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公开(公告)号:US11767548B2
公开(公告)日:2023-09-26
申请号:US17350662
申请日:2021-06-17
Applicant: ModernaTX, Inc.
Inventor: Amy E. Rabideau , Athanasios Dousis , Kanchana Ravichandran , Elissa Hobert
CPC classification number: C12P19/34 , C12N9/1247 , C12Y207/11023
Abstract: The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.
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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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公开(公告)号:US20230235298A1
公开(公告)日:2023-07-27
申请号:US17928680
申请日:2021-06-01
Applicant: ModernaTX, Inc.
Inventor: Lisa M. Rice , Patrick Finn , Athanasios Dousis
CPC classification number: C12N9/0071 , C12Y114/16001 , C12N15/63 , A61P3/00 , A61K9/1271
Abstract: Variant phenylalanine hydroxylase polypeptides having substitutions at selected amino acid residues are disclosed. Also disclosed are methods of using variant phenylalanine hydroxylase polypeptides, or polynucleotides encoding variant phenylalanine hydroxylase polypeptides, to treat disorders such as phenylketonuria.
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公开(公告)号:US10465190B1
公开(公告)日:2019-11-05
申请号:US15387263
申请日:2016-12-21
Applicant: ModernaTX, Inc.
Inventor: Jesse Chen , Athanasios Dousis
IPC: C07H21/04 , C12N15/113 , C12N9/12 , A61K31/70
Abstract: The present disclosure provides, in some aspects, in vitro transcription systems (including, for example, nucleic acid constructs and polymerases), the use of which increases transcription efficiency while reducing the amount of truncated single-stranded ribonucleic acid transcript produced during an in vitro transcription reaction.
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公开(公告)号:US12195778B2
公开(公告)日:2025-01-14
申请号:US18448856
申请日:2023-08-11
Applicant: ModernaTX, Inc.
Inventor: Amy E. Rabideau , Athanasios Dousis , Kanchana Ravichandran , Elissa Hobert
Abstract: The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.
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公开(公告)号:US12180518B2
公开(公告)日:2024-12-31
申请号:US17816696
申请日:2022-08-01
Applicant: ModernaTX, Inc.
Inventor: Athanasios Dousis , Kanchana Ravichandran , Amy E. Rabideau , Margaret Franklin , Kevin Smith , Michelle Lynn Hall
Abstract: The present disclosure provides RNA polymerase variants for high efficiency transcription.
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公开(公告)号:US20230009009A1
公开(公告)日:2023-01-12
申请号:US17275053
申请日:2019-09-12
Applicant: ModernaTX, Inc.
Inventor: Paolo G. V. Martini , Athanasios Dousis , Vladimir Presnyak , Jingsong Cao
Abstract: This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof. 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 G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.
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公开(公告)号:US10730924B2
公开(公告)日:2020-08-04
申请号:US16025302
申请日:2018-07-02
Applicant: ModernaTX, Inc.
Inventor: Barry Ticho , Nadege Briancon-Eris , Zhinan Xia , Athanasios Dousis , Seymour de Picciotto , Vladimir Presnyak , Stephen Hoge , Iain Mcfadyen , Kerry Benenato , Ellalahewage Sathyajith Kumarasinghe
IPC: C12N15/62 , A61K47/14 , C07K14/64 , A61K31/7105 , A61K47/69 , A61P9/00 , A61K38/17 , C07K16/26 , C12N15/52 , A61K9/127 , A61K38/00 , A61K9/00
Abstract: The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. 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 relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.
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公开(公告)号:US20180371047A1
公开(公告)日:2018-12-27
申请号:US16025302
申请日:2018-07-02
Applicant: ModernaTX, Inc.
Inventor: Barry Ticho , Nadege Briancon-Eris , Zhinan Xia , Athanasios Dousis , Seymour de Picciotto , Vladimir Presnyak , Stephen Hoge , Iain Mcfadyen , Kerry Benenato , Ellalahewage Sathyajith Kumarasinghe
Abstract: The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. 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 relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.
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公开(公告)号:US12103955B2
公开(公告)日:2024-10-01
申请号:US16944014
申请日:2020-07-30
Applicant: MODERNATX, INC.
Inventor: Barry Ticho , Nadege Briancon-Eris , Zhinan Xia , Athanasios Dousis , Seymour de Picciotto , Vladimir Presnyak , Stephen Hoge , Iain Mcfadyen , Kerry Benenato , Ellalahewage Sathyajith Kumarasinghe
IPC: C12N15/62 , A61K31/7105 , A61K38/17 , A61K47/14 , A61K47/69 , A61K48/00 , A61P9/00 , C07K14/64 , C07K16/26 , C12N15/52 , A61K9/00 , A61K9/127 , A61K38/00
CPC classification number: C07K14/64 , A61K31/7105 , A61K38/1796 , A61K47/6929 , A61P9/00 , C07K16/26 , C12N15/52 , C12N15/62 , A61K9/0019 , A61K9/127 , A61K38/00 , C07K2319/00 , C07K2319/31
Abstract: The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. 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 relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.
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