CN111032057A - 寡核苷酸组合物及其方法 - Google Patents

寡核苷酸组合物及其方法 Download PDF

Info

Publication number
CN111032057A
CN111032057A CN201880052699.2A CN201880052699A CN111032057A CN 111032057 A CN111032057 A CN 111032057A CN 201880052699 A CN201880052699 A CN 201880052699A CN 111032057 A CN111032057 A CN 111032057A
Authority
CN
China
Prior art keywords
wing
oligonucleotide
composition
sugar
core
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.)
Pending
Application number
CN201880052699.2A
Other languages
English (en)
Chinese (zh)
Inventor
钱德拉·瓦尔格赛
钟忠
娜奥基·艾娃莫托
詹森·敬新·张
让-科斯梅·多达特
刘元婧
帕查穆图·坎德萨米
塞瑟马达范·迪瓦卡拉梅农
陆根良
苏布拉马尼安·马拉潘
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Wave Life Sciences Pte Ltd
Original Assignee
Wave Life Sciences Pte Ltd
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 Wave Life Sciences Pte Ltd filed Critical Wave Life Sciences Pte Ltd
Publication of CN111032057A publication Critical patent/CN111032057A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1072—Differential gene expression library synthesis, e.g. subtracted libraries, differential screening
    • 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
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
CN201880052699.2A 2017-08-08 2018-08-07 寡核苷酸组合物及其方法 Pending CN111032057A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762542778P 2017-08-08 2017-08-08
US62/542,778 2017-08-08
PCT/US2018/045659 WO2019032612A1 (en) 2017-08-08 2018-08-07 OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED METHODS

Publications (1)

Publication Number Publication Date
CN111032057A true CN111032057A (zh) 2020-04-17

Family

ID=65271538

Family Applications (2)

Application Number Title Priority Date Filing Date
CN201880051313.6A Pending CN110996968A (zh) 2017-08-08 2018-08-07 寡核苷酸组合物及其方法
CN201880052699.2A Pending CN111032057A (zh) 2017-08-08 2018-08-07 寡核苷酸组合物及其方法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
CN201880051313.6A Pending CN110996968A (zh) 2017-08-08 2018-08-07 寡核苷酸组合物及其方法

Country Status (11)

Country Link
US (3) US20200362337A1 (enExample)
EP (2) EP3664815A4 (enExample)
JP (3) JP2020534253A (enExample)
KR (1) KR20200035301A (enExample)
CN (2) CN110996968A (enExample)
AR (1) AR112779A1 (enExample)
AU (1) AU2018316159A1 (enExample)
CA (1) CA3072076A1 (enExample)
SG (1) SG11202000274RA (enExample)
TW (1) TW201920672A (enExample)
WO (2) WO2019032612A1 (enExample)

