JP7675716B2 - 3’-rnaオリゴヌクレオチドの合成 - Google Patents

3’-rnaオリゴヌクレオチドの合成 Download PDF

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JP7675716B2
JP7675716B2 JP2022530757A JP2022530757A JP7675716B2 JP 7675716 B2 JP7675716 B2 JP 7675716B2 JP 2022530757 A JP2022530757 A JP 2022530757A JP 2022530757 A JP2022530757 A JP 2022530757A JP 7675716 B2 JP7675716 B2 JP 7675716B2
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base
uracil
nucleoside
alkyl
group
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JP2023503985A5 (https=
JP2023503985A (ja
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ケー ナイル,ジャヤプラカーシュ
シー サリナス,ジュアン
フレデリック ブリオネス,ジョン
ケー シュレーゲル,マルク
マツダ,シゲオ
ヴィ ケリン,アレクサンダー
ヂャン,リーガン
エー マイアー,マーティン
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Alnylam Pharmaceuticals Inc
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7028Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/704Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P35/00Antineoplastic agents
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    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06Pyrimidine radicals
    • C07H19/067Pyrimidine radicals with ribosyl as the saccharide radical
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    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/319Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-position
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    • C12N2330/00Production
    • C12N2330/30Production chemically synthesised
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02PCLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00Technologies relating to chemical industry
    • Y02P20/50Improvements relating to the production of bulk chemicals
    • Y02P20/55Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

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  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
  • Dispersion Chemistry (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022530757A 2019-11-27 2020-11-23 3’-rnaオリゴヌクレオチドの合成 Active JP7675716B2 (ja)

Priority Applications (1)

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JP2025074104A JP2025105841A (ja) 2019-11-27 2025-04-28 3’-rnaオリゴヌクレオチドの合成

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962941153P 2019-11-27 2019-11-27
US62/941,153 2019-11-27
PCT/US2020/061755 WO2021108291A1 (en) 2019-11-27 2020-11-23 Synthesis of 3'-rna oligonucleotides

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JP2023503985A JP2023503985A (ja) 2023-02-01
JP2023503985A5 JP2023503985A5 (https=) 2023-10-03
JP7675716B2 true JP7675716B2 (ja) 2025-05-13

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JP2025074104A Pending JP2025105841A (ja) 2019-11-27 2025-04-28 3’-rnaオリゴヌクレオチドの合成

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US (1) US20250325575A2 (https=)
EP (1) EP4065715A4 (https=)
JP (2) JP7675716B2 (https=)
KR (1) KR20220107246A (https=)
CN (2) CN118791543A (https=)
AU (1) AU2020391116A1 (https=)
CA (1) CA3162717A1 (https=)
IL (2) IL293327B2 (https=)
MX (1) MX2022006221A (https=)
WO (1) WO2021108291A1 (https=)

Families Citing this family (1)

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Publication number Priority date Publication date Assignee Title
WO2026085164A2 (en) 2024-10-16 2026-04-23 Alnylam Pharmaceuticals, Inc. Snca irna formulations and methods of use thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012024776A1 (en) 2010-08-23 2012-03-01 The Royal Institution For The Advancement Of Learning/Mcgill University Block synthesis of oligoribonucleotides
US20130261026A1 (en) 2001-03-14 2013-10-03 The Regents Of The University Of Michigan Linkers and co-coupling agents for optimization of oligonucleotide synthesis and purification on solid supports

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US4924624A (en) * 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
ATE299509T1 (de) * 1992-07-23 2005-07-15 Isis Pharmaceuticals Inc Neue 2'-o-alkyl-nukleoside und -phosphoramidite, verfahren zu ihrer herstellung und ihre verwendungen
US6649750B1 (en) * 2000-01-05 2003-11-18 Isis Pharmaceuticals, Inc. Process for the preparation of oligonucleotide compounds
WO2002018405A2 (en) * 2000-09-01 2002-03-07 Ribozyme Pharmaceuticals, Incorporated Methods for synthesizing nucleosides, nucleoside derivatives and non-nucleoside derivatives
WO2003039523A2 (en) * 2001-11-05 2003-05-15 Exiqon A/S OLIGONUCLEOTIDES MODIFIED WITH NOVEL α-L-RNA ANALOGUES
CN100484949C (zh) * 2005-07-18 2009-05-06 张必良 用于rna寡核苷酸合成中的核苷亚磷酰胺及其合成方法
US8911948B2 (en) * 2008-04-30 2014-12-16 Integrated Dna Technologies, Inc. RNase H-based assays utilizing modified RNA monomers
US8309707B2 (en) * 2008-09-06 2012-11-13 Chemgenes Corporation RNA synthesis-phosphoramidites for synthetic RNA in the reverse direction, and application in convenient introduction of ligands, chromophores and modifications of synthetic RNA at the 3′-end
US8541569B2 (en) * 2008-09-06 2013-09-24 Chemgenes Corporation Phosphoramidites for synthetic RNA in the reverse direction, efficient RNA synthesis and convenient introduction of 3'-end ligands, chromophores and modifications of synthetic RNA
CA2900238A1 (en) * 2013-02-22 2014-08-28 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) molecules containing a 2' internucleoside linkage
EP3154996B1 (en) * 2014-06-10 2020-03-11 Agilent Technologies, Inc. Protecting groups for "z nucleotide" and methods thereof
EP3455232B1 (en) * 2016-05-12 2020-05-06 Roche Innovation Center Copenhagen A/S Enhanced coupling of stereodefined oxazaphospholidine phosphoramidite monomers to nucleoside or oligonucleotide
WO2018019748A1 (en) * 2016-07-27 2018-02-01 F. Hoffmann-La Roche Ag 5's-lna nucleotides and oligonucleotides

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130261026A1 (en) 2001-03-14 2013-10-03 The Regents Of The University Of Michigan Linkers and co-coupling agents for optimization of oligonucleotide synthesis and purification on solid supports
WO2012024776A1 (en) 2010-08-23 2012-03-01 The Royal Institution For The Advancement Of Learning/Mcgill University Block synthesis of oligoribonucleotides

Non-Patent Citations (6)

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Title
Bioconjugate Chemistry,2003年,Vol. 14,pp. 690-696
GREENE, Theodora W. et al.,Protective Groups in Organic Synthesis, Third Edition,1999年04月09日,pp. 123-125
Helvetica Chimica Acta,2003年,Vol. 86,pp. 2726-2740
Journal of Sciences, Islamic Republic of Iran,1990年,Vol. 1,pp. 355-360
The Journal of Organic Chemistry,1990年,Vol. 55,pp. 4717-4724
Triisopropylsilyl Chloride,Encyclopedia of Reagents for Organic Synthesis,2001年,pp. 1-5,10.1002/047084289X.rt262

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WO2021108291A1 (en) 2021-06-03
IL293327B2 (en) 2026-04-01
EP4065715A1 (en) 2022-10-05
CA3162717A1 (en) 2021-06-03
IL324170A (en) 2025-12-01
US20230021879A1 (en) 2023-01-26
EP4065715A4 (en) 2024-04-10
CN115038790B (zh) 2024-06-21
IL293327B1 (en) 2025-12-01
MX2022006221A (es) 2022-08-10
CN118791543A (zh) 2024-10-18
IL293327A (en) 2022-07-01
JP2023503985A (ja) 2023-02-01
AU2020391116A1 (en) 2022-07-14
JP2025105841A (ja) 2025-07-10
US20250325575A2 (en) 2025-10-23
KR20220107246A (ko) 2022-08-02
CN115038790A (zh) 2022-09-09

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