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公开(公告)号：US12246030B2
公开(公告)日：2025-03-11
申请号：US18167984
申请日：2023-02-13
Applicant: ModernaTX, Inc.
Inventor： Stephen Hoge , Tirtha Chakraborty , Gilles Besin , Ruchi Jain
IPC: C07H21/04 , A61K9/00 , A61K9/127 , A61K31/7115 , C12N15/113 , C12N15/67
Abstract: The disclosure features methods of reducing or inhibiting an anti-drug antibody response in a subject, as well as methods of reducing or inhibiting unwanted immune cell activation in a subject to be treated with a messenger RNA (mRNA), comprising administering to the subject a mRNA, e.g., a chemically modified messenger RNA (mmRNA), encoding a polypeptide of interest, wherein the mRNA comprises at least one microRNA (miR) binding site for a miR expressed in immune cells, such as miR-126 binding site and/or miR-142 binding site, such that an anti-drug antibody response to the polypeptide or interest, or unwanted immune cell activation (e.g., B cell activation, cytokine secretion), is reduced or inhibited in the subject. The disclosure further provides therapeutic treatment regimens designed to reduce or inhibit ADA or unwanted immune cell activation (e.g., B cell activation, cytokine secretion) in a subject being treated with mRNA-based therapeutics.
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公开(公告)号：US20230285538A1
公开(公告)日：2023-09-14
申请号：US18318689
申请日：2023-05-16
Applicant: ModernaTX, Inc.
Inventor： Stephen Hoge , Giuseppe Giaramella
IPC: A61K39/145 , A61K9/00 , A61K9/127 , C12N15/113 , G01N33/50
CPC classification number: A61K39/145 , A61K9/0019 , A61K9/127 , C12N15/1131 , G01N33/505 , C12N2320/30
Abstract: High quality vaccines are identified and formulated based on a threshold differential T-cell activation potential. The vaccines are mRNA vaccines formulated in a carrier, wherein the mRNA encodes an antigen.
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公开(公告)号：US20220096522A1
公开(公告)日：2022-03-31
申请号：US17523060
申请日：2021-11-10
Applicant: ModernaTX, Inc.
Inventor： Stephen Hoge , William Issa , Edward J. Miracco , Jennifer Nelson , Amy E. Rabideau , Gabor Butora
IPC: A61K31/7105 , C07K14/505 , C12P19/34 , A61P33/00 , A61P31/12 , A61P31/04 , A61P35/00 , C12N15/10 , G01N33/68
Abstract: The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by inproved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.
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公开(公告)号：US20220096521A1
公开(公告)日：2022-03-31
申请号：US17523034
申请日：2021-11-10
Applicant: ModernaTX, Inc.
Inventor： Stephen Hoge , William Issa , Edward J. Miracco , Jennifer Nelson , Amy E. Rabideau , Gabor Butora
IPC: A61K31/7105 , C07K14/505 , C12P19/34 , A61P33/00 , A61P31/12 , A61P31/04 , A61P35/00 , C12N15/10 , G01N33/68
Abstract: The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by improved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.
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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：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：US12246029B2
公开(公告)日：2025-03-11
申请号：US17523034
申请日：2021-11-10
Applicant: ModernaTX, Inc.
Inventor： Stephen Hoge , William Issa , Edward J. Miracco , Jennifer Nelson , Amy E. Rabideau , Gabor Butora
IPC: A61K31/7105 , A61P31/04 , A61P31/12 , A61P33/00 , A61P35/00 , C07K14/505 , C12N15/10 , C12P19/34 , G01N33/68
Abstract: The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by improved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.
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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：US11801227B2
公开(公告)日：2023-10-31
申请号：US16302370
申请日：2017-05-18
Applicant: ModernaTX, Inc.
Inventor： Kerry Benenato , Stephen Hoge , Iain McFadyen , Vladimir Presnyak , Paolo Martini , Ellalahewage Sathyajith Kumarasinghe
IPC: A61K38/18 , A61K9/51 , A61K9/00 , A61K9/127 , C07K14/705 , C07K14/47 , A61P11/00 , A61K38/17 , C12N15/88
CPC classification number: A61K9/5123 , A61K9/0043 , A61K9/0053 , A61K9/0073 , A61K38/177 , A61P11/00 , C07K14/47 , C12N15/88 , A61K9/1271
Abstract: The invention relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR), isoforms thereof, functional fragments thereof, and fusion proteins comprising CFTR. 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 CFTR expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient CFTR activity in subjects.
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