Families Citing this family (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112007045A (zh) 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
ES2862073T3 (es) 2012-07-13 2021-10-06 Wave Life Sciences Ltd Grupo auxiliar asimétrico
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
RU2708237C2 (ru) 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
NZ781029A (en) 2015-10-09 2025-07-25 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
US11407775B2 (en) 2016-03-13 2022-08-09 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45188A (fr) 2016-06-03 2019-04-10 Wave Life Sciences Ltd Oligonucléotides, compositions et méthodes associées
CN109477103A (zh) 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ 单链rna-编辑寡核苷酸
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
EP3551641A4 (en) 2016-12-08 2021-01-13 University of Utah Research Foundation STAUFEN1 AGENTS AND ASSOCIATED PROCESSES
JP7557941B2 (ja) 2017-06-02 2024-09-30 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
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2018237194A1 (en) 2017-06-21 2018-12-27 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
CN110996968A (zh) 2017-08-08 2020-04-10 波涛生命科学有限公司 寡核苷酸组合物及其方法
KR20200052369A (ko) 2017-09-18 2020-05-14 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 제조 기술
WO2019075357A1 (en) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
AU2019265904A1 (en) 2018-05-11 2020-11-12 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
JP2021527649A (ja) * 2018-06-14 2021-10-14 ユニバーシティー オブ ユタ リサーチ ファウンデーションＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｕｔａｈ Ｒｅｓｅａｒｃｈ Ｆｏｕｎｄａｔｉｏｎ Ｓｔａｕｆｅｎ１調節剤および関連する方法
EP3890752A1 (en) 2018-12-06 2021-10-13 Biogen MA Inc. Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis
CA3126845A1 (en) * 2019-02-01 2020-08-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2020191212A1 (en) * 2019-03-20 2020-09-24 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
WO2020227395A2 (en) * 2019-05-06 2020-11-12 University Of Massachusetts Anti-c9orf72 oligonucleotides and related methods
CN114502177A (zh) * 2019-05-09 2022-05-13 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
CA3145291A1 (en) * 2019-07-05 2021-01-14 University Of Florida Research Foundation, Incorporated Methods for treating ran protein-associated neurological diseases
WO2021071788A2 (en) * 2019-10-06 2021-04-15 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
EP4097236A4 (en) * 2020-01-28 2024-03-27 University of Southern California COMPOSITIONS AND METHODS FOR TREATING NEUROLOGICAL DISEASES
WO2021159008A2 (en) 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
BR112022020412A2 (pt) * 2020-04-09 2023-05-02 Association Inst De Myologie Sequências antissenso para tratar esclerose lateral amiotrófica
JP2023526533A (ja) 2020-05-22 2023-06-21 ウェイブ ライフ サイエンシズ リミテッド 二本鎖オリゴヌクレオチド組成物及びそれに関連する方法
WO2022216759A1 (en) 2021-04-06 2022-10-13 Maze Therapeutics, Inc. Compositions and methods for treating tdp-43 proteinopathy
TW202308663A (zh) * 2021-06-04 2023-03-01 美商艾拉倫製藥股份有限公司 人類染色體９開讀框７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ劑組成物及其使用方法
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
EP4522742A2 (en) 2022-05-13 2025-03-19 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
EP4555085A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Chemically modified oligonucleotides for adar-mediated rna editing
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
CN120283053A (zh) 2022-10-05 2025-07-08 追踪神经科学公司 Unc13a反义寡核苷酸和其用途
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
KR20250113455A (ko) 2022-11-24 2025-07-25 프로큐알 테라퓨틱스 Ⅱ 비.브이. 유전성 hfe-혈색소침착증의 치료를 위한 안티센스 올리고뉴클레오티드
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
KR20250115388A (ko) 2022-12-09 2025-07-30 프로큐알 테라퓨틱스 Ⅱ 비.브이. 심혈관 질환 치료용 안티센스 올리고뉴클레오타이드
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
KR102599078B1 (ko) 2023-03-21 2023-11-06 고아라 큐티클 케어 세트 및 이를 이용한 큐티클 케어 방법
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
CN121039282A (zh) 2023-03-24 2025-11-28 ProQR治疗上市公司Ⅱ 用于治疗神经系统病症的反义寡核苷酸
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105637090A (zh) * 2013-10-11 2016-06-01 Ionis制药公司 用于调节c9orf72表达的组合物
US20170044526A1 (en) * 2014-04-28 2017-02-16 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
WO2017062862A2 (en) * 2015-10-09 2017-04-13 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Family Cites Families (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6242589B1 (en) * 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
US6867295B2 (en) * 2001-09-07 2005-03-15 Dionex Corporation Ion exchange cryptands covalently bound to substrates
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
ES2550609T3 (es) 2002-07-10 2015-11-11 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Interferencia de ARN mediante de moléculas de ARN de cadena sencilla
