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1.发明公开公开(公告)号：US20240226025A1
公开(公告)日：2024-07-11
申请号：US18282676
申请日：2022-03-24
Applicant: ModernaTX, Inc.
Inventor： Kerry Benenato , Mark Cornebise , Edward Hennessy , Ruchi Jain , Vladimir Presnyak
CPC classification number: A61K9/5123 , A61K38/52 , A61P3/00 , C12N9/90 , C12Y504/99002
Abstract: This disclosure relates to messenger RNA (mRNA) therapy for the treatment of methylmalonic acidemia. mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase. mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.
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公开(公告)号：US20240189449A1
公开(公告)日：2024-06-13
申请号：US18282647
申请日：2022-03-24
Applicant: ModernaTX, Inc.
Inventor： Kerry Benenato , Alicia Anne Bicknell , Mark Cornebise , Athanasios Dousis , Andrew Jacob Giessel , Edward Hennessy , Ruchi Jain , Caroline Köhrer , Stuart Spencer Licht , Kanchana Ravichandran , David Reid
CPC classification number: A61K48/005 , A61K9/1271 , A61K9/5123 , A61K31/7105 , A61K48/0041 , A61P7/00 , C12N9/1018 , C12N15/67 , C12Y201/03003
Abstract: This disclosure relates to mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC). mRNA therapies of the disclosure increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the disclosure further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.
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3.发明公开公开(公告)号：US20240216288A1
公开(公告)日：2024-07-04
申请号：US18282640
申请日：2022-03-24
Applicant: ModernaTX, Inc.
Inventor： Kerry Benenato , Mark Cornebise , Edward Hennessy
CPC classification number: A61K9/5123 , A61K48/0041 , C12N9/93 , C12N15/88 , C12Y604/01003
Abstract: This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding propionyl-CoA carboxylase alpha (PCCA) and propionyl-CoA carboxylase beta (PCCB). Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of PCCA and/or PCCB expression and/or activity in subjects and are useful for the treatment of propionic academia.
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4.发明授权公开(公告)号：US11597698B2
公开(公告)日：2023-03-07
申请号：US17324426
申请日：2021-05-19
Applicant: ModernaTX, Inc.
Inventor： Kerry E. Benenato , Mark Cornebise , Edward Hennessy , Ellalahewage S. Kumarasinghe
IPC: C07C229/12 , A61K47/00 , A61K9/00 , C07C229/24 , A61K47/54 , A61K9/127 , A61K31/713 , A61K48/00
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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Patent Agency Ranking
5.发明公开公开(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：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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7.发明公开公开(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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8.发明公开公开(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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9.发明授权公开(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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