JP2023526975A - オリゴヌクレオチド組成物及びその方法 - Google Patents

オリゴヌクレオチド組成物及びその方法 Download PDF

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Publication number
JP2023526975A
JP2023526975A JP2022571745A JP2022571745A JP2023526975A JP 2023526975 A JP2023526975 A JP 2023526975A JP 2022571745 A JP2022571745 A JP 2022571745A JP 2022571745 A JP2022571745 A JP 2022571745A JP 2023526975 A JP2023526975 A JP 2023526975A
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optionally substituted
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optionally
groups
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JP2022571745A
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Japanese (ja)
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JPWO2021237223A5 (enExample
Inventor
カンダサミー，パチャムトゥ
クマラサミー，ジャヤカンタン
ヴァルギーズ，チャンドラ
マラッパン，スブラマニアン
レディ ボミネニ，ゴパル
護 清水
直樹 岩本
ミッシェル スタンドレー，ステファニー
リウ，ユアンジン
ジェイダ アンドレウッチ，エイミー
ルー，ゲンリャン
チバタカーン，オナノン
フセイン カーン，アクバル
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Publication of JP2023526975A publication Critical patent/JP2023526975A/ja
Publication of JPWO2021237223A5 publication Critical patent/JPWO2021237223A5/ja
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/02—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/06—Phosphorus compounds without P—C bonds
    • C07F9/16—Esters of thiophosphoric acids or thiophosphorous acids
    • C07F9/165—Esters of thiophosphoric acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H1/00—Processes for the preparation of sugar derivatives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022571745A 2020-05-22 2021-05-24 オリゴヌクレオチド組成物及びその方法 Pending JP2023526975A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063029387P 2020-05-22 2020-05-22
US63/029,387 2020-05-22
PCT/US2021/033945 WO2021237223A1 (en) 2020-05-22 2021-05-24 Oligonucleotide compositions and methods thereof

Publications (2)

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JP2023526975A true JP2023526975A (ja) 2023-06-26
JPWO2021237223A5 JPWO2021237223A5 (enExample) 2024-05-31

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JP2022571745A Pending JP2023526975A (ja) 2020-05-22 2021-05-24 オリゴヌクレオチド組成物及びその方法

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US (1) US20230203087A1 (enExample)
EP (1) EP4153604A4 (enExample)
JP (1) JP2023526975A (enExample)
AU (1) AU2021277407A1 (enExample)
CA (1) CA3176986A1 (enExample)
WO (1) WO2021237223A1 (enExample)

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CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
MX2016009290A (es) 2014-01-16 2017-02-28 Wave Life Sciences Ltd Diseño quiral.
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US11013757B2 (en) 2016-06-03 2021-05-25 Wave Life Sciences Ltd. Oligonucleotides, compositions and methods thereof
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
WO2018098264A1 (en) 2016-11-23 2018-05-31 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2018223081A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP7402696B2 (ja) 2017-06-21 2023-12-21 ウェイブ ライフ サイエンシズ リミテッド 合成のための化合物、組成物、及び方法
EP3664816A4 (en) 2017-08-08 2021-05-19 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES
AU2018333065B2 (en) 2017-09-18 2024-04-18 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
WO2019075357A1 (en) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
CA3098624A1 (en) 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
EP4623084A1 (en) 2022-11-24 2025-10-01 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CA3276262A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
AR132964A1 (es) 2023-06-16 2025-08-13 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de enfermedades neurodegenerativas
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025070241A1 (ja) * 2023-09-25 2025-04-03 藤本化学製品株式会社 化合物又はその塩、並びにその製造方法
WO2025072886A1 (en) * 2023-09-28 2025-04-03 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2025096809A1 (en) * 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
WO2025151895A1 (en) * 2024-01-12 2025-07-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease

Citations (3)

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WO2016028187A1 (en) * 2014-08-22 2016-02-25 Noogen Llc Modified oligonucleotides and methods for their synthesis
WO2019200185A1 (en) * 2018-04-12 2019-10-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019217784A1 (en) * 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof

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US5216168A (en) * 1992-04-01 1993-06-01 G. D. Searle & Co. 2- and 3- amino and azido derivatives of 1,5-iminosugars
WO2011005860A2 (en) * 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
IL263044B2 (en) * 2016-05-24 2024-06-01 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
WO2016028187A1 (en) * 2014-08-22 2016-02-25 Noogen Llc Modified oligonucleotides and methods for their synthesis
WO2019200185A1 (en) * 2018-04-12 2019-10-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019217784A1 (en) * 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof

Also Published As

Publication number Publication date
US20230203087A1 (en) 2023-06-29
WO2021237223A1 (en) 2021-11-25
AU2021277407A1 (en) 2022-11-17
EP4153604A4 (en) 2024-11-27
EP4153604A1 (en) 2023-03-29
CA3176986A1 (en) 2021-11-25

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