US8729036B2 (en) 2002-08-07 2014-05-20 University Of Massachusetts 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
WO2005023828A1 (ja) 2003-09-02 2005-03-17 Takeshi Wada 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
EP2410053B2 (en) * 2006-10-18 2020-07-15 Ionis Pharmaceuticals, Inc. Antisense compounds
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
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
EP3208347B1 (en) * 2010-02-08 2019-08-14 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
US8859755B2 (en) 2010-03-05 2014-10-14 Chiralgen, Ltd. Method for preparing ribonucleoside phosphorothioate
US20140011860A1 (en) * 2010-07-19 2014-01-09 Isis Pharmaceuticals, Inc. Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
WO2012073857A1 (ja) 2010-11-30 2012-06-07 株式会社キラルジェン ２'－ｏ－修飾ｒｎａ
BR112014001244A2 (pt) 2011-07-19 2017-02-21 Wave Life Sciences Pte Ltd métodos para a síntese de ácidos nucléicos funcionalizados
EP3202914B2 (en) 2011-08-31 2023-06-28 The University of Manchester Method for treating a neurodegenerative disease
WO2013119979A1 (en) * 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Methods and compositions for modulating factor vii expression
WO2013159108A2 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
ES2862073T3 (es) 2012-07-13 2021-10-06 Wave Life Sciences Ltd Grupo auxiliar asimétrico
CN112007045A (zh) * 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
BR112015000723A2 (pt) 2012-07-13 2017-06-27 Shin Nippon Biomedical Laboratories Ltd adjuvante de ácido nucléico quiral
EP2906696B2 (en) * 2012-10-15 2022-12-14 Ionis Pharmaceuticals, Inc. Methods for modulating c9orf72 expression
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
WO2016024205A1 (en) * 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
WO2016138353A1 (en) 2015-02-26 2016-09-01 Ionis Pharmaceuticals, Inc. Allele specific modulators of p23h rhodopsin
MX366128B (es) * 2015-04-16 2019-06-28 Ionis Pharmaceuticals Inc Composiciones para modular la expresion de c90rf72.
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US11260073B2 (en) * 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
US11407775B2 (en) 2016-03-13 2022-08-09 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
AU2017248637A1 (en) 2016-04-13 2018-09-27 Ionis Pharmaceuticals, Inc. Methods for reducing C9ORF72 expression
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45188A (fr) 2016-06-03 2019-04-10 Wave Life Sciences Ltd Oligonucléotides, compositions et méthodes associées
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
JP7557941B2 (ja) 2017-06-02 2024-09-30 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
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP2020522265A (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
WO2018237194A1 (en) 2017-06-21 2018-12-27 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
US11814407B2 (en) 2017-06-28 2023-11-14 Roche Innovation Center Copenhagen A/S Multiple coupling and oxidation method
CN110996968A (zh) 2017-08-08 2020-04-10 波涛生命科学有限公司 寡核苷酸组合物及其方法
KR20200052369A (ko) 2017-09-18 2020-05-14 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 제조 기술
WO2019075357A1 (en) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
CA3096667A1 (en) 2018-04-12 2019-10-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
AU2019265904A1 (en) 2018-05-11 2020-11-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
AU2019392928A1 (en) 2018-12-06 2021-06-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
CA3126845A1 (en) 2019-02-01 2020-08-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
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
US20220307019A1 (en) 2019-03-25 2022-09-29 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex and use thereof
MX2021012981A (es) 2019-04-25 2022-01-18 Wave Life Sciences Ltd Composiciones de oligonucleotido y metodos de uso de las mismas.
AU2020261434A1 (en) 2019-04-25 2021-11-11 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN114502177A (zh) 2019-05-09 2022-05-13 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
AU2020363391A1 (en) 2019-10-06 2022-03-24 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2021071788A2 (en) 2019-10-06 2021-04-15 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
TW202146650A (zh) 2020-03-01 2021-12-16 新加坡商波濤生命科學有限公司 寡核苷酸組成物及其方法
JP2023526975A (ja) 2020-05-22 2023-06-26 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
WO2022046667A1 (en) 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cells and non-human animals engineered to express adar1 and uses thereof
EP4200616A1 (en) 2020-08-24 2023-06-28 Wave Life Sciences Ltd. Immunoassays for detecting wild type huntingtin protein and methods of treatment employing such immunoassays
MX2023005326A (es) 2020-11-08 2023-07-31 Wave Life Sciences Ltd Composiciones de oligonucleótidos y métodos de las mismas.

