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公开(公告)号：US20210163928A1
公开(公告)日：2021-06-03
申请号：US17041332
申请日：2019-04-11
Applicant: ModernaTX, Inc.
Inventor： David REID , Caroline KÖHRER , Ruchi JAIN , Melissa J. MOORE , Scott DONOVAN , Aaron LARSEN , Vladimir PRESNYAK
IPC: C12N15/11 , A61K48/00 , C12Q1/6897
Abstract: The present disclosure provides messenger RNAs (mRNAs) having chemical and/or structural modifications, including RNA elements and/or modified nucleotides, in particular C-rich or CG-rich elements, which provide a desired translational regulatory activity to the mRNA.
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公开(公告)号：US20200149052A1
公开(公告)日：2020-05-14
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
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：US20200054747A1
公开(公告)日：2020-02-20
申请号：US16457300
申请日：2019-06-28
Applicant: ModernaTX, Inc.
Inventor： Joshua P. FREDERICK , Susannah HEWITT , Ailin BAI , Stephen G. HOGE , Vladimir PRESNYAK , Iain MCFADYEN , Kerry BENENATO , Ellalahewage Sathyajith KUMARASINGHE
IPC: A61K39/395 , A61K39/00 , A61K31/713 , A61K45/06 , A61K39/39 , C07K16/28 , C07K14/705 , C07K14/54
Abstract: The present disclosure relates to the use of nucleic acid (e.g., mRNA) combination therapies for the treatment of cancer. The disclosure provides compositions, and methods for their preparation, manufacture, and therapeutic use, wherein those compositions comprise at least two polynucleotides (e.g., mRNAs) in combination wherein the at least two polynucleotides are selected from the group consisting of (i) a polynucleotide encoding an immune response primer (e.g., IL23), (ii) a polynucleotide encoding an immune response co-stimulatory signal (e.g., OX40L), (iii) a polynucleotide encoding a checkpoint inhibitor (e.g., an anti CTLA-4 antibody), and, (iv) a combination thereof. The therapeutic methods disclosed herein comprise, e.g., the administration of a combination therapy disclosed herein for the treatment of cancer, e.g., by reducing the size of a tumor or inhibiting the growth of a tumor, in a subject in need thereof. In some aspects, the combination therapies disclosed herein disclosed are administered intratumorally.
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公开(公告)号：US20190125839A1
公开(公告)日：2019-05-02
申请号：US16192274
申请日：2018-11-15
Applicant: ModernaTX, Inc.
Inventor： Joshua FREDERICK , Susannah HEWITT , Ailin BAI , Stephen HOGE , Vladimir PRESNYAK , Iain MCFADYEN , Kerry BENENATO , Ellalahewage Sathyajith KUMARASINGHE
Abstract: The present disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human interleukin-12 (IL12), functional fragments thereof, and fusion proteins comprising IL12. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the disclosure provides sequence-optimized polynucleotides comprising nucleotides encoding the polypeptide sequence of human IL12, or sequences having high sequence identity with those sequence optimized polynucleotides.
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公开(公告)号：US20230323371A1
公开(公告)日：2023-10-12
申请号：US18296596
申请日：2023-04-06
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 , C12N9/2465 , C12Y302/01022 , A61P3/00 , A61K38/47 , 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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Patent Agency Ranking
公开(公告)号：US20230041964A1
公开(公告)日：2023-02-09
申请号：US17826387
申请日：2022-05-27
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
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：US20220096630A1
公开(公告)日：2022-03-31
申请号：US17202829
申请日：2021-03-16
Applicant: ModernaTX, Inc.
Inventor： Joshua P. FREDERICK , Susannah HEWITT , Ailin BAI , Stephen G. HOGE , Vladimir PRESNYAK , Iain MCFADYEN , Kerry BENENATO , Ellalahewage Sathyajith KUMARASINGHE
IPC: A61K39/395 , C07K14/54 , C07K14/705 , C07K16/28 , A61K39/39 , A61K45/06 , A61K31/713 , A61K48/00 , A61K39/00
Abstract: The present disclosure relates to the use of nucleic acid (e.g., mRNA) combination therapies for the treatment of cancer. The disclosure provides compositions, and methods for their preparation, manufacture, and therapeutic use, wherein those compositions comprise at least two polynucleotides (e.g., mRNAs) in combination wherein the at least two polynucleotides are selected from the group consisting of (i) a polynucleotide encoding an immune response primer (e.g., IL23), (ii) a polynucleotide encoding an immune response co-stimulatory signal (e.g., OX40L), (iii) a polynucleotide encoding a checkpoint inhibitor (e.g., an anti CTLA-4 antibody), and, (iv) a combination thereof. The therapeutic methods disclosed herein comprise, e.g., the administration of a combination therapy disclosed herein for the treatment of cancer, e.g., by reducing the size of a tumor or inhibiting the growth of a tumor, in a subject in need thereof. In some aspects, the combination therapies disclosed herein disclosed are administered intratumorally.
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公开(公告)号：US20210038529A1
公开(公告)日：2021-02-11
申请号：US17071685
申请日：2020-10-15
Applicant: ModernaTX, Inc.
Inventor： Joshua FREDERICK , Ailin BAI , Vladimir PRESNYAK , Stephen G. 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 , A61P35/00 , A61K39/395
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：US20200208145A1
公开(公告)日：2020-07-02
申请号：US16614245
申请日：2018-05-18
Applicant: MODERNATX, INC.
Inventor： Melissa J. MOORE , Caroline KÖHRER , Ruchi JAIN , Vladimir PRESNYAK
Abstract: The present disclosure provides messenger RNAs (mRNAs) having chemical and/or structural modifications, including RNA elements and/or modified nucleotides, which provide a desired translational regulatory activity to the mRNA.
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30.发明申请公开(公告)号：US20190298658A1
公开(公告)日：2019-10-03
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
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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