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公开(公告)号：US20180161447A1
公开(公告)日：2018-06-14
申请号：US15842820
申请日：2017-12-14
申请人： Ligandal, Inc.
摘要： Provided are methods and compositions for delivering a nucleic acid, protein, and/or ribonucleoprotein payload to a cell. Also provided are delivery molecules that include a peptide targeting ligand conjugated to a protein or nucleic acid payload (e.g., an siRNA molecule), or conjugated to a charged polymer polypeptide domain (e.g., poly-arginine such as 9R or a poly-histidine such as 6H, and the like). The targeting ligand provides for (i) targeted binding to a cell surface protein, and (ii) engagement of a long endosomal recycling pathway. As such, when the targeting ligand engages the intended cell surface protein, the delivery molecule enters the cell (e.g., via endocytosis) but is preferentially directed away from the lysosomal degradation pathway.
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公开(公告)号：US12123012B2
公开(公告)日：2024-10-22
申请号：US17104552
申请日：2020-11-25
申请人： LIGANDAL, INC.
IPC分类号： A61K48/00 , A61K9/00 , A61K9/51 , A61K31/40 , A61K31/7105 , A61K47/34 , A61K47/42 , A61K47/54 , A61K47/64 , A61K47/68 , A61P3/10 , A61P35/00 , A61P37/00 , B82Y5/00 , C12N15/10 , C12N15/11 , C12N15/85 , C12N15/87 , C12N9/22
CPC分类号： C12N15/85 , A61K9/0019 , A61K9/0085 , A61K9/5146 , A61K31/40 , A61K31/7105 , A61K47/34 , A61K47/42 , A61K47/549 , A61K47/64 , A61K47/645 , A61K47/6455 , A61K47/6807 , A61K48/0016 , A61K48/0041 , A61K48/0075 , A61P3/10 , A61P35/00 , A61P37/00 , B82Y5/00 , C12N15/102 , C12N15/111 , C12N15/87 , C12N9/22 , C12N2310/14 , C12N2310/20 , C12N2310/3517 , C12N2320/32
摘要： Provided are methods and compositions for delivering a nucleic acid, protein, and/or ribonucleoprotein payload to a cell. Also provided are delivery molecules that include a peptide targeting ligand conjugated to a protein or nucleic acid payload (e.g., an siRNA molecule), or conjugated to a charged polymer polypeptide domain (e.g., poly-arginine such as 9R or a poly-histidine such as 6H, and the like). The targeting ligand provides for (i) targeted binding to a cell surface protein, and (ii) engagement of a long endosomal recycling pathway. As such, when the targeting ligand engages the intended cell surface protein, the delivery molecule enters the cell (e.g., via endocytosis) but is preferentially directed away from the lysosomal degradation pathway.
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公开(公告)号：US20180179553A1
公开(公告)日：2018-06-28
申请号：US15842829
申请日：2017-12-14
申请人： Ligandal, Inc.
IPC分类号： C12N15/85 , A61K47/34 , A61K47/64 , A61K48/00 , C12N15/10 , A61K9/51 , A61P35/00 , A61P3/10 , A61P37/00
摘要： Provided are methods and compositions for nanoparticle delivery of payloads (e.g., nucleic acid and/or protein payloads) to cells. In some embodiments, a subject nanoparticle includes a core and a sheddable layer encapsulating the core, where the core includes (i) an anionic polymer composition; (ii) a cationic polymer composition; (iii) a cationic polypeptide composition; and (iv) a nucleic acid and/or protein payload; and where: (a) the anionic polymer composition includes polymers of D-isomers of an anionic amino acid and polymers of L-isomers of an anionic amino acid, and/or (b) the cationic polymer composition comprises polymers of D-isomers of a cationic amino acid and polymers of L-isomers of a cationic amino acid. In some cases, the polymers of D-isomers of an anionic and/or cationic amino acid are present at a ratio, relative to the polymers of L-isomers, in a range of from 10:1 to 1:10.
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公开(公告)号：US20230059921A1
公开(公告)日：2023-02-23
申请号：US17453336
申请日：2021-11-02
申请人： Ligandal, Inc.
发明人： Andre Ronald Watson , Shahab Chizari , Ryan Spencer , Christian Foster , Shuailiang Lin , Sara Marie Peyrot , Pranali Deshpande , Matthew Dobbin , William Connors
IPC分类号： G01N33/566 , C40B30/04
摘要： Provided are methods and compositions for the heterologous expression of a payload (e.g., DNA, RNA, protein) of interest in a target cell (e.g., cancer cell). In some cases payload delivery results in expression (e.g., by a cancer cell in vivo) of a secreted immune signal such as a cytokine, a plasma membrane-tethered affinity marker (thus resulting in an induced immune response), or a cytotoxic protein such as an apoptosis inducer (e.g., by a cancer cell in vivo). Payloads are delivered with a delivery vehicle and in some cases the delivery vehicle is a nanoparticle. In some cases a subject nanoparticle includes a targeting ligand for targeted delivery to a specific cell type/tissue type (e.g., a cancerous tissue/cell). In some embodiments, payload delivery is “personalized” in the sense that the delivery vehicle and/or payload can be designed based on patient-specific information.
