JP7472018B2 - オリゴヌクレオチド調製のための技術 - Google Patents

オリゴヌクレオチド調製のための技術 Download PDF

Info

Publication number
JP7472018B2
JP7472018B2 JP2020515190A JP2020515190A JP7472018B2 JP 7472018 B2 JP7472018 B2 JP 7472018B2 JP 2020515190 A JP2020515190 A JP 2020515190A JP 2020515190 A JP2020515190 A JP 2020515190A JP 7472018 B2 JP7472018 B2 JP 7472018B2
Authority
JP
Japan
Prior art keywords
independently
ring
phosphorus
nitrogen
optionally substituted
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2020515190A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021500311A (ja
JP2021500311A5 (enExample
Inventor
アンドリュー ボウマン，キース
バーギーズ，チャンドラ
チャールズ ドネル バトラー，デイヴィッド
カンダサミ，パチャムトゥ
ロウション アラム，モハメド
護 清水
ミッシェル スタンドレー，ステファニー
アデュダ，ヴィンセント
レッディ ボミネニ，ゴーパール
トゥリパーティー，スネーラタ
コーボウ，イリア
Original Assignee
ウェーブ ライフ サイエンシーズ リミテッド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ウェーブ ライフ サイエンシーズ リミテッド filed Critical ウェーブ ライフ サイエンシーズ リミテッド
Publication of JP2021500311A publication Critical patent/JP2021500311A/ja
Publication of JP2021500311A5 publication Critical patent/JP2021500311A5/ja
Priority to JP2024063134A priority Critical patent/JP2024105256A/ja
Application granted granted Critical
Publication of JP7472018B2 publication Critical patent/JP7472018B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • C07H21/02—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 ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
JP2020515190A 2017-09-18 2018-09-17 オリゴヌクレオチド調製のための技術 Active JP7472018B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024063134A JP2024105256A (ja) 2017-09-18 2024-04-10 オリゴヌクレオチド調製のための技術

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762560169P 2017-09-18 2017-09-18
US62/560,169 2017-09-18
PCT/US2018/051398 WO2019055951A1 (en) 2017-09-18 2018-09-17 OLIGONUCLEOTIDE PREPARATION TECHNOLOGIES

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024063134A Division JP2024105256A (ja) 2017-09-18 2024-04-10 オリゴヌクレオチド調製のための技術

Publications (3)

Publication Number Publication Date
JP2021500311A JP2021500311A (ja) 2021-01-07
JP2021500311A5 JP2021500311A5 (enExample) 2021-11-25
JP7472018B2 true JP7472018B2 (ja) 2024-04-22

Family

ID=65723422

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020515190A Active JP7472018B2 (ja) 2017-09-18 2018-09-17 オリゴヌクレオチド調製のための技術
JP2024063134A Pending JP2024105256A (ja) 2017-09-18 2024-04-10 オリゴヌクレオチド調製のための技術

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024063134A Pending JP2024105256A (ja) 2017-09-18 2024-04-10 オリゴヌクレオチド調製のための技術

Country Status (9)

Country Link
US (3) US11608355B2 (enExample)
EP (1) EP3684755A4 (enExample)
JP (2) JP7472018B2 (enExample)
KR (2) KR20250123934A (enExample)
CN (1) CN111108096A (enExample)
AU (2) AU2018333065B2 (enExample)
CA (1) CA3072110A1 (enExample)
SG (1) SG11202000276YA (enExample)
WO (1) WO2019055951A1 (enExample)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
CN108779132B (zh) 2016-03-13 2022-04-15 波涛生命科学有限公司 用于亚磷酰胺和寡核苷酸合成的组合物和方法
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
JP2019520339A (ja) 2016-06-03 2019-07-18 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド、その組成物および方法
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
US11873316B2 (en) 2016-11-23 2024-01-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
JP2020522510A (ja) 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
CA3065523A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
EP3664816A4 (en) 2017-08-08 2021-05-19 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES
CA3072110A1 (en) 2017-09-18 2019-03-21 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
SG11202009877XA (en) 2018-04-12 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
US12391942B2 (en) 2018-05-11 2025-08-19 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
JP2022525541A (ja) 2019-03-20 2022-05-17 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド調製に有用な技術
MX2022014606A (es) 2020-05-22 2023-03-08 Wave Life Sciences Ltd Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas.
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
JP2025524566A (ja) 2022-07-15 2025-07-30 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ Ａｄａｒ媒介ｒｎａ編集のためのオリゴヌクレオチドおよびその使用
US20260027237A1 (en) 2022-07-15 2026-01-29 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
WO2024110565A1 (en) 2022-11-24 2024-05-30 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
AU2023390632A1 (en) 2022-12-09 2025-07-10 Proqr Therapeutics Ii B.V. 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
EP4689110A1 (en) 2023-03-24 2026-02-11 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
EP4688143A1 (en) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of liver disease
TW202516003A (zh) 2023-06-16 2025-04-16 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療神經退化性疾病之反義寡核苷酸
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
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
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
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
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026022136A1 (en) 2024-07-23 2026-01-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2026050243A1 (en) 2024-08-26 2026-03-05 Korro Bio, Inc. Galnac conjugated oligonucleotides for rna editing
WO2026060374A2 (en) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
JP2015528002A (ja) 2012-07-13 2015-09-24 ウェイブ ライフ サイエンス プライベート リミテッド キラル制御
WO2017160741A1 (en) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis

