TW201920672A - 寡核苷酸組合物及其使用方法 - Google Patents

寡核苷酸組合物及其使用方法 Download PDF

Info

Publication number
TW201920672A
TW201920672A TW107127371A TW107127371A TW201920672A TW 201920672 A TW201920672 A TW 201920672A TW 107127371 A TW107127371 A TW 107127371A TW 107127371 A TW107127371 A TW 107127371A TW 201920672 A TW201920672 A TW 201920672A
Authority
TW
Taiwan
Prior art keywords
oligonucleotide
wing
c9orf72
composition
core
Prior art date
Application number
TW107127371A
Other languages
English (en)
Chinese (zh)
Inventor
珍 克斯美 道達特
劉元婧
錢德拉 瓦吉斯
忠 鐘
岩本直樹
敬新 張
帕恰姆蘇 肯達賽美
塞斯麥哈凡 戴瓦卡拉曼恩
根良 陸
薩瑞曼尼恩 馬拉帕
Original Assignee
新加坡商波濤生命科學有限公司
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 新加坡商波濤生命科學有限公司 filed Critical 新加坡商波濤生命科學有限公司
Publication of TW201920672A publication Critical patent/TW201920672A/zh

Links

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)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (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)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
TW107127371A 2017-08-08 2018-08-07 寡核苷酸組合物及其使用方法 TW201920672A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762542778P 2017-08-08 2017-08-08
US62/542,778 2017-08-08

Publications (1)

Publication Number Publication Date
TW201920672A true TW201920672A (zh) 2019-06-01

Family

ID=65271538

Family Applications (1)

Application Number Title Priority Date Filing Date
TW107127371A TW201920672A (zh) 2017-08-08 2018-08-07 寡核苷酸組合物及其使用方法

Country Status (11)

Country Link
US (3) US20200362337A1 (OSRAM)
EP (2) EP3664815A4 (OSRAM)
JP (4) JP7422068B2 (OSRAM)
KR (1) KR20200035301A (OSRAM)
CN (2) CN110996968A (OSRAM)
AR (1) AR112779A1 (OSRAM)
AU (1) AU2018316159A1 (OSRAM)
CA (1) CA3072076A1 (OSRAM)
SG (1) SG11202000274RA (OSRAM)
TW (1) TW201920672A (OSRAM)
WO (2) WO2019032607A1 (OSRAM)

Families Citing this family (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2872485T3 (da) 2012-07-13 2021-03-08 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
CN112007045B (zh) 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
KR102423317B1 (ko) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
RU2708237C2 (ru) 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
WO2017015575A1 (en) 2015-07-22 2017-01-26 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3359523A4 (en) 2015-10-09 2019-07-24 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR
AU2017234150B2 (en) 2016-03-13 2021-09-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
JP2019516680A (ja) 2016-05-04 2019-06-20 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およびその方法
US11013757B2 (en) 2016-06-03 2021-05-25 Wave Life Sciences Ltd. Oligonucleotides, compositions and methods thereof
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
CA3035293A1 (en) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
JP7296882B2 (ja) 2016-11-23 2023-06-23 ウェイブ ライフ サイエンシズ リミテッド ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法
EP3551641A4 (en) 2016-12-08 2021-01-13 University of Utah Research Foundation STAUFEN1 ACTIVE SUBSTANCES AND RELATED PROCEDURES
WO2018223081A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN111050806A (zh) 2017-06-02 2020-04-21 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
WO2018237194A1 (en) 2017-06-21 2018-12-27 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
US11608355B2 (en) 2017-09-18 2023-03-21 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
US11596646B2 (en) 2017-10-12 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
CA3090901A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
SG11202010131QA (en) 2018-05-11 2020-11-27 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
EP3807291A4 (en) * 2018-06-14 2022-02-23 University of Utah Research Foundation STAUFEN1 REGULATORY AGENTS AND ASSOCIATED PROCEDURES
KR20210102294A (ko) 2018-12-06 2021-08-19 바이오젠 엠에이 인코포레이티드 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질
BR112021014940A2 (pt) * 2019-02-01 2021-09-28 Wave Life Sciences Ltd. Oligonucleotídeo, composição de oligonucleotídeo quiralmente controlada, composição farmacêutica, e método de tratamento, prevenção, atraso de início e/ou diminuição da gravidade de pelo menos um sintoma de doença de huntington
WO2020191212A1 (en) * 2019-03-20 2020-09-24 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
US12590115B2 (en) 2019-03-20 2026-03-31 Wave Life Sciences Ltd. Technologies useful for oligonucleotide preparation
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
EP3994159A4 (en) * 2019-07-05 2023-08-09 University of Florida Research Foundation, Incorporated METHODS OF TREATING NEUROLOGICAL DISEASES ASSOCIATED WITH THE RAN PROTEIN
CA3156176A1 (en) * 2019-10-06 2021-04-15 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US20230098111A1 (en) * 2020-01-28 2023-03-30 University Of Southern California Compositions and methods to treat neurological diseases
WO2021159008A2 (en) 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
JP7787094B2 (ja) * 2020-04-09 2025-12-16 アソシエーション・アンスティトゥート・ドゥ・マイオロジー 筋萎縮性側索硬化症を治療するためのアンチセンス配列
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
AU2022255175A1 (en) 2021-04-06 2023-11-23 Trace Neuroscience, Inc. Compositions and methods for treating tdp-43 proteinopathy
BR112023025224A2 (pt) * 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
JP2025516680A (ja) 2022-05-13 2025-05-30 アルナイラム ファーマシューティカルズ， インコーポレイテッド 一本鎖ループオリゴヌクレオチド
EP4555086A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucleotides for adar-mediated rna editing and use thereof
EP4555085A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Chemically modified oligonucleotides for adar-mediated rna editing
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
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
CN120322556A (zh) 2022-12-09 2025-07-15 ProQR治疗上市公司Ⅱ 用于治疗心血管疾病的反义寡核苷酸
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
JP2026510408A (ja) 2023-03-24 2026-04-02 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 神経障害の処置のためのアンチセンスオリゴヌクレオチド
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
CN121002181A (zh) 2023-03-27 2025-11-21 ProQR治疗上市公司Ⅱ 用于治疗肝脏疾病的反义寡核苷酸
CN121729491A (zh) 2023-05-12 2026-03-24 阿尔尼拉姆制药公司 单链环寡核苷酸
AR132964A1 (es) 2023-06-16 2025-08-13 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de enfermedades neurodegenerativas
AU2024287308A1 (en) 2023-07-13 2025-12-18 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
AU2024379264A1 (en) 2023-11-16 2026-05-14 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
WO2026093339A1 (en) 2024-10-29 2026-05-07 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of peroxisome biogenesis disorders in the zellweger spectrum (pbd-zsd)

