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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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2.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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公开(公告)号：US20230338410A1
公开(公告)日：2023-10-26
申请号：US18167984
申请日：2023-02-13
Applicant: ModernaTX, Inc.
Inventor： Stephen HOGE , Tirtha CHAKRABORTY , Gilles BESIN , Ruchi JAIN
IPC: A61K31/7115 , C12N15/67 , A61K9/00 , A61K9/127 , C12N15/113
CPC classification number: A61K31/7115 , A61K9/0019 , A61K9/127 , C12N15/113 , C12N15/67 , C12N2310/141 , C12N2800/107 , C12N2800/22
Abstract: The disclosure features methods of reducing or inhibiting an anti-drug antibody response in a subject, as well as methods of reducing or inhibiting unwanted immune cell activation in a subject to be treated with a messenger RNA (mRNA), comprising administering to the subject a mRNA, e.g., a chemically modified messenger RNA (mmRNA), encoding a polypeptide of interest, wherein the mRNA comprises at least one microRNA (miR) binding site for a miR expressed in immune cells, such as miR-126 binding site and/or miR-142 binding site, such that an anti-drug antibody response to the polypeptide or interest, or unwanted immune cell activation (e.g., B cell activation, cytokine secretion), is reduced or inhibited in the subject. The disclosure further provides therapeutic treatment regimens designed to reduce or inhibit ADA or unwanted immune cell activation (e.g., B cell activation, cytokine secretion) in a subject being treated with mRNA-based therapeutics.
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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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5.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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Patent Agency Ranking
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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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：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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9.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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