Family Cites Families (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5049656A (en) * 1988-12-21 1991-09-17 Board Of Regents Of The University Of Nebraska Sequential peptide and oligonucleotide syntheses using immunoaffinity techniques
US5210264A (en) 1992-01-10 1993-05-11 Isis Pharmaceuticals, Inc. S-(2,4-dichlorobenzyl)-β-cyanoethyl phosphorothioate diester
WO1998008809A1 (en) 1996-08-30 1998-03-05 Hybridon, Inc. Novel sulfur transfer reagents for oligonucleotide synthesis
JP4348044B2 (ja) 2002-02-12 2009-10-21 株式会社キラルジェン 立体規則性の高いジヌクレオシドホスホロチオエートの製造法
US8101348B2 (en) 2002-07-10 2012-01-24 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. RNA-interference by single-stranded RNA molecules
AU2003261449A1 (en) 2002-08-07 2004-02-25 Compositions for rna interference and methods of use thereof
JP2005089441A (ja) 2003-08-08 2005-04-07 Toudai Tlo Ltd 立体規則性の高いリン原子修飾ヌクレオチド類縁体の製造法
JP4580870B2 (ja) 2003-09-02 2010-11-17 株式会社キラルジェン リボヌクレオチド又はリボヌクレオチド誘導体の製造方法
JP4616175B2 (ja) 2003-09-02 2011-01-19 株式会社キラルジェン ５’−ホスフィチル化モノマーおよびｈ−ホスホネートオリゴヌクレオチド誘導体の製造方法
WO2005070859A1 (ja) 2004-01-27 2005-08-04 Takeshi Wada フルオラス担体およびそれを用いたオリゴヌクレオチド誘導体の製造方法
WO2005085272A1 (ja) 2004-03-05 2005-09-15 Takeshi Wada ボラノホスフェートモノマーおよびそれを用いたオリゴヌクレオチド誘導体の製造方法
WO2005092909A1 (ja) 2004-03-25 2005-10-06 Toudai Tlo, Ltd. 立体規則性の高いリボヌクレオチド類縁体及びデオキシリボヌクレオチド類縁体の製造法
WO2007102499A1 (ja) 2006-03-06 2007-09-13 Japan Tobacco Inc. ４－オキソキノリン化合物の製造方法
CN102282155B (zh) 2008-12-02 2017-06-09 日本波涛生命科学公司 磷原子修饰的核酸的合成方法
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
ES2586683T3 (es) 2009-09-16 2016-10-18 Wave Life Sciences Japan, Inc. Grupo protector novedoso para sintetizar ARN y derivados del mismo
JP5847700B2 (ja) 2010-03-05 2016-01-27 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ リボヌクレオシドホスホロチオエートの製造方法
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
JP6093924B2 (ja) 2010-11-30 2017-03-15 株式会社Wave Life Sciences Japan ２’−ｏ−修飾ｒｎａ
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
CA2879066C (en) 2012-07-13 2019-08-13 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
CN108713061B (zh) * 2016-03-09 2022-04-05 学校法人东京理科大学 Rna杂交形成用核酸低聚物
CN109462995A (zh) 2016-05-04 2019-03-12 波涛生命科学有限公司 生物活性剂的方法和组合物
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
JP2019520339A (ja) 2016-06-03 2019-07-18 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド、その組成物および方法
WO2018022473A1 (en) 2016-07-25 2018-02-01 Wave Life Sciences Ltd. Phasing
US11873316B2 (en) 2016-11-23 2024-01-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
CA3065523A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN120330183A (zh) 2017-06-02 2025-07-18 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
JP2020522510A (ja) 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
WO2019002237A1 (en) 2017-06-28 2019-01-03 Roche Innovation Center Copenhagen A/S METHOD OF MULTIPLE COUPLING AND OXIDATION
EP3664816A4 (en) 2017-08-08 2021-05-19 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES
CA3072110A1 (en) 2017-09-18 2019-03-21 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
SG11202009877XA (en) 2018-04-12 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
US12391942B2 (en) 2018-05-11 2025-08-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP2022513719A (ja) 2018-12-06 2022-02-09 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその方法
CN113423385A (zh) 2019-02-01 2021-09-21 波涛生命科学有限公司 寡核苷酸组合物及其方法
JP2022525541A (ja) 2019-03-20 2022-05-17 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド調製に有用な技術
WO2020196662A1 (ja) 2019-03-25 2020-10-01 国立大学法人東京医科歯科大学 二本鎖核酸複合体及びその使用
SG11202111387YA (en) 2019-04-25 2021-11-29 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
BR112021021203A2 (pt) 2019-04-25 2022-01-04 Wave Life Sciences Ltd Oligonucleotídeo; composição de oligonucleotídeo quiralmente controlada; composição farmacêutica; método para prevenir, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a e/ou prevenir, atrasar o início, desenvolvimento e/ou progresso, e/ou tratar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para aumentar o nível de salto de um éxon prejudicial em uma transcrição de gene ush2a ou seu produto de gene em uma célula; método para prevenir, atrasar o início ou progressão de, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para saltar um éxon prejudicial 13 em um alelo de ush2a em um indivíduo; método para produzir ou aumentar o nível de um éxon de proteína ush2a com salto 13 em um sistema; e composto, oligonucleotídeo, composição ou método
EP3965780A4 (en) 2019-05-09 2023-10-25 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
JP7766589B2 (ja) 2019-10-06 2025-11-10 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
JP2022550915A (ja) 2019-10-06 2022-12-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
JP2023515862A (ja) 2020-03-01 2023-04-14 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその方法
AU2021277407A1 (en) 2020-05-22 2022-11-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
MX2022014606A (es) 2020-05-22 2023-03-08 Wave Life Sciences Ltd Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas.
WO2022046667A1 (en) 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cells and non-human animals engineered to express adar1 and uses thereof
WO2022046723A1 (en) 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Immunoassays for detecting wild type huntingtin protein and methods of treatment employing such immunoassays
WO2022067023A1 (en) 2020-09-24 2022-03-31 Mayo Foundation For Medical Education And Research Screening platforms
CN116507724A (zh) 2020-11-08 2023-07-28 波涛生命科学有限公司 寡核苷酸组合物及其方法
KR20230118716A (ko) 2020-11-08 2023-08-11 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법
EP4396354A4 (en) 2021-09-26 2025-12-03 Wave Life Sciences Ltd OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES
EP4404942A2 (en) 2021-09-26 2024-07-31 Wave Life Sciences Ltd. Compositions for editing mecp2 transcripts and methods thereof
CA3236136A1 (en) 2021-10-27 2023-05-04 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
EP4422645A4 (en) 2021-10-27 2025-12-03 Wave Life Sciences Ltd OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODS OF USE
EP4476202A1 (en) 2022-02-11 2024-12-18 Wave Life Sciences Ltd. Stereoselective technologies for chiral compounds
AU2023227808A1 (en) 2022-03-02 2024-09-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof for exon skipping
EP4507700A2 (en) 2022-04-15 2025-02-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods relating thereto
US20250302995A1 (en) 2022-05-12 2025-10-02 Chikdu Shakti Shivalila Oligonucleotide compositions and methods thereof
AU2023321711A1 (en) 2022-08-11 2025-02-20 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
JP2015523316A (ja) 2012-07-13 2015-08-13 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
JP2015528002A (ja) 2012-07-13 2015-09-24 ウェイブ ライフ サイエンス プライベート リミテッド キラル制御
WO2017160741A1 (en) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Nucleic Acids Res.，1997年，25，3590-3593