Family Cites Families (71)

* 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
US8101348B2 (en) 2002-07-10 2012-01-24 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. RNA-interference by single-stranded RNA molecules
WO2004014933A1 (en) 2002-08-07 2004-02-19 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
JP4945129B2 (ja) 2004-01-27 2012-06-06 株式会社キラルジェン 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
CA2667055C (en) * 2006-10-18 2017-05-09 Isis Pharmaceuticals, Inc. Antisense compounds
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
CN102574888A (zh) 2009-09-16 2012-07-11 株式会社启拉坚 用于rna及其衍生物的合成的新型保护基
EP2534262B1 (en) * 2010-02-08 2016-12-14 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2011108682A1 (ja) 2010-03-05 2011-09-09 国立大学法人 東京大学 リボヌクレオシドホスホロチオエートの製造方法
WO2012027033A1 (en) * 2010-07-19 2012-03-01 Isis Pharmaceuticals, Inc. Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
EP2647644B1 (en) 2010-11-30 2020-09-09 Wave Life Sciences Japan, Inc. 2'-o-modified rna
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
CA2846307C (en) 2011-08-31 2020-03-10 University College Cardiff Consultants Limited Method for diagnosing a neurodegenerative disease
CA2863958A1 (en) 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Methods and compositions for modulating factor vii expression
EP2839006B1 (en) * 2012-04-20 2018-01-03 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
DK2872485T3 (da) 2012-07-13 2021-03-08 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
JP6246121B2 (ja) 2012-07-13 2017-12-13 株式会社新日本科学 キラル核酸アジュバント
CN112007045B (zh) * 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
US9963699B2 (en) 2012-10-15 2018-05-08 Ionis Pharmaceuticals, Inc. Methods for modulating C9ORF72 expression
SG10201808903UA (en) 2013-10-11 2018-11-29 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
WO2015108048A1 (ja) 2014-01-15 2015-07-23 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
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
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
KR102423317B1 (ko) * 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
WO2015168172A1 (en) * 2014-04-28 2015-11-05 Isis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
WO2016024205A1 (en) 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
US10426789B2 (en) 2015-02-26 2019-10-01 Ionis Pharmaceuticals, Inc. Allele specific modulators of P23H rhodopsin
SI3283080T1 (sl) 2015-04-16 2020-06-30 Ionis Pharmaceuticals, Inc. Sestavki za moduliranje izražanja C9ORF72
WO2017015575A1 (en) 2015-07-22 2017-01-26 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3359523A4 (en) 2015-10-09 2019-07-24 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR
US11260073B2 (en) 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
AU2017234150B2 (en) 2016-03-13 2021-09-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
ES2933435T3 (es) 2016-04-13 2023-02-08 Ionis Pharmaceuticals Inc Métodos para reducir la expresión de C9ORF72
US20190249173A1 (en) 2016-05-04 2019-08-15 Chandra Vargeese Methods and compositions of biologically active agents
JP2019516680A (ja) 2016-05-04 2019-06-20 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およびその方法
US11013757B2 (en) 2016-06-03 2021-05-25 Wave Life Sciences Ltd. Oligonucleotides, compositions and methods thereof
WO2018022473A1 (en) 2016-07-25 2018-02-01 Wave Life Sciences Ltd. Phasing
JP7296882B2 (ja) 2016-11-23 2023-06-23 ウェイブ ライフ サイエンシズ リミテッド ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法
WO2018223081A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN111050806A (zh) 2017-06-02 2020-04-21 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
EP3630788A4 (en) 2017-06-02 2021-04-28 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR THEIR USE
WO2018237194A1 (en) 2017-06-21 2018-12-27 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
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
US11608355B2 (en) 2017-09-18 2023-03-21 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
US11596646B2 (en) 2017-10-12 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
SG11202010131QA (en) 2018-05-11 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
CN113383078A (zh) 2018-12-06 2021-09-10 波涛生命科学有限公司 寡核苷酸组合物及其方法
BR112021014940A2 (pt) 2019-02-01 2021-09-28 Wave Life Sciences Ltd. Oligonucleotídeo, composição de oligonucleotídeo quiralmente controlada, composição farmacêutica, e método de tratamento, prevenção, atraso de início e/ou diminuição da gravidade de pelo menos um sintoma de doença de huntington
US12590115B2 (en) 2019-03-20 2026-03-31 Wave Life Sciences Ltd. Technologies useful for oligonucleotide preparation
JPWO2020196662A1 (OSRAM) 2019-03-25 2020-10-01
EP3958872A4 (en) 2019-04-25 2024-07-03 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
AU2020262485A1 (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
CA3156176A1 (en) 2019-10-06 2021-04-15 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CA3154768A1 (en) 2019-10-06 2021-04-15 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
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
EP4153604A4 (en) 2020-05-22 2024-11-27 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
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
CN115989041A (zh) 2020-08-24 2023-04-18 波涛生命科学有限公司 经工程化为表达adar1的细胞和非人动物及其用途
KR20230118716A (ko) 2020-11-08 2023-08-11 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법