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公开(公告)号：US20200208177A1
公开(公告)日：2020-07-02
申请号：US16387507
申请日：2019-04-17
申请人： Ligandal, Inc.
摘要： Provided are methods and compositions for genome editing using sticky ends. Subject methods include (a) generating a staggered cut at each of two locations in genomic DNA of a target cell, thus generating two genomic staggered ends; and (b) providing/introducing a linear double stranded donor DNA that has staggered ends (i.e., sticky ends) that match/correspond to the sticky ends of the genomic DNA such that the sticky ends of the donor DNA hybridize with the sticky ends of the genomic DNA and the donor DNA is inserted into the genome. In some cases, the staggered cuts are generated by introducing into a target cell one or more sequence specific nucleases (or one or more nucleic acids encoding the one or more sequence specific nucleases).
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公开(公告)号：US20230323401A1
公开(公告)日：2023-10-12
申请号：US18148964
申请日：2022-12-30
申请人： Ligandal, Inc.
IPC分类号： C12N15/90 , C07K14/705 , C12N9/22 , C12N15/113 , C12N15/52
CPC分类号： C12N15/90 , C07K14/70596 , C12N9/22 , C12N15/113 , C12N15/52 , C12N15/907 , C12N2310/20 , B82Y5/00
摘要： Provided are methods and compositions for genome editing using a delivery vehicle with multiple payloads. In some embodiments the delivery vehicle includes a payload that includes (a) one or more sequence specific nucleases that cleave the cell's genome or one or more nucleic acids encoding same, (b) a first donor DNA, which includes a nucleotide sequence that is inserted into the cell's genome, where insertion of said nucleotide sequence produces, in the cell's genome at the site of insertion, a target sequence (e.g., an attP site) for a site-specific recombinase; (c) the site-specific recombinase (or a nucleic acid encoding same) (e.g., ϕC31, ϕC31 RDF, Cre, FLP), where the site-specific recombinase recognizes said target sequence; and (d) a second donor DNA, which includes a nucleotide sequence that is inserted into the cell's genome as a result of recognition of said target sequence by the site-specific recombinase.
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公开(公告)号：US10975388B2
公开(公告)日：2021-04-13
申请号：US15842820
申请日：2017-12-14
申请人： Ligandal, Inc.
IPC分类号： A61K38/00 , C12N15/85 , A61K47/68 , A61K48/00 , A61K47/54 , A61K31/7105 , A61K31/40 , A61K47/42 , B82Y5/00 , A61K47/64 , C12N15/87 , A61K9/00 , C12N15/11 , A61P37/00 , A61P3/10 , A61P35/00 , A61K9/51 , A61K47/34 , C12N15/10 , C12N9/22
摘要： Provided are methods and compositions for delivering a nucleic acid, protein, and/or ribonucleoprotein payload to a cell. Also provided are delivery molecules that include a peptide targeting ligand conjugated to a protein or nucleic acid payload (e.g., an siRNA molecule), or conjugated to a charged polymer polypeptide domain (e.g., poly-arginine such as 9R or a poly-histidine such as 6H, and the like). The targeting ligand provides for (i) targeted binding to a cell surface protein, and (ii) engagement of a long endosomal recycling pathway. As such, when the targeting ligand engages the intended cell surface protein, the delivery molecule enters the cell (e.g., via endocytosis) but is preferentially directed away from the lysosomal degradation pathway.
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公开(公告)号：US20200149070A1
公开(公告)日：2020-05-14
申请号：US16393886
申请日：2019-04-24
申请人： Ligandal, Inc.
IPC分类号： C12N15/90 , C12N9/22 , C12N15/113 , C12N15/52 , C07K14/705
摘要： Provided are methods and compositions for genome editing using a delivery vehicle with multiple payloads. In some embodiments the delivery vehicle includes a payload that includes (a) one or more sequence specific nucleases that cleave the cell's genome or one or more nucleic acids encoding same, (b) a first donor DNA, which includes a nucleotide sequence that is inserted into the cell's genome, where insertion of said nucleotide sequence produces, in the cell's genome at the site of insertion, a target sequence (e.g., an attP site) for a site-specific recombinase; (c) the site-specific recombinase (or a nucleic acid encoding same) (e.g., ϕC31, ϕC31 RDF, Cre, FLP), where the site-specific recombinase recognizes said target sequence; and (d) a second donor DNA, which includes a nucleotide sequence that is inserted into the cell's genome as a result of recognition of said target sequence by the site-specific recombinase.
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