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11.
公开(公告)号:US20240207374A1
公开(公告)日:2024-06-27
申请号:US18282643
申请日:2022-03-24
Applicant: ModernaTX, Inc.
Inventor: Kerry Benenato , Mark Cornebise , Edward Hennessy
CPC classification number: A61K38/465 , A61K9/5123 , A61K47/183 , A61K48/0033
Abstract: This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding glucose-6-phosphatase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of glucose-6-phosphatase expression and/or activity in subjects and are useful for the treatment of glycogen storage disease type 1a (GSD-Ia). Lipid nanoparticle/mRNA therapies of the invention increase glucose production and reduce the abnormal accumulation of glycogen and glucose-6-phosphate associated with GSD-Ia.
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公开(公告)号:US20230285310A1
公开(公告)日:2023-09-14
申请号:US18040485
申请日:2021-08-06
Applicant: ModernaTX, Inc.
Inventor: Ana Cadete Pires , Jean C. Sung , Mark Cornebise , Jeffrey Hrkach
CPC classification number: A61K9/5123 , A61K9/5146 , A61K47/28 , C07J41/0005 , C07J41/0055 , C07J41/0088 , C07J43/003
Abstract: The present disclosure provides LNPs comprising payload molecules, e.g., mRNA therapeutics, for the treatment of diseases or disorders, which would benefit from delivery of payload molecules to airway cells.
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公开(公告)号:US20170233332A1
公开(公告)日:2017-08-17
申请号:US15498698
申请日:2017-04-27
Applicant: MODERNATX, Inc.
IPC: C07C229/12 , C07C227/16 , A61K38/18 , A61K9/16
CPC classification number: C07C229/12 , A61K9/0019 , A61K9/0043 , A61K9/0073 , A61K9/127 , A61K9/1271 , A61K9/1272 , A61K9/1617 , A61K9/1641 , A61K9/5123 , A61K9/5146 , A61K31/7105 , A61K38/1725 , A61K38/1816 , A61K47/543 , A61K47/6911 , A61K48/00 , A61K48/0033 , A61K48/005 , C07C227/16 , C07C227/18 , C07C229/16 , C07C233/36 , C07C235/10 , C07C255/24 , C07C271/20 , C07C275/14 , C07C279/12 , C07C279/24 , C07C279/28 , C07C279/32 , C07C311/05 , C07C335/08 , C07C2601/02 , C07C2601/04 , C07C2601/14 , C07C2601/18 , C07D207/27 , C07D233/72 , C07D249/04 , C07D263/20 , C07D265/33 , C07D271/06 , C07D271/10 , C07D277/38 , C07F9/091 , C07K14/505
Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
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公开(公告)号:US20170224844A1
公开(公告)日:2017-08-10
申请号:US15493281
申请日:2017-04-21
Applicant: MODERNATX, Inc.
IPC: A61K48/00 , C07C227/18 , A61K38/18 , A61K31/7105 , A61K38/17 , C07C229/16 , A61K9/51
CPC classification number: C07C229/12 , A61K9/0019 , A61K9/0043 , A61K9/0073 , A61K9/127 , A61K9/1271 , A61K9/1272 , A61K9/1617 , A61K9/1641 , A61K9/5123 , A61K9/5146 , A61K31/7105 , A61K38/1725 , A61K38/1816 , A61K47/543 , A61K47/6911 , A61K48/00 , A61K48/0033 , A61K48/005 , C07C227/16 , C07C227/18 , C07C229/16 , C07C233/36 , C07C235/10 , C07C255/24 , C07C271/20 , C07C275/14 , C07C279/12 , C07C279/24 , C07C279/28 , C07C279/32 , C07C311/05 , C07C335/08 , C07C2601/02 , C07C2601/04 , C07C2601/14 , C07C2601/18 , C07D207/27 , C07D233/72 , C07D249/04 , C07D263/20 , C07D265/33 , C07D271/06 , C07D271/10 , C07D277/38 , C07F9/091 , C07K14/505
Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
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公开(公告)号:US12263248B2
公开(公告)日:2025-04-01
申请号:US17278040
申请日:2019-09-19
Applicant: ModernaTX, Inc.
