CN114585737A - 寡核苷酸组合物及其使用方法 - Google Patents

寡核苷酸组合物及其使用方法 Download PDF

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Publication number
CN114585737A
CN114585737A CN202080069504.2A CN202080069504A CN114585737A CN 114585737 A CN114585737 A CN 114585737A CN 202080069504 A CN202080069504 A CN 202080069504A CN 114585737 A CN114585737 A CN 114585737A
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China
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oligonucleotide
oligonucleotides
nucleic acid
target
composition
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Pending
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CN202080069504.2A
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Chinese (zh)
Inventor
普拉尚·莫尼安
奇克杜·沙克蒂·希瓦利拉
苏布拉马尼安·马拉潘
钱德拉·瓦尔格赛
帕查穆图·坎德萨米
陆根良
于辉
大卫·查尔斯·唐奈·巴特勒
卢恰诺·恩里克·阿波尼
清水护
斯特凡尼·米歇尔·斯坦德利
大卫·约翰·布莱
安德鲁·古齐奥·赫什
吉加·德塞
杰克·大卫·戈弗雷
杨海林
岩本直树
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Publication of CN114585737A publication Critical patent/CN114585737A/zh
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • C12N15/113—Non-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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202080069504.2A 2019-10-06 2020-10-06 寡核苷酸组合物及其使用方法 Pending CN114585737A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962911334P 2019-10-06 2019-10-06
US62/911,334 2019-10-06
US202062959917P 2020-01-11 2020-01-11
US62/959,917 2020-01-11
US202063022559P 2020-05-10 2020-05-10
US63/022,559 2020-05-10
US202063069696P 2020-08-24 2020-08-24
US63/069,696 2020-08-24
PCT/US2020/054436 WO2021071858A1 (en) 2019-10-06 2020-10-06 Oligonucleotide compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
CN114585737A true CN114585737A (zh) 2022-06-03

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CN202080069504.2A Pending CN114585737A (zh) 2019-10-06 2020-10-06 寡核苷酸组合物及其使用方法

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US (1) US20230220384A1 (https=)
EP (1) EP4022059A4 (https=)
JP (2) JP7766589B2 (https=)
KR (1) KR20220076508A (https=)
CN (1) CN114585737A (https=)
AU (1) AU2020363391A1 (https=)
BR (1) BR112022006205A2 (https=)
CA (1) CA3154768A1 (https=)
IL (1) IL291933A (https=)
MX (1) MX2022004101A (https=)
WO (1) WO2021071858A1 (https=)

Families Citing this family (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
JP6453212B2 (ja) 2012-07-13 2019-01-16 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． キラル制御
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
CN108135921B (zh) 2015-07-22 2023-10-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
CA3015823A1 (en) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
CN109562122A (zh) 2016-06-03 2019-04-02 波涛生命科学有限公司 寡核苷酸、组合物及其方法
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11597927B2 (en) 2017-06-02 2023-03-07 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
TW201920672A (zh) 2017-08-08 2019-06-01 新加坡商波濤生命科學有限公司 寡核苷酸組合物及其使用方法
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
EP3790596A4 (en) 2018-05-11 2022-04-06 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
CN113748116A (zh) * 2019-03-20 2021-12-03 波涛生命科学有限公司 可用于寡核苷酸制备的技术
WO2022046667A1 (en) * 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cells and non-human animals engineered to express adar1 and uses thereof
EP4240849A4 (en) * 2020-11-08 2026-04-01 Wave Life Sciences Ltd OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES
US20230194709A9 (en) * 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278410A1 (en) * 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
AU2022352779A1 (en) * 2021-09-26 2024-03-28 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
CN119452084A (zh) * 2022-05-12 2025-02-14 波涛生命科学有限公司 寡核苷酸组合物及其方法
WO2023220428A1 (en) * 2022-05-12 2023-11-16 Wave Life Sciences Ltd. Compositions for editing ass1 transcripts and methods thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
EP4555086A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucleotides for adar-mediated rna editing and use thereof
JP2025525564A (ja) * 2022-07-18 2025-08-05 エフ． ホフマン－ラ ロシュ アーゲー オリゴヌクレオチドの編集
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
AR131145A1 (es) 2022-11-24 2025-02-19 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria
WO2024114908A1 (en) 2022-11-30 2024-06-06 Eberhard Karls Universität Tübingen Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing
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 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
EP4669753A1 (en) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024175586A2 (en) 2023-02-21 2024-08-29 Vib Vzw Inhibitors of synaptogyrin-3 expression
JP2026507051A (ja) 2023-02-21 2026-02-27 ブイアイビー ブイゼットダブリュ シナプトギリン－３発現を調節するためのオリゴヌクレオチド
EP4432289A1 (en) 2023-03-15 2024-09-18 Shape Therapeutics Inc. Generative sequence screening with conditional gans, diffusion models, and denoising diffusion conditional gans
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024256620A1 (en) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurodegenerative disease
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
AU2024325709A1 (en) 2023-08-15 2026-01-29 Airna Corporation Chemically modified antisense oligonucleotides (asos) and compositions for rna editing
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
AU2024354150A1 (en) * 2023-09-28 2026-03-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2025073902A1 (en) 2023-10-06 2025-04-10 Eberhard Karls Universität Tübingen Chemical and sequence optimisation of antisense oligonucleotides for adar-mediated rna editing
WO2025083268A1 (en) 2023-10-20 2025-04-24 Airna Corporation Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing
CN121909203A (zh) 2023-10-31 2026-04-21 科罗生物公司 包含氨基磷酸酯核苷酸间键的寡核苷酸
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
WO2025172372A1 (en) 2024-02-12 2025-08-21 Airna Corporation Conjugate for targeted delivery of an antisense oligonucleotide
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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014169206A2 (en) * 2013-04-11 2014-10-16 Carnegie Mellon University Divalent nucleobase compounds and uses therefor
ES2917473T3 (es) * 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
RU2708237C2 (ru) * 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
US10787664B2 (en) * 2015-05-26 2020-09-29 City Of Hope Compounds of chemically modified oligonucleotides and methods of use thereof
JP7511326B2 (ja) * 2015-10-09 2024-07-05 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およびその方法
CN109562122A (zh) * 2016-06-03 2019-04-02 波涛生命科学有限公司 寡核苷酸、组合物及其方法
US10941402B2 (en) * 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
US11718638B2 (en) * 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis

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EP4022059A4 (en) 2023-11-01
JP2025165976A (ja) 2025-11-05
AU2020363391A1 (en) 2022-03-24
JP7766589B2 (ja) 2025-11-10
US20230220384A1 (en) 2023-07-13
CA3154768A1 (en) 2021-04-15
KR20220076508A (ko) 2022-06-08
IL291933A (en) 2022-06-01
JP2022551124A (ja) 2022-12-07
BR112022006205A2 (pt) 2022-07-19
WO2021071858A1 (en) 2021-04-15
MX2022004101A (es) 2022-04-26
EP4022059A1 (en) 2022-07-06

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