 , C12N9/12
CPC分类号： C12N5/0646 , A61K35/17 , A61K45/06 , A61K2039/515 , A61K2039/572 , C07K14/4703 , C07K14/7051 , C12N9/12 , C12N9/22 , C12N15/63 , C12N2510/00
摘要： The present invention relates to methods for improving therapeutic activity of NK cell, such as their cytotoxic/cytolytic activity, to be used in immunotherapy, by gene editing. In particular, these methods comprise a step of reduction or inactivation of gene expression using specific endonuclease such as TAL-nuclease, CRISPR or Argonaute. An additional genetic modification can be performed by (over)expressing at least one gene involved in N K function. The present invention encompasses also engineered NK cell, pharmaceutical composition containing the same.
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