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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
Applicant: ReCode Therapeutics, Inc.
Inventor: 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 classification number: A61K9/145 , A61K9/0078 , A61K47/24 , A61K47/26 , A61K47/28 , A61K48/0008 , C07K14/705
Abstract: 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:US12257318B2
公开(公告)日:2025-03-25
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 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: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: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: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: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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公开(公告)号:US20250032424A1
公开(公告)日:2025-01-30
申请号:US18886134
申请日:2024-09-16
Applicant: ReCode Therapeutics, Inc.
Inventor: 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
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:US12133923B2
公开(公告)日:2024-11-05
申请号:US18413653
申请日:2024-01-16
Applicant: ReCode Therapeutics, Inc.
Inventor: 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
IPC: A61K9/51 , A61K8/86 , A61K9/00 , A61K31/7105 , A61K47/14 , A61K47/18 , A61K47/22 , A61K47/24 , A61K47/28 , A61K48/00 , C07C69/708 , C07C223/02 , C07C321/14 , C07D317/32
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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