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公开(公告)号:US11858884B2
公开(公告)日:2024-01-02
申请号:US17566666
申请日:2021-12-30
Inventor: Daniel J. Siegwart , Kejin Zhou
IPC: C07C321/14 , A61K9/51 , A61K31/7105 , A61K31/7125 , A61K47/59 , A61K47/60 , A61K47/69 , A61K48/00 , C07C323/52 , C07D295/15 , A61K47/20 , A61K47/22
CPC classification number: C07C321/14 , A61K9/513 , A61K9/5146 , A61K31/7105 , A61K31/7125 , A61K47/20 , A61K47/22 , A61K47/59 , A61K47/60 , A61K47/6931 , A61K48/0041 , C07C323/52 , C07D295/15
Abstract: Disclosed herein are methods for modulating an amount or activity of a gene or a gene product in a cell. The methods herein may comprise contacting a cell with a therapeutic agent assembled with a lipid composition, which lipid composition may comprise a dendrimer or dendron which may comprise one or more degradable diacyl group, in which may result in modulating the amount or activity of the gene or the gene product in the cell. The therapeutic agent modulating a gene or gene product in a cell may be sufficient to treat a disease or disorder in a subject. Further disclosed herein are pharmaceutical compositions, kits, and lipid compositions for modulating an amount or activity of a gene or a gene product in a cell.
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公开(公告)号:US10562849B2
公开(公告)日:2020-02-18
申请号:US15597049
申请日:2017-05-16
Inventor: Daniel J. Siegwart , Jason B. Miller
IPC: C07C311/32 , A61K48/00 , C07C309/14 , C12N15/11 , C12N15/113
Abstract: The present disclosure provides one or more amino lipids such as an amino lipids containing a sulfonic acid or sulfonic acid derivative of the formulas: wherein the variables are as defined herein. These amino lipids may be used in compositions with one or more helper lipids and a nucleic acid therapeutic agent. These compositions may be used to treat a disease or disorder such as cancer, cystic fibrosis, or other genetic diseases.
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公开(公告)号:US11648209B2
公开(公告)日:2023-05-16
申请号:US17839699
申请日:2022-06-14
Inventor: Qiang Cheng , Tuo Wei , Daniel J. Siegwart
CPC classification number: A61K9/5123 , A61K48/0033 , C12N9/22 , C12N15/11 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/20 , C12N2320/32
Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.
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公开(公告)号:US11590085B2
公开(公告)日:2023-02-28
申请号:US17572615
申请日:2022-01-10
Inventor: Qiang Cheng , Tuo Wei , Daniel J. Siegwart
Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.
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公开(公告)号:US20170349543A1
公开(公告)日:2017-12-07
申请号:US15597049
申请日:2017-05-16
Inventor: Daniel J. Siegwart , Jason B. Miller
IPC: C07C311/32 , C07C309/14 , C12N15/113 , C12N15/11 , A61K48/00 , C12N2310/20
Abstract: The present disclosure provides one or more amino lipids such as an amino lipids containing a sulfonic acid or sulfonic acid derivative of the formulas: wherein the variables are as defined herein. These amino lipids may be used in compositions with one or more helper lipids and a nucleic acid therapeutic agent. These compositions may be used to treat a disease or disorder such as cancer, cystic fibrosis, or other genetic diseases.
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公开(公告)号:US09801944B2
公开(公告)日:2017-10-31
申请号:US15261245
申请日:2016-09-09
Inventor: Daniel J. Siegwart , Jing Hao , Kejin Zhou , Jason Miller , Petra Kos , Lian Xue
IPC: A61K47/34 , C07H21/02 , C07H21/04 , A61K47/28 , A61K47/22 , C08G65/48 , C08G63/91 , A61K31/713 , A61K9/00 , A61K47/24 , C08G63/688 , C12N15/113 , A61K9/51
CPC classification number: A61K47/34 , A61K9/0019 , A61K9/5153 , A61K31/713 , A61K47/22 , A61K47/24 , A61K47/28 , C08G63/688 , C08G63/912 , C08G65/48 , C12N15/113 , C12N2310/14
Abstract: Polymers produced by ring opening polymerization which comprises an amino group that can be used in compositions to deliver a nucleic acid such as a miRNA or a siRNA. In some embodiments, compositions which comprise the polymers described herein and a nucleic acid are also provided herein. In some embodiments, these compositions are used to silence one or more genes in vivo or treat a disease or disorder.
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公开(公告)号:US20240401007A1
公开(公告)日:2024-12-05
申请号:US18811649
申请日:2024-08-21
Inventor: John W. Schoggins , Wenchun Fan , Daniel J. Siegwart , Qiang Cheng
Abstract: Embodiments of the instant disclosure relate to novel antiviral compositions and methods for treating viral infections. In accordance with these embodiments, antiviral compositions can include at least one mRNA encoding for a TRIM7 protein encapsulated into a lipid nanoparticle (LNP). In other embodiments, methods of making antiviral compositions are disclosed as well as methods of administering a composition having at least one mRNA encoding for a TRIM7 protein encapsulated into LNP into a subject.
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公开(公告)号:US11766408B2
公开(公告)日:2023-09-26
申请号:US17191975
申请日:2021-03-04
Inventor: Qiang Cheng , Tuo Wei , Daniel J. Siegwart
IPC: A61K9/51 , A61K9/127 , A61K31/7105 , A61K8/55 , A61K31/713 , A61K38/46 , C07F9/10
CPC classification number: A61K9/5123 , A61K8/553 , A61K9/127 , A61K9/1271 , A61K9/1272 , A61K31/713 , A61K31/7105 , A61K38/465 , C07F9/10 , A61K9/51 , C12N2320/32
Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.
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公开(公告)号:US11510880B2
公开(公告)日:2022-11-29
申请号:US17711911
申请日:2022-04-01
Inventor: Qiang Cheng , Tuo Wei , Daniel J. Siegwart
Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.
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公开(公告)号:US20210023120A1
公开(公告)日:2021-01-28
申请号:US17064505
申请日:2020-10-06
Inventor: Daniel J. Siegwart , Philip J. Thomas , Arthur E. Johnson , Michael J. Torres , Jason B. Miller
IPC: A61K31/7105 , A61K9/127 , A61K9/51 , A61K31/7088 , A61K47/18 , A61K47/20 , A61K47/34
Abstract: In some aspects, the present disclosure provides a nanoparticle composition comprising tRNA and an aminolipid delivery compound. The aminolipid delivery compound may be a dendrimer, dendron, or dendritic lipid, a polymer such as a polyamide or polyester, or a lipid with one or more hydrophobic components. In some embodiments, these compositions may be administered to a patient to treat a genetic disease or disorder such as cystic fibrosis, Duchene muscular dystrophy, or cancer.
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