Also Published As

Publication number Publication date
KR20200052369A (ko) 2020-05-14
KR20250123934A (ko) 2025-08-18
AU2018333065A1 (en) 2020-01-30
US11608355B2 (en) 2023-03-21
US20230348524A1 (en) 2023-11-02
AU2018333065B2 (en) 2024-04-18
CN111108096A (zh) 2020-05-05
AU2024204904A1 (en) 2024-08-01
US20200231620A1 (en) 2020-07-23
JP2024105256A (ja) 2024-08-06
SG11202000276YA (en) 2020-04-29
US20260008806A1 (en) 2026-01-08
JP2021500311A (ja) 2021-01-07
US12435105B2 (en) 2025-10-07
EP3684755A1 (en) 2020-07-29
CA3072110A1 (en) 2019-03-21
WO2019055951A1 (en) 2019-03-21
EP3684755A4 (en) 2022-03-02

Similar Documents

Publication Publication Date Title
JP7472018B2 (ja) オリゴヌクレオチド調製のための技術
AU2026202225A1 (en) Technologies useful for oligonucleotide preparation
US12428442B2 (en) Compounds, compositions and methods for synthesis
CN103068834B (zh) 用于反向合成rna的亚磷酰胺
BR112020020670A2 (pt) composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo
CN110088113A (zh) 用于亚磷酰胺和寡核苷酸合成的组合物和方法
TW202237847A (zh) 聚－𠰌啉代寡核苷酸缺口體
KR20240166519A (ko) 치료제의 표적화된 전달을 위한 비사이클릭 헤테로사이클 및 이의 리간드
TW202237848A (zh) 靶向ｔａｕ之寡核苷酸缺口體
HK40019008A (en) Technologies for oligonucleotide preparation
BR112021018492B1 (pt) Método para preparar um oligonucleotídeo e oligonucleotídeo

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20201111

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210917

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210917

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211008

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221011

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221226

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230411

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230711

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230925

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240111

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20240312

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20240410

R150 Certificate of patent or registration of utility model

Ref document number: 7472018

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150