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公开(公告)号：US20240245620A1
公开(公告)日：2024-07-25
申请号：US18590737
申请日：2024-02-28
Inventor： Qiang CHENG , Tuo WEI , Daniel J. SIEGWART
IPC: A61K9/51 , A61K48/00 , C12N9/22 , C12N15/11 , C12N15/113
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：US20240025848A1
公开(公告)日：2024-01-25
申请号：US18335990
申请日：2023-06-15
Inventor： Daniel J. SIEGWART , Kejin ZHOU
IPC: C07C321/14 , A61K47/59 , C07C323/52 , A61K9/51 , A61K47/69 , A61K47/60 , A61K31/7105 , A61K48/00 , A61K31/7125 , A61K47/20 , A61K47/22 , C07D295/15
CPC classification number: C07C321/14 , A61K47/59 , C07C323/52 , A61K9/5146 , A61K47/6931 , A61K47/60 , A61K9/513 , A61K31/7105 , A61K48/0041 , 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：US20250064752A1
公开(公告)日：2025-02-27
申请号：US18930467
申请日：2024-10-29
Inventor： Daniel J. SIEGWART , Philip J. THOMAS , Arthur E. JOHNSON , Michael J. TORRES , Jason B. MILLER
IPC: A61K9/51 , A61K48/00 , C12N9/22 , C12N15/11 , C12N15/113
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：US20240325315A1
公开(公告)日：2024-10-03
申请号：US18744370
申请日：2024-06-14
Inventor： Daniel J. SIEGWART , Qiang CHENG
IPC: A61K9/51 , A61K48/00 , C12N9/22 , C12N15/11 , C12N15/113
CPC classification number: A61K9/5123 , A61K48/0033 , C12N9/22 , C12N15/11 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/20 , C12N2320/32
Abstract: In some aspects, the present disclosure provides compositions of lipid nanoparticles useful for the delivery of large RNAs including mRNAs. These compositions may include a cationic ionizable lipid, a phospholipid, a PEGylated lipid, and a steroid including using less of a cationic ionizable lipid than compositions with shorter nucleic acids. These compositions may be used to treat a disease or disorder for which the delivery of an mRNA is therapeutically effective.
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5.发明公开公开(公告)号：US20240207442A1
公开(公告)日：2024-06-27
申请号：US18556614
申请日：2022-04-22
Inventor： Daniel J. SIEGWART , Lukas FARBIAK
CPC classification number: A61K48/0041 , A61K9/5123 , A61K48/005 , C12N9/22 , C12N15/102 , C12N15/11 , C12N15/88 , C12N15/907 , C12N2310/20
Abstract: In some aspects, the present disclosure provides compositions comprising one or more of each of the following nucleic acids: (1) a mRNA; (2) a sgRNA; and (3) a DNA; and a lipid nanoparticle comprising at least one ionizable lipid; wherein the each of the nucleic acids are encapsulated within the lipid nanoparticle, and pharmaceutical compositions thereof. The present disclosure also provides methods employing said compositions and/or pharmaceutical compositions, such as methods of repairing genes, methods of performing homology directed repair on the genome, and methods of treating diseases or disorders.
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Patent Agency Ranking
公开(公告)号：US20240116859A1
公开(公告)日：2024-04-11
申请号：US18508192
申请日：2023-11-13
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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公开(公告)号：US20220387337A1
公开(公告)日：2022-12-08
申请号：US17839699
申请日：2022-06-14
Inventor： Qiang CHENG , Tuo WEI , Daniel J. SIEGWART
IPC: A61K9/51 , A61K48/00 , C12N9/22 , C12N15/11 , C12N15/113
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：US20220133637A1
公开(公告)日：2022-05-05
申请号：US17572615
申请日：2022-01-10
Inventor： Qiang CHENG , Tuo WEI , Daniel J. SIEGWART
IPC: A61K9/51 , C12N9/22 , C12N15/11 , C12N15/113 , A61K48/00
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：US20240391869A1
公开(公告)日：2024-11-28
申请号：US18800894
申请日：2024-08-12
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
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：US20240010614A1
公开(公告)日：2024-01-11
申请号：US18335974
申请日：2023-06-15
Inventor： Daniel J. SIEGWART , Kejin ZHOU
IPC: C07C321/14 , A61K47/59 , C07C323/52 , A61K9/51 , A61K47/69 , A61K47/60 , A61K31/7105 , A61K48/00 , A61K31/7125 , A61K47/20 , A61K47/22 , C07D295/15
CPC classification number: C07C321/14 , A61K47/59 , C07C323/52 , A61K9/5146 , A61K47/6931 , A61K47/60 , A61K9/513 , A61K31/7105 , A61K48/0041 , 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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