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公开(公告)号：US20230070540A1
公开(公告)日：2023-03-09
申请号：US17818669
申请日：2022-08-09
摘要： Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.
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公开(公告)号：US20220169988A1
公开(公告)日：2022-06-02
申请号：US17538699
申请日：2021-11-30
发明人： Viktoriia KYRYCHENKO , Wai Lun LEUNG , Alireza REZANIA , Valentin SLUCH , Danielle SWAIN , Patrick Claudio OVANDO ROCHE
IPC分类号： C12N5/0783 , C12N5/074 , C12N5/0735 , C12N5/10 , C12N15/66
摘要： The present invention relates to, inter alia, an engineered cell (e.g., iPSC, IPS-derived NK, or NK cell) comprising a disrupted B2M gene and an inserted polynucleotide encoding one or more of SERPINB9, a fusion of IL15 and IL15Rα, and/or HLA-E. The engineered cell can further comprise a disrupted CIITA gene and an inserted polynucleotide encoding a CAR, wherein the CAR can be an anti-BCMA CAR or an anti-CD30 CAR. The engineered cell may further comprise a disrupted ADAM17 gene, a disrupted FAS gene, a disrupted CISH gene, and/or a disrupted REGNASE-1 gene. Methods for producing the engineered cells are also provided, and therapeutic uses of the engineered cells are also described. Guide RNA sequences targeting described target sequences are also described.
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公开(公告)号：US20210071201A1
公开(公告)日：2021-03-11
申请号：US17013208
申请日：2020-09-04
IPC分类号： C12N15/90 , C07K14/705 , C07K14/74 , C12N5/10 , C12N5/0735 , C12N5/074
摘要： Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.
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公开(公告)号：US20210070837A1
公开(公告)日：2021-03-11
申请号：US17013162
申请日：2020-09-04
IPC分类号： C07K14/74 , C12N15/90 , C07K14/705 , C12N5/0735 , C12N5/074
摘要： Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.
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公开(公告)号：US20210070835A1
公开(公告)日：2021-03-11
申请号：US17013135
申请日：2020-09-04
IPC分类号： C07K14/74 , A61K35/39 , C12N5/0735 , C12N5/074
摘要： Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.
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公开(公告)号：US20230130564A1
公开(公告)日：2023-04-27
申请号：US17823091
申请日：2022-08-30
发明人： Valentin SLUCH , Alireza REZANIA , Jason SAGERT
IPC分类号： C07K14/74 , A61K35/17 , C07K14/54 , C07K14/725 , C07K14/715 , C07K14/81 , C07K16/28 , C12N5/0783 , C12N15/90 , C12N5/0735 , C12N5/074 , C12N5/10 , C12N15/66
摘要： The present invention relates to, inter alia, an engineered cell (e.g., iPSC, IPS-derived NK, or NK cell) comprising a disrupted B2M gene and an inserted polynucleotide encoding one or more of SERPINB9, a fusion of IL15 and IL15Rα, and/or HLA-E. The engineered cell can further comprise a disrupted CIITA gene and an inserted polynucleotide encoding a CAR, wherein the CAR can be an anti-BCMA CAR or an anti-CD30 CAR. The engineered cell may further comprise a disrupted ADAM17 gene, a disrupted FAS gene, a disrupted CISH gene, and/or a disrupted REGNASE-1 gene. Methods for producing the engineered cells are also provided, and therapeutic uses of the engineered cells are also described. Guide RNA sequences targeting described target sequences are also described.
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公开(公告)号：US20220204934A1
公开(公告)日：2022-06-30
申请号：US17538719
申请日：2021-11-30
IPC分类号： C12N5/0783 , C12N5/0789
摘要： The disclosure features methods and compositions for differentiating stem cells into hematopoietic stem and progenitor cells (HSPC) and/or Natural Killer (NK) cells. The methods and compositions described herein are used to differentiate stem or progenitor cells having at least one gene-edit that is maintained in the differentiated cell. Also provided are differentiated cells produced using the methods and compositions described herein for therapeutic applications.
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公开(公告)号：US20220169700A1
公开(公告)日：2022-06-02
申请号：US17538566
申请日：2021-11-30
发明人： Viktoriia KYRYCHENKO , Wai Lun LEUNG , Alireza REZANIA , Valentin SLUCH , Danielle SWAIN , Patrick Claudio OVANDO ROCHE
IPC分类号： C07K14/74 , C07K14/54 , C07K14/715 , C07K14/81 , A61K35/17 , C12N5/0783 , C07K14/725 , C07K16/28 , C12N15/90
摘要： The present invention relates to, inter alia, an engineered cell (e.g., iPSC, IPS-derived NK, or NK cell) comprising a disrupted B2M gene and an inserted polynucleotide encoding one or more of SERPINB9, a fusion of IL15 and IL15Rα, and/or HLA-E. The engineered cell can further comprise a disrupted CIITA gene and an inserted polynucleotide encoding a CAR, wherein the CAR can be an anti-BCMA CAR or an anti-CD30 CAR. The engineered cell may further comprise a disrupted ADAM17 gene, a disrupted FAS gene, a disrupted CISH gene, and/or a disrupted REGNASE-1 gene. Methods for producing the engineered cells are also provided, and therapeutic uses of the engineered cells are also described. Guide RNA sequences targeting described target sequences are also described.
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公开(公告)号：US20230016422A1
公开(公告)日：2023-01-19
申请号：US17830273
申请日：2022-06-01
发明人： Valentin SLUCH , Danielle SWAIN , Alireza REZANIA
摘要： Provided herein are cells engineered to have improved protection against natural killer cell killing. The cells are engineered to comprise an insertion of a polynucleotide encoding SERPINB9. Also provided herein are methods of making the engineered cells and therapeutic uses of the engineered cells. The engineered cells can also comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor. The engineered cells can be stem cells and the engineered stem cells can be differentiated into various lineages having protection against NK cell killing.
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公开(公告)号：US20210070836A1
公开(公告)日：2021-03-11
申请号：US17013154
申请日：2020-09-04
IPC分类号： C07K14/74 , C12N15/90 , C12N5/0735 , C12N5/074
摘要： Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.
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