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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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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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公开(公告)号：US20180028664A1
公开(公告)日：2018-02-01
申请号：US15674872
申请日：2017-08-11
Applicant: ModernaTX, Inc.
Inventor： Gilles Besin , Stephen Hoge , Joseph Senn , Kerry Benenato , Staci Sabnis
IPC: A61K47/10 , C12N15/117 , A61K39/39 , C08G65/333 , C08G65/332 , C08G65/335 , A61K31/713 , C08G65/334
CPC classification number: A61K48/0033 , A61K9/127 , A61K31/7115 , A61K31/713 , A61K39/39 , A61K47/10 , A61K47/543 , A61K47/544 , A61K47/60 , A61K47/6929 , A61K48/0066 , A61K2039/55555 , C08G65/3322 , C08G65/3326 , C08G65/33306 , C08G65/3331 , C08G65/33317 , C08G65/33324 , C08G65/3348 , C08G65/3356 , C08G2650/04 , C12N15/117 , C12N15/88 , C12N2310/141 , C12N2310/17 , C12N2320/32
Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
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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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Patent Agency Ranking
公开(公告)号：US11969506B2
公开(公告)日：2024-04-30
申请号：US16493814
申请日：2018-03-15
Applicant: ModernaTX, Inc. , Oregon State University
Inventor： Siddharth Patel , Emily Robinson , Anna Brown , Orn Almarsson , Kerry E. Benenato , Staci Sabnis , Gaurav Sahay , Ashwani Kumar Narayana
IPC: A61K9/51 , A61K31/7088 , A61K45/06 , A61K48/00 , C12N15/113
CPC classification number: A61K9/5123 , A61K9/5146 , A61K31/7088 , A61K45/06 , A61K48/0033 , C12N15/113 , C12N2320/32
Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include an ionizable lipid, a phospholipid, a first sterol or a tocopherol, and optionally a second sterol different from the first sterol. 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.发明授权公开(公告)号：US10258698B2
公开(公告)日：2019-04-16
申请号：US14775773
申请日：2014-03-14
Applicant: ModernaTX, Inc.
Inventor： Stephen G. Hoge , Örn Almarsson , David D. Hile , Ciarán Lawlor , John Podobinski , Staci Sabnis , Antonin de Fougerolles , Divakar Ramakrishnan , Kristy M. Wood
Abstract: The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor. The formulation composition may further include a modified nucleic acid molecule and a delivery agent. The present invention further provides nucleic acids useful for encoding polypeptides capable of modulating a cell's function and/or activity.
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公开(公告)号：US20180147298A1
公开(公告)日：2018-05-31
申请号：US15674107
申请日：2017-08-10
Applicant: ModernaTX, Inc.
Inventor： Gilles Besin , Stephen Hoge , Joseph Senn , Kerry Benenato , Staci Sabnis
Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
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公开(公告)号：US12252704B2
公开(公告)日：2025-03-18
申请号：US17154309
申请日：2021-01-21
Applicant: ModernaTX, Inc.
Inventor： Paolo Martini , Stephen Hoge , Kerry Benenato , Vladimir Presnyak , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Ding An , Staci Sabnis
Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
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10.发明申请公开(公告)号：US20210269830A1
公开(公告)日：2021-09-02
申请号：US17154309
申请日：2021-01-21
Applicant: ModernaTX, Inc.
Inventor： Paolo Martini , Stephen Hoge , Kerry Benenato , Vladimir Presnyak , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Ding An , Staci Sabnis
Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
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