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公开(公告)号：US20190175517A1
公开(公告)日：2019-06-13
申请号：US16302360
申请日：2017-05-18
Applicant: ModernaTX, Inc.
Inventor： Paolo Martini , Stephen Hoge , Kerry Benenato , Vladimir Presnyak , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Jingsong Cao , Lin Tung Guey , Staci Sabnis
Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.
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公开(公告)号：US11649461B2
公开(公告)日：2023-05-16
申请号：US16570351
申请日：2019-09-13
Applicant: ModernaTX, Inc.
Inventor： Paolo Martini , Stephen G. Hoge , Kerry Benenato , Vladimir Presnyak , Iain Mcfadyen , Ellalahewage Sathyajith Kumarasinghe , Xuling Zhu , Lin Tung Guey , Staci Sabnis
CPC classification number: C12N15/67 , A61K9/5123 , A61K38/47 , A61P3/00 , C12N9/2465 , C12Y302/01022 , A61K48/00
Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
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公开(公告)号：US12239742B2
公开(公告)日：2025-03-04
申请号：US17154325
申请日：2021-01-21
Applicant: ModernaTX, Inc.
Inventor： Paolo Martini , Stephen Hoge , Kerry Benenato , Vladimir Presnyak , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Jingsong Cao , Lin Tung Guey , Staci Sabnis
Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.
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公开(公告)号：US20220265856A1
公开(公告)日：2022-08-25
申请号：US16765632
申请日：2018-11-21
Applicant: ModernaTX, Inc.
Inventor： Lei Jiang , Lin Tung Guey , Paolo G.V. Martini , Vladimir Presnyak
Abstract: This disclosure relates to mRNA therapy for the treatment of propionic acidemia (PA). mRNAs for use in the invention, when administered in vivo, encode human propionyl-CoA carboxylase alpha (PCCA) and/or human propionyl-CoA carboxylase beta (PCCB), and isoforms thereof, functional fragments thereof, and fusion proteins comprising PCCA and/or PCCB. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of propionyl-CoA carboxylase (PCC) expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity, in subjects.
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公开(公告)号：US20220071915A1
公开(公告)日：2022-03-10
申请号：US17154325
申请日：2021-01-21
Applicant: ModernaTX, Inc.
Inventor： Paolo Martini , Stephen Hoge , Kerry Benenato , Vladimir Presnyak , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Jingsong Cao , Lin Tung Guey , Staci Sabnis
Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.
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Patent Agency Ranking
公开(公告)号：US10993918B2
公开(公告)日：2021-05-04
申请号：US16302360
申请日：2017-05-18
Applicant: ModernaTX, Inc.
Inventor： Paolo Martini , Stephen Hoge , Kerry Benenato , Vladimir Presnyak , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Jingsong Cao , Lin Tung Guey , Staci Sabnis
Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.
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公开(公告)号：US10519455B2
公开(公告)日：2019-12-31
申请号：US16110309
申请日：2018-08-23
Applicant: ModernaTX, Inc.
Inventor： Paolo Martini , Stephen G. Hoge , Kerry Benenato , Vladimir Presnyak , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Xuling Zhu , Lin Tung Guey , Staci Sabnis
Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
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8.发明公开公开(公告)号：US20240024506A1
公开(公告)日：2024-01-25
申请号：US18327728
申请日：2023-06-01
Applicant: ModernaTX, Inc.
Inventor： Lei Jiang , Lin Tung Guey , Paolo G. V. Martini , Vladimir Presnyak
IPC: C12N9/00
CPC classification number: C12N9/93 , C12Y604/01003
Abstract: This disclosure relates to mRNA therapy for the treatment of propionic acidemia (PA). mRNAs for use in the invention, when administered in vivo, encode human propionyl-CoA carboxylase alpha (PCCA) and/or human propionyl-CoA carboxylase beta (PCCB), and isoforms thereof, functional fragments thereof, and fusion proteins comprising PCCA and/or PCCB. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of propionyl-CoA carboxylase (PCC) expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity, in subjects.
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9.发明申请公开(公告)号：US20220370354A1
公开(公告)日：2022-11-24
申请号：US17608691
申请日：2020-05-08
Applicant: ModernaTX, Inc.
Inventor： Tal Zaks , Kelly Lindert , Lin Tung Guey
IPC: A61K9/127 , A61K31/7115 , A61K9/00 , A61K9/51 , A61P3/00
Abstract: This disclosure relates to mRNA therapy for the treatment of methylmalonic acidemia (MMA). mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase (MUT). mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.
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10.发明申请公开(公告)号：US20200085916A1
公开(公告)日：2020-03-19
申请号：US16302339
申请日：2017-05-18
Inventor： Paolo Martini , Stephen Hoge , Kerry Benenato , Vladimir Presnyak , Lei Jiang , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Antonio Fontanellas Roma , Pedro Berraondo Lopez , Matias Antonio Avila Zaragoza , Lin Tung Guey , Staci Sabnis
Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).
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