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公开(公告)号:US20170354672A1
公开(公告)日:2017-12-14
申请号:US15597063
申请日:2017-05-16
Inventor: Daniel J. Siegwart , Philip J. Thomas , Arthur E. Johnson , Michael J. Torres , Jason B. Miller
IPC: A61K31/7105 , A61K47/18 , A61K47/20 , A61K47/34
CPC classification number: A61K31/7105 , A61K9/127 , A61K9/5123 , 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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公开(公告)号:US12252464B2
公开(公告)日:2025-03-18
申请号:US18315446
申请日:2023-05-10
Inventor: Daniel J. Siegwart , Jason B. Miller
IPC: C07C311/32 , A61K47/54 , A61K47/69 , A61K48/00 , B82Y5/00 , C07C229/06 , C07C309/14 , C07F9/09 , C07F9/10 , C12N15/11 , C12N15/113 , C12N15/88
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:US12104181B2
公开(公告)日:2024-10-01
申请号:US17710464
申请日:2022-03-31
Inventor: John W. Schoggins , Wenchun Fan , Daniel J. Siegwart , Qiang Cheng
CPC classification number: C12N9/104 , A61K9/5123 , A61K48/0066 , A61P31/14
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:US20240216515A1
公开(公告)日:2024-07-04
申请号:US18283406
申请日:2022-03-21
Inventor: Michael Torres , Daniel J. Siegwart , Jackson Eby , Rumpa Bhattacherjee , Brandon Wustman , Vladimir Kharitonov , Qiang Cheng , Tuo Wei
IPC: A61K47/54 , A61K47/14 , A61K47/24 , A61K47/28 , C12N15/113
CPC classification number: A61K47/543 , A61K47/14 , A61K47/24 , A61K47/28 , A61K47/545 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/141 , C12N2310/531
Abstract: Described herein are compositions, kits, and methods for potent delivery to a ceil of a subject. The cell can be of a particular cell type, such as a basal cell. In some cases, the cell can be a lung cell of a particular cell type. Also described herein are pharmaceutical compositions comprising a therapeutic or prophylactic agent assembled with a lipid composition. Tire lipid composition can comprise an ionizable cationic lipid, and a selective organ targeting lipid. The lipid composition can further comprise a phospholipid. Further described herein are high-potency intravenous dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.
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公开(公告)号:US12024486B2
公开(公告)日:2024-07-02
申请号:US18335974
申请日:2023-06-15
Inventor: Daniel J. Siegwart , Kejin Zhou
IPC: A61K9/51 , A61K31/7105 , A61K31/7125 , A61K47/20 , A61K47/22 , A61K47/59 , A61K47/60 , A61K47/69 , A61K48/00 , C07C321/14 , C07C323/52 , C07D295/15
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: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.
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公开(公告)号:US11648210B2
公开(公告)日:2023-05-16
申请号:US17929704
申请日:2022-09-04
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:US11247968B2
公开(公告)日:2022-02-15
申请号:US15265064
申请日:2016-09-14
Inventor: Daniel J. Siegwart , Kejin Zhou
IPC: C07C321/14 , A61K47/59 , C07C323/52 , A61K9/51 , A61K31/7125 , A61K47/20 , A61K47/22 , C07D295/15
Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.
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公开(公告)号:US09517270B2
公开(公告)日:2016-12-13
申请号:US14962422
申请日:2015-12-08
Inventor: Daniel J. Siegwart , Jing Hao , Kejin Zhou , Jason Miller , Petra Kos , Lian Xue
IPC: A61K47/34 , C07H21/02 , C07H21/04 , A61K47/28 , A61K47/24 , A61K47/22 , C08G65/48 , C08G63/91 , A61K31/713
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.
Abstract translation: 通过开环聚合制备的聚合物,其包含可用于递送核酸如miRNA或siRNA的组合物中的氨基。 在一些实施方案中,本文还提供了包含本文所述的聚合物和核酸的组合物。 在一些实施方案中,这些组合物用于在体内沉默一种或多种基因或治疗疾病或病症。
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公开(公告)号:US12133924B2
公开(公告)日:2024-11-05
申请号:US18534974
申请日:2023-12-11
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:US11999683B2
公开(公告)日:2024-06-04
申请号:US18335960
申请日:2023-06-15
Inventor: Daniel J. Siegwart , Kejin Zhou
IPC: C07C321/14 , A61K9/51 , A61K31/7105 , A61K31/7125 , A61K47/20 , A61K47/22 , A61K47/59 , A61K47/60 , A61K47/69 , A61K48/00 , C07C323/52 , C07D295/15
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: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.
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