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105637090A (zh) * 2013-10-11 2016-06-01 Ionis制药公司 用于调节c9orf72表达的组合物
US20170044526A1 (en) * 2014-04-28 2017-02-16 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
WO2017062862A2 (en) * 2015-10-09 2017-04-13 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Also Published As

Publication number Publication date
US11739325B2 (en) 2023-08-29
EP3664816A4 (en) 2021-05-19
EP3664815A1 (en) 2020-06-17
US20200362337A1 (en) 2020-11-19
WO2019032607A1 (en) 2019-02-14
KR20200035301A (ko) 2020-04-02
CA3072076A1 (en) 2019-02-14
US20210032620A1 (en) 2021-02-04
JP2020534253A (ja) 2020-11-26
JP7422068B2 (ja) 2024-01-25
AR112779A1 (es) 2019-12-11
SG11202000274RA (en) 2020-02-27
JP2024009824A (ja) 2024-01-23
TW201920672A (zh) 2019-06-01
WO2019032612A1 (en) 2019-02-14
US20250066775A1 (en) 2025-02-27
CN110996968A (zh) 2020-04-10
AU2018316159A1 (en) 2020-02-06
JP2020534254A (ja) 2020-11-26
EP3664816A1 (en) 2020-06-17
EP3664815A4 (en) 2021-08-11

Similar Documents

Publication Publication Date Title
CN111032057A (zh) 寡核苷酸组合物及其方法
TWI877133B (zh) 寡核苷酸、其組成物、包含其的藥物組成物、其用途及其製造方法
CN115210377A (zh) 寡核苷酸组合物及其方法
CN114585737A (zh) 寡核苷酸组合物及其使用方法
TW201722439A (zh) 寡核苷酸組合物及其方法
TW201707711A (zh) 寡核苷酸組合物及其方法
CN109477108A (zh) 寡核苷酸组合物和其方法
JP2008501693A (ja) 遺伝子調節で使用するための個別に調節された鎖を有する二本鎖組成物
KR20240129626A (ko) 변형된 백본 화학을 갖는 스플라이스 스위처 안티센스 올리고뉴클레오티드
HK40019007A (en) Oligonucleotide compositions and methods thereof
RU2847121C2 (ru) Композиции на основе олигонуклеотидов и способы, связанные с ними
HK40027139A (en) Oligonucleotide compositions and methods thereof
WO2025054596A2 (en) Inhibin subunit beta e-related double stranded oligonucleotide compositions and methods relating thereto
HK40005788A (en) Oligonucleotide compositions and methods thereof

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40019007

Country of ref document: HK

CB03 Change of inventor or designer information

Inventor after: Chandra Walgsay

Inventor after: Subramanian Malapan

Inventor after: Zhong Zhong

Inventor after: Iwamoto Naoto

Inventor after: Jason Jingxin Zhang

Inventor after: Jean cosme dodat

Inventor after: Liu Yuanjing

Inventor after: Pachamutu Kandessami

Inventor after: Cesar Motor Van Diva Caramennon

Inventor after: Lu Genliang

Inventor before: Chandra Walgsay

Inventor before: Subramanian Malapan

Inventor before: Zhong Zhong

Inventor before: Naoji Eva Moto

Inventor before: Jason Jingxin Zhang

Inventor before: Jean cosme dodat

Inventor before: Liu Yuanjing

Inventor before: Pachamutu Kandessami

Inventor before: Cesar Motor Van Diva Caramennon

Inventor before: Lu Genliang

CB03 Change of inventor or designer information
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20200417

WD01 Invention patent application deemed withdrawn after publication