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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
Applicant: Joe Cefalu , ReCode Therapeutics, Inc.
Inventor: 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 classification number: A61K9/5123 , A61K9/0078 , A61K48/0033
Abstract: 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:US20240207178A1
公开(公告)日:2024-06-27
申请号:US18283344
申请日:2022-03-22
Inventor: Mirko Hennig , Vladimir Kharitonov , Brandon Wustman , Jackson Eby , Rumpa Bhattacharjee , Daniel Siegwart
CPC classification number: A61K9/0078 , A61K31/7105 , A61K38/465 , A61K47/183 , A61K47/22 , A61K47/24 , C12N15/11
Abstract: 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:US20240197641A1
公开(公告)日:2024-06-20
申请号:US18283615
申请日:2022-03-23
Inventor: David J. Lockhart , Vladimir Kharitonov , Brandon Wustman , Daniel Siegwart , Xueliang Yu , Jackson Eby
IPC: A61K9/51 , A61K9/00 , A61K31/7105
CPC classification number: A61K9/5123 , A61K9/0019 , A61K31/7105
Abstract: Described herein are compositions, kits, and methods for potent systemic delivery to a cell of a subject. 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:US20250019409A1
公开(公告)日:2025-01-16
申请号:US18283523
申请日:2022-03-23
Applicant: ReCode Therapeutics, Inc.
Inventor: Mirko Hennig , Daniella Ishimaru , David J. Lockhart , Michael Torres , Jackson Eby , Dmitri Boudko , Brandon A. Wustman
Abstract: 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:US20240390272A1
公开(公告)日:2024-11-28
申请号:US18797360
申请日:2024-08-07
Applicant: ReCode Therapeutics, Inc.
Inventor: Mirko Hennig , Vladimir Kharitonov , Brandon A. Wustman , Jackson Eby , Rumpa Bhattacharjee
IPC: A61K9/00 , A61K9/127 , A61K9/51 , A61K31/7105 , A61K31/7115 , A61K38/46 , A61K47/10 , A61K47/14 , A61K47/18 , A61K47/22 , A61K47/24 , A61K47/28 , A61K47/54 , A61P11/00 , C07K14/47 , C12N9/14 , C12N15/11 , C12N15/113 , C12N15/88
Abstract: 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:US20240374514A1
公开(公告)日:2024-11-14
申请号:US18778746
申请日:2024-07-19
Applicant: ReCode Therapeutics, Inc.
Inventor: Mirko Hennig , Vladimir Kharitonov , Brandon A. Wustman , Jackson Eby , Rumpa Bhattacharjee
IPC: A61K9/00 , A61K9/127 , A61K9/51 , A61K31/7105 , A61K31/7115 , A61K38/46 , A61K47/10 , A61K47/14 , A61K47/18 , A61K47/22 , A61K47/24 , A61K47/28 , A61K47/54 , A61P11/00 , C07K14/47 , C12N9/14 , C12N15/11 , C12N15/113 , C12N15/88
Abstract: 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:US20240374512A1
公开(公告)日:2024-11-14
申请号:US18778439
申请日:2024-07-19
Applicant: ReCode Therapeutics, Inc.
Inventor: Mirko Hennig , Vladimir Kharitonov , Brandon A. Wustman , Jackson Eby , Rumpa Bhattacharjee
IPC: A61K9/00 , A61K9/127 , A61K9/51 , A61K31/7105 , A61K31/7115 , A61K38/46 , A61K47/10 , A61K47/14 , A61K47/18 , A61K47/22 , A61K47/24 , A61K47/28 , A61K47/54 , A61P11/00 , C07K14/47 , C12N9/14 , C12N15/11 , C12N15/113 , C12N15/88
Abstract: 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:US20240269325A1
公开(公告)日:2024-08-15
申请号:US18431504
申请日:2024-02-02
Applicant: ReCode Therapeutics, Inc.
Inventor: Mirko Hennig , Daniella Ishimaru , David J. Lockhart , Michael Torres , Jackson Eby , Dmitri Boudko , Brandon Wustman
CPC classification number: A61K48/005 , A61K9/0078 , A61K9/12 , A61K9/1271 , A61K9/5123 , A61K38/1709 , A61K48/0075
Abstract: 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: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:US20250114477A1
公开(公告)日:2025-04-10
申请号:US18283143
申请日:2022-03-21
Applicant: ReCode Therapeutics, Inc.
Inventor: Brandon A. Wustman , David J. Lockhart , Mirko Hennig , Daniella Ishimaru , Rumpa Bhattacharjee
Abstract: 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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