公开(公告)号：US20240245620A1

公开(公告)日：2024-07-25

申请号：US18590737

申请日：2024-02-28

申请人： The Board of Regents of The University of Texas System

发明人： Qiang CHENG ,  Tuo WEI ,  Daniel J. SIEGWART

IPC分类号： A61K9/51 ,  A61K48/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113

CPC分类号： A61K9/5123 ,  A61K48/0033 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2320/32

摘要： 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.

公开(公告)号：US20240025848A1

公开(公告)日：2024-01-25

申请号：US18335990

申请日：2023-06-15

申请人： The Board of Regents of The University of Texas System

发明人： Daniel J. SIEGWART ,  Kejin ZHOU

IPC分类号： 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分类号： 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

摘要： 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.

公开(公告)号：US20240325315A1

公开(公告)日：2024-10-03

申请号：US18744370

申请日：2024-06-14

申请人： The Board of Regents of The University of Texas System

发明人： Daniel J. SIEGWART ,  Qiang CHENG

IPC分类号： A61K9/51 ,  A61K48/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113

CPC分类号： A61K9/5123 ,  A61K48/0033 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2320/32

摘要： 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.

公开(公告)号：US20240207442A1

公开(公告)日：2024-06-27

申请号：US18556614

申请日：2022-04-22

申请人： The Board of Regents of The University of Texas System

发明人： Daniel J. SIEGWART ,  Lukas FARBIAK

IPC分类号： A61K48/00 ,  A61K9/51 ,  C12N9/22 ,  C12N15/10 ,  C12N15/11 ,  C12N15/88 ,  C12N15/90

CPC分类号： A61K48/0041 ,  A61K9/5123 ,  A61K48/005 ,  C12N9/22 ,  C12N15/102 ,  C12N15/11 ,  C12N15/88 ,  C12N15/907 ,  C12N2310/20

摘要： 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.

公开(公告)号：US20240116859A1

公开(公告)日：2024-04-11

申请号：US18508192

申请日：2023-11-13

申请人： The Board of Regents of The University of Texas System

发明人： Daniel J. SIEGWART ,  Kejin ZHOU

IPC分类号： 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分类号： 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

摘要： 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.

公开(公告)号：US20220387337A1

公开(公告)日：2022-12-08

申请号：US17839699

申请日：2022-06-14

申请人： The Board of Regents of The University of Texas System

发明人： Qiang CHENG ,  Tuo WEI ,  Daniel J. SIEGWART

IPC分类号： A61K9/51 ,  A61K48/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113

摘要： 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.

公开(公告)号：US20220133637A1

公开(公告)日：2022-05-05

申请号：US17572615

申请日：2022-01-10

申请人： The Board of Regents of The University of Texas System

发明人： Qiang CHENG ,  Tuo WEI ,  Daniel J. SIEGWART

IPC分类号： A61K9/51 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  A61K48/00

摘要： 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.

公开(公告)号：US20240010614A1

公开(公告)日：2024-01-11

申请号：US18335974

申请日：2023-06-15

申请人： The Board of Regents of The University of Texas System

发明人： Daniel J. SIEGWART ,  Kejin ZHOU

IPC分类号： 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分类号： 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

摘要： 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.

公开(公告)号：US20230026843A1

公开(公告)日：2023-01-26

申请号：US17929704

申请日：2022-09-04

申请人： The Board of Regents of The University of Texas System

发明人： Qiang CHENG ,  Tuo WEI ,  Daniel J. SIEGWART

IPC分类号： A61K9/51 ,  A61K48/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113

摘要： 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.

公开(公告)号：US20220257792A1

公开(公告)日：2022-08-18

申请号：US17566666

申请日：2021-12-30

申请人： The Board of Regents of The University of Texas System

发明人： Daniel J. SIEGWART ,  Kejin ZHOU

IPC分类号： A61K48/00 ,  A61K9/127 ,  A61K31/7105 ,  A61K47/34

摘要： 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.