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公开(公告)号：US20230338411A1
公开(公告)日：2023-10-26
申请号：US18312305
申请日：2023-05-04
发明人： Mirko Hennig , Vladimir Kharitonov , Brandon A. Wustman , Jackson Eby , Rumpa Bhattacharjee , Daniel Siegwart
IPC分类号： A61K31/7115 , A61K47/28 , A61K47/24 , A61K9/127 , A61K9/51
CPC分类号： A61K31/7115 , A61K9/1272 , A61K9/51 , A61K47/24 , A61K47/28
摘要： Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory 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 to a lipid composition. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Further described herein are high-potency dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.
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公开(公告)号：US20230302138A1
公开(公告)日：2023-09-28
申请号：US18311616
申请日：2023-05-03
发明人： Mirko Hennig , Vladimir Kharitonov , Brandon A. Wustman , Jackson Eby , Rumpa Bhattacharjee , Daniel Siegwart
CPC分类号： A61K47/543 , C12N15/113 , A61K47/544 , A61K47/10 , A61K47/28 , A61K9/0073 , A61P11/00
摘要： Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory 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 to a lipid composition. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Further described herein are high-potency dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.
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公开(公告)号：US20240207178A1
公开(公告)日：2024-06-27
申请号：US18283344
申请日：2022-03-22
发明人： Mirko Hennig , Vladimir Kharitonov , Brandon Wustman , Jackson Eby , Rumpa Bhattacharjee , Daniel Siegwart
CPC分类号： A61K9/0078 , A61K31/7105 , A61K38/465 , A61K47/183 , A61K47/22 , A61K47/24 , C12N15/11
摘要： Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory 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 to a lipid composition. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Further described herein are high-potency dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.
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公开(公告)号：US20230293429A1
公开(公告)日：2023-09-21
申请号：US18311274
申请日：2023-05-03
发明人： Mirko Hennig , Vladimir Kharitonov , Brandon A. Wustman , Jackson Eby , Rumpa Bhattacharjee , Daniel Siegwart
CPC分类号： A61K9/0073 , C12N15/88 , C12Y306/04002 , C12N9/14 , A61K47/14 , A61K47/24 , C07K14/47
摘要： Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory 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 to a lipid composition. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Further described herein are high-potency dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.
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公开(公告)号：US20240285533A1
公开(公告)日：2024-08-29
申请号：US18593245
申请日：2024-03-01
发明人： Mirko Hennig , Ali Ahmed Alfaifi , Sakya Sing Mohapatra , Daniella Ishimaru , Vladimir Grigor'evich Kharitonov , Julia Jung-un Baek , Shuang Li , David J. Lockhart , Brandon A. Wustman , Yufeng Wang , Joseph S. Cefalu
CPC分类号： A61K9/145 , A61K9/0078 , A61K47/24 , A61K47/26 , A61K47/28 , A61K48/0008 , C07K14/705
摘要： Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for cystic fibrosis (CF), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject, wherein the LNPs comprise mRNA encoding a Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein.
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公开(公告)号：US20240261432A1
公开(公告)日：2024-08-08
申请号：US18596148
申请日：2024-03-05
发明人： Vladimir Kharitonov , Brandon Wustman , Jackson Eby , Rumpa Bhattacharjee , Daniella Ishimaru , Mirko Hennig
CPC分类号： A61K48/0033 , A61K9/0073 , A61K9/5123 , A61K38/1709 , A61K47/18 , A61K47/20 , A61K47/22 , A61P11/00
摘要： Compositions of polynucleotide(s) are disclosed. A polynucleotide may encode for a polypeptide, protein, or functional fragment thereof associated with primary ciliary dyskinesia (PCD), such as dynein axonemal intermediate chain 1 (DNAI1). Pharmaceutical compositions, kits, and methods for treating a disease or condition associated with cilia maintenance and function, and impaired function of the axoneme are also disclosed. The polynucleotide may be assembled with a lipid composition for delivery to an organ, such as the lung, of a subject. The lipid composition may comprise an ionizable cationic lipid. The polynucleotide can be expressed within cells of the organ of the subject.
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公开(公告)号：US20240269325A1
公开(公告)日：2024-08-15
申请号：US18431504
申请日：2024-02-02
发明人： Mirko Hennig , Daniella Ishimaru , David J. Lockhart , Michael Torres , Jackson Eby , Dmitri Boudko , Brandon Wustman
CPC分类号： A61K48/005 , A61K9/0078 , A61K9/12 , A61K9/1271 , A61K9/5123 , A61K38/1709 , A61K48/0075
摘要： Compositions of polynucleotide(s), pharmaceutical compositions thereof, and methods of use thereof are disclosed. A polynucleotide may encode for a cystic fibrosis transmembrane conductance regulator (CFTR) protein or a functional fragment thereof. The polynucleotide may be assembled with a lipid composition for targeted delivery to a cell or an organ, such as a lung cell or a lung of a subject. Methods for enhancing an expression or activity of CFTR protein in a cell are provided. Methods for treating a subject having or suspected of having a CFTR-associated condition are also provided.
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公开(公告)号：US20240299310A1
公开(公告)日：2024-09-12
申请号：US18596141
申请日：2024-03-05
发明人： Vladimir Kharitonov , Brandon Wustman , Jackson Eby , Rumpa Bhattacharjee , Daniella Ishimaru , Mirko Hennig
CPC分类号： A61K9/5123 , A61K9/0073 , A61K38/1709 , A61K48/0033 , A61P11/00
摘要： Compositions of polynucleotide(s) are disclosed. A polynucleotide may encode for a polypeptide, protein, or functional fragment thereof associated with primary ciliary dyskinesia (PCD), such as dynein axonemal intermediate chain 1 (DNAI1). Pharmaceutical compositions, kits, and methods for treating a disease or condition associated with cilia maintenance and function, and impaired function of the axoneme are also disclosed. The polynucleotide may be assembled with a lipid composition for delivery to an organ, such as the lung, of a subject. The lipid composition may comprise an ionizable cationic lipid. The polynucleotide can be expressed within cells of the organ of the subject.
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公开(公告)号：US20240216290A1
公开(公告)日：2024-07-04
申请号：US18413653
申请日：2024-01-16
发明人： Mirko Hennig , Ali Alfaifi , Sakya Sing Mohapatra , Daniella Ishimaru , Vladimir Kharitonov , Jung Un Baek , Shuang Li , David J. Lockhart , Brandon A. Wustman , Yufeng Wang , Joe Cefalu
CPC分类号： A61K9/5123 , A61K9/0078 , A61K48/0033
摘要： Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for primary ciliary dyskinesia (PCD), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject.
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公开(公告)号：US12121610B2
公开(公告)日：2024-10-22
申请号：US18311616
申请日：2023-05-03
IPC分类号： A61K47/14 , A61K9/00 , A61K9/127 , A61K9/51 , A61K31/7115 , A61K47/10 , A61K47/24 , A61K47/28 , A61K47/54 , A61P11/00 , C07K14/47 , C12N9/14 , C12N15/113 , C12N15/88
CPC分类号： A61K9/0073 , A61K9/1272 , A61K9/51 , A61K31/7115 , A61K47/10 , A61K47/14 , A61K47/24 , A61K47/28 , A61K47/543 , A61K47/544 , A61P11/00 , C07K14/47 , C12N9/14 , C12N15/113 , C12N15/88 , C12Y306/04002
摘要： Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory 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 to a lipid composition. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Further described herein are high-potency dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.
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