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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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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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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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Patent Agency Ranking
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：US20190000932A1
公开(公告)日：2019-01-03
申请号：US16110788
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：US20230346928A1
公开(公告)日：2023-11-02
申请号：US18190453
申请日：2023-03-27
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
CPC classification number: A61K39/39558 , C07K14/54 , C07K14/5434 , C07K14/5443 , C07K14/705 , C07K14/70503 , C07K14/70532 , C07K14/70575 , C07K14/70596 , C07K16/2818 , A61K39/39 , A61K45/06 , A61K31/713 , A61K48/005 , A61K39/0011 , A61K2039/505 , A61K2039/53 , C07K2317/76 , C07K2319/30 , C07K2319/32 , A61K2039/585 , A61K2039/51 , A61P35/00
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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19.发明申请公开(公告)号：US20230112986A1
公开(公告)日：2023-04-13
申请号：US17813984
申请日：2022-07-21
Inventor： Paolo MARTINI , Stephen HOGE , Kerry BENENATO , Vladimir PRESNVAK , Lei JIANG , Iain MCFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Antonio FONTANELLAS ROMA , Pedro BERRAONDO LOPEZ , Matias Antonio AVILA ZARAGOZA , Lin Tung GUEY , Staci SABNIS
Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).
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公开(公告)号：US20210299221A1
公开(公告)日：2021-09-30
申请号：US17308686
申请日：2021-05-05
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
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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