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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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公开(公告)号:US11696892B2
公开(公告)日:2023-07-11
申请号:US16760406
申请日:2018-10-31
Applicant: ModernaTX, Inc.
Inventor: Kenny Mikael Hansson , Kerry Benenato , Maria Wågberg , Annika Pålsson , Regina Fritsche-Danielson
CPC classification number: A61K9/1272 , A61K9/0019 , A61K31/7088 , A61K38/1866 , A61P43/00 , B82Y5/00
Abstract: The disclosure relates to nanoparticles comprising a lipid component and a modified RNA encoding a VEGF-A polypeptide. Aspects of the disclosure further relate to uses of nanoparticles comprising a lipid component and a modified RNA encoding a VEGF-A polypeptide, for improving wound healing in a subject.
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公开(公告)号:US11596609B2
公开(公告)日:2023-03-07
申请号:US17930369
申请日:2022-09-07
Applicant: ModernaTX, Inc.
Inventor: Joshua Frederick , Ailin Bai , Vladimir Presnyak , Stephen Hoge , Kerry Benenato , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Susannah Hewitt
IPC: A61K9/51 , A61K48/00 , A61K38/17 , A61K38/20 , A61K9/00 , A61K39/39 , A61K45/06 , A61K9/127 , C07K14/54 , C07K14/545 , C07K14/705 , A61K31/7088 , A61K31/7115 , A61P35/00 , A61K39/395 , A61K39/00 , B82Y5/00 , C12N15/88
Abstract: The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of combinations of immunomodulatory polynucleotides (e.g., mRNAs) encoding an immune response primer polypeptide (e.g., an interleukin 23 (IL-23) polypeptide or an interleukin 36γ (IL-36-gamma) polypeptide), and an immune response co-stimulatory signal polypeptide (e.g., an OX40L polypeptide).
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公开(公告)号:US10322091B2
公开(公告)日:2019-06-18
申请号:US16219418
申请日:2018-12-13
Applicant: ModernaTX, Inc.
Inventor: Joshua Frederick , Ailin Bai , Vladimir Presnyak , Stephen Hoge , Kerry Benenato , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Susannah Hewitt
IPC: A61K48/00 , A61K9/51 , A61K38/17 , A61K38/20 , A61K9/00 , A61K39/39 , A61K45/06 , A61K9/127 , C07K14/54 , C07K14/545 , C07K14/705 , A61P35/00 , A61K39/395 , A61K39/00 , B82Y5/00 , C12N15/88
Abstract: The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of combinations of immunomodulatory polynucleotides (e.g., mRNAs) encoding an immune response primer polypeptide (e.g., an interleukin 23 (IL-23) polypeptide or an interleukin 36γ (IL-36-gamma) polypeptide), and an immune response co-stimulatory signal polypeptide (e.g., an OX40L polypeptide).
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公开(公告)号:US20190105281A1
公开(公告)日:2019-04-11
申请号:US16222155
申请日:2018-12-17
Applicant: ModernaTX, Inc.
Inventor: Joshua Frederick , Ailin Bai , Vladimir Presnyak , Stephen Hoge , Kerry Benenato , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Susannah Hewitt
IPC: A61K9/51 , A61K9/00 , C07K14/54 , A61K38/17 , A61K38/20 , C07K14/705 , A61P35/00 , A61K39/395
CPC classification number: A61K9/5123 , A61K9/00 , A61K9/0019 , A61K9/1272 , A61K9/5146 , A61K38/177 , A61K38/1774 , A61K38/20 , A61K38/2006 , A61K38/208 , A61K38/2086 , A61K39/39 , A61K39/3955 , A61K45/06 , A61K48/00 , A61K48/005 , A61K2039/505 , A61K2039/51 , A61K2039/53 , A61K2039/54 , A61K2039/545 , A61K2039/585 , A61P35/00 , B82Y5/00 , C07K14/54 , C07K14/5434 , C07K14/5443 , C07K14/5446 , C07K14/545 , C07K14/70575 , C07K2319/00 , C12N15/88 , A61K2300/00
Abstract: The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of combinations of immunomodulatory polynucleotides (e.g., mRNAs) encoding an immune response primer polypeptide (e.g., an interleukin 23 (IL-23) polypeptide or an interleukin 36γ (IL-36-gamma) polypeptide), and an immune response co-stimulatory signal polypeptide (e.g., an OX40L polypeptide).
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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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8.
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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公开(公告)号:US20220118073A9
公开(公告)日:2022-04-21
申请号:US17155592
申请日:2021-01-22
Applicant: ModernaTX, Inc.
Inventor: Giuseppe Ciaramella , Sunny Himansu , Vladimir Presnyak , Kerry Benenato , Ellalahewage Sathyajith Kumarasinghe
IPC: A61K39/12 , A61P31/14 , A61K31/7115 , C12N15/86
Abstract: The disclosure relates to Lassa virus, Nipah virus, and betacoronavirus ribonucleic acid vaccines as well as methods of using the vaccines and compositions comprising the vaccines.
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