Also Published As

Publication number Publication date
AU2018316159A1 (en) 2020-02-06
JP2024009824A (ja) 2024-01-23
AR112779A1 (es) 2019-12-11
EP3664816A1 (en) 2020-06-17
KR20200035301A (ko) 2020-04-02
US20200362337A1 (en) 2020-11-19
US20250066775A1 (en) 2025-02-27
CN111032057A (zh) 2020-04-17
US11739325B2 (en) 2023-08-29
CA3072076A1 (en) 2019-02-14
JP7422068B2 (ja) 2024-01-25
EP3664815A1 (en) 2020-06-17
US20210032620A1 (en) 2021-02-04
JP2020534253A (ja) 2020-11-26
SG11202000274RA (en) 2020-02-27
JP2020534254A (ja) 2020-11-26
EP3664815A4 (en) 2021-08-11
CN110996968A (zh) 2020-04-10
EP3664816A4 (en) 2021-05-19
JP2026016363A (ja) 2026-02-03
WO2019032607A1 (en) 2019-02-14
WO2019032612A1 (en) 2019-02-14

Similar Documents

Publication Publication Date Title
US20250066775A1 (en) Oligonucleotide compositions and methods thereof
TW202126810A (zh) 寡核苷酸組成物及其使用方法
JP2025125554A (ja) オリゴヌクレオチド組成物及びその使用方法
TW202045189A (zh) 寡核苷酸組成物及其方法
KR20210005057A (ko) 올리고뉴클레오티드 조성물 및 이의 이용 방법
JP2024543214A (ja) Ｕｎｃ１３ａ遺伝子転写物のモジュレーターを使用する神経疾患の処置
JP2024545052A (ja) 修飾骨格化学を有するスプライススイッチャーアンチセンスオリゴヌクレオチド
CA3185488A1 (en) Treatment of neurological diseases using modulators of gene transcripts
RU2847121C2 (ru) Композиции на основе олигонуклеотидов и способы, связанные с ними
HK40121031A (zh) 具有经修饰的主链化学的剪接转换器反义寡核苷酸
CN116528878A (zh) 使用基因转录物调控剂治疗神经学疾病
HK40027139A (en) Oligonucleotide compositions and methods thereof
HK40097883A (zh) 使用基因转录物调控剂治疗神经学疾病
HK40121931A (zh) 具有修饰的主链化学的gapmer反义寡核苷酸
HK40019007A (en) Oligonucleotide compositions and methods thereof