Inventor: Kerry E. Benenato , Mark Cornebise , Edward Hennessy
IPC: A61K9/127 , A61K31/167 , A61K31/519 , A61K31/573 , A61K31/7105 , A61K45/06 , A61K47/10 , A61K47/24 , A61K47/28 , C07C215/08 , C07C229/12 , C07C229/14 , C07C229/16 , C07C233/47 , C07C235/10 , C07C275/14 , C07C279/28 , C07C279/36 , C07C311/05 , C07C311/14 , C07C311/64 , C07C335/08 , C07D209/48 , C07D233/61 , C07D239/47 , C07D239/54 , C07D249/04 , C07D295/13 , C07D473/18 , C07D473/34 , C07F9/09 , A61K48/00
Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
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16.
公开(公告)号:US20240207444A1
公开(公告)日:2024-06-27
申请号:US18282639
申请日:2022-03-24
Applicant: ModernaTX, Inc.
Inventor: Kerry Benenato , Mark Cornebise , Edward Hennessy
CPC classification number: A61K48/005 , A61K9/1271 , A61K9/5123 , A61K48/0033 , A61K48/0075 , C12N9/0071 , C12Y114/16001
Abstract: This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding phenylalanine hydroxylase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of phenylalanine hydroxylase expression and/or activity in subjects and are useful for the treatment of phenylketonuria
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17.
公开(公告)号:US20230286903A1
公开(公告)日:2023-09-14
申请号:US18164136
申请日:2023-02-03
Applicant: ModernaTX, Inc.
Inventor: Kerry E. BENENATO , Mark Cornebise , Edward Hennessy , Ellalahewage S. Kumarasinghe
IPC: C07C229/24 , A61K47/54 , A61K9/127 , A61K31/713 , A61K48/00
CPC classification number: C07C229/24 , A61K47/543 , A61K9/1272 , A61K31/713 , A61K48/0033 , A61K48/005
Abstract: The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
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公开(公告)号:US11220476B2
公开(公告)日:2022-01-11
申请号:US16555045
申请日:2019-08-29
Applicant: ModernaTX, Inc.
Inventor: Kerry E. Benenato , Mark Cornebise
IPC: C07C229/12 , C07C271/20 , C07C279/12 , C07C279/28 , C07C279/32 , C07D233/72 , C07D249/04 , C07D263/20 , C07D265/33 , C07D271/06 , C07D271/10 , C07D207/27 , C07D277/38 , C07C275/14 , C07C279/24 , C07C311/05 , C07C229/16 , C07C335/08 , C07C233/36 , C07C235/10 , C07C255/24 , C07F9/09 , A61K47/54 , A61K47/69 , A61K9/00 , A61K9/127 , C07C233/72 , C07C263/20 , A61K9/16 , A61K38/18 , A61K48/00 , C07K14/505 , C07C227/16 , A61K9/51 , A61K31/7105 , A61K38/17 , C07C227/18
Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
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19.
公开(公告)号:US11066355B2
公开(公告)日:2021-07-20
申请号:US17025779
申请日:2020-09-18
Applicant: ModernaTX, Inc.
Inventor: Kerry E. Benenato , Mark Cornebise , Edward Hennessy , Ellalahewage S. Kumarasinghe
IPC: C07C229/00 , A61K47/00 , A61K9/00 , C07C229/24 , A61K48/00 , A61K31/713 , A61K9/127 , A61K47/54
Abstract: The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
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公开(公告)号:US20170210697A1
公开(公告)日:2017-07-27
申请号:US15476253
申请日:2017-03-31
Applicant: MODERNATX, Inc.
IPC: C07C229/12 , C07K14/505 , A61K38/18 , A61K9/16 , A61K48/00
CPC classification number: C07C229/12 , A61K9/0019 , A61K9/0043 , A61K9/0073 , A61K9/127 , A61K9/1271 , A61K9/1272 , A61K9/1617 , A61K9/1641 , A61K9/5123 , A61K9/5146 , A61K31/7105 , A61K38/1725 , A61K38/1816 , A61K47/543 , A61K47/6911 , A61K48/00 , A61K48/0033 , A61K48/005 , C07C227/16 , C07C227/18 , C07C229/16 , C07C233/36 , C07C235/10 , C07C255/24 , C07C271/20 , C07C275/14 , C07C279/12 , C07C279/24 , C07C279/28 , C07C279/32 , C07C311/05 , C07C335/08 , C07C2601/02 , C07C2601/04 , C07C2601/14 , C07C2601/18 , C07D207/27 , C07D233/72 , C07D249/04 , C07D263/20 , C07D265/33 , C07D271/06 , C07D271/10 , C07D277/38 , C07F9/091 , C07K14/505
Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
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