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公开(公告)号：US20240318187A1
公开(公告)日：2024-09-26
申请号：US18477788
申请日：2023-09-29
Applicant: ModernaTX, Inc.
Inventor： Stephen G. HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain MCFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Staci SABNIS , William BUTCHER
IPC: C12N15/67 , A61K31/7115 , A61K48/00
CPC classification number: C12N15/67 , A61K31/7115 , A61K48/0066 , C12N2800/22
Abstract: The invention related to polyribonucleotides comprising an open reading frame of linked nucleosides encoding a polypeptide of interest (e.g., a therapeutic polypeptide), isoforms thereof, functional fragments thereof, and fusion proteins comprising the polypeptide. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the invention provides sequence-optimized polyribonucleotides comprising nucleotides encoding the sequence of the polypeptide of interest, or sequence having high sequence identity with those sequence optimized polyribonucleotides.
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公开(公告)号：US20200376081A1
公开(公告)日：2020-12-03
申请号：US16842300
申请日：2020-04-07
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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3.发明申请公开(公告)号：US20190300906A1
公开(公告)日：2019-10-03
申请号：US16302341
申请日：2017-05-18
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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公开(公告)号：US20190000933A1
公开(公告)日：2019-01-03
申请号：US16110833
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：US20240024422A1
公开(公告)日：2024-01-25
申请号：US18164796
申请日：2023-02-06
Applicant: ModernaTX, Inc.
Inventor： Joshua FREDERICK , Susannah HEWITT , Ailin BAI , Stephen HOGE , Vladimir PRESNYAK , Iain MCFADYEN , Kerry BENENATO , Ellalahewage Sathyajith KUMARASINGHE
IPC: A61K38/20 , A61K9/51 , C07K14/54 , A61K31/7088 , A61K31/7115 , A61K48/00 , C12N15/62 , A61P35/00 , A61K9/00
CPC classification number: A61K38/208 , A61K9/5123 , C07K14/5434 , A61K31/7088 , A61K31/7115 , A61K48/00 , A61K48/0025 , A61K48/005 , C12N15/62 , A61P35/00 , A61K9/0019 , A61K48/0008
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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Patent Agency Ranking
公开(公告)号：US20210206818A1
公开(公告)日：2021-07-08
申请号：US16071131
申请日：2017-01-20
Applicant: ModernaTX, Inc.
Inventor： Eric Yi-Chun HUANG , Joshua P. FREDERICK , Kristine MCKINNEY , Christina HENDERSON , Kahlin CHEUNG-ONG , Joseph BOLEN , Stephen Michael KELSEY , Michael MORIN , Sushma GURUMURTHY , Kerry BENENATO , Stephen HOGE , Iain MCFADYEN , Vladimir PRESNYAK
Abstract: The invention features isolated mRNAs encoding at least one intracellular binding domain, including mRNAs comprising one or more modified nucleobase and preferably lacking an encoded scaffold polypeptide, and methods of using the same, for example, for inducing apoptosis and/or treating cancer (e.g., liver cancer or colorectal cancer).
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公开(公告)号：US20190167811A1
公开(公告)日：2019-06-06
申请号：US16092612
申请日：2017-04-13
Applicant: ModernaTX, Inc.
Inventor： Kerry BENENATO , Bo YING , Luis BRITO
Abstract: The present application provides a composition comprising (1) a lipid composition comprising an ionizable amino lipid and a quaternary amine compound and (2) a polynucleotide. The present application also provides a composition comprising (1) a lipid composition comprising an asymmetric phospholipid, an ionizable amino lipid, and optionally a quaternary amine compound and (2) a polynucleotide, wherein the composition is formulated for intratumoral delivery of the polynucleotide. The present application further provides pharmaceutical compositions for intratumoral delivery comprising (1) a lipid composition comprising a compound of formula (I) and (2) therapeutic agent or a polynucleotide encoding the therapeutic agent, e.g., an mRNA encoding a therapeutic protein or a fragment thereof. Further provided is a method of increasing retention of a polynucleotide in a tumor tissue by using such a composition.
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公开(公告)号：US20190111003A1
公开(公告)日：2019-04-18
申请号：US16227810
申请日：2018-12-20
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
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：US20190002890A1
公开(公告)日：2019-01-03
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：US20180369374A1
公开(公告)日：2018-12-27
申请号：US15996146
申请日：2018-06-01
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
CPC classification number: A61K39/39558 , A61K31/713 , A61K39/0011 , A61K39/39 , A61K45/06 , A61K2039/505 , A61K2039/53 , A61K2039/585 , A61P35/00 , C07K14/54 , C07K14/5434 , C07K14/5443 , C07K14/705 , C07K14/70503 , C07K14/70532 , C07K14/70575 , C07K14/70596 , C07K16/2818 , C07K2317/76 , C07K2319/30 , C07K2319/32 , A61K2300/00
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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