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 PDFInfo
- 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
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)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (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)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
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) | US11739325B2 (enExample) |
| EP (2) | EP3664816A4 (enExample) |
| JP (3) | JP7422068B2 (enExample) |
| KR (1) | KR20200035301A (enExample) |
| CN (2) | CN111032057A (enExample) |
| AR (1) | AR112779A1 (enExample) |
| AU (1) | AU2018316159A1 (enExample) |
| CA (1) | CA3072076A1 (enExample) |
| SG (1) | SG11202000274RA (enExample) |
| TW (1) | TW201920672A (enExample) |
| WO (2) | WO2019032607A1 (enExample) |
Families Citing this family (61)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
| MX2016009290A (es) | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| 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 |
| SG10201912902TA (en) | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| CN114685589A (zh) | 2016-03-13 | 2022-07-01 | 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 |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US11013757B2 (en) | 2016-06-03 | 2021-05-25 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| ES2837076T3 (es) | 2016-09-01 | 2021-06-29 | Proqr Therapeutics Ii Bv | Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente |
| WO2018098264A1 (en) | 2016-11-23 | 2018-05-31 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| EP3551641A4 (en) | 2016-12-08 | 2021-01-13 | University of Utah Research Foundation | STAUFEN1 AGENTS AND ASSOCIATED PROCESSES |
| WO2018223056A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2018223081A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| JP7402696B2 (ja) | 2017-06-21 | 2023-12-21 | ウェイブ ライフ サイエンシズ リミテッド | 合成のための化合物、組成物、及び方法 |
| EP3664816A4 (en) | 2017-08-08 | 2021-05-19 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES |
| AU2018333065B2 (en) | 2017-09-18 | 2024-04-18 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
| WO2019075357A1 (en) | 2017-10-12 | 2019-04-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| AU2019218987B2 (en) | 2018-02-12 | 2025-04-24 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| CA3098624A1 (en) | 2018-05-11 | 2019-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| EP3807291A4 (en) * | 2018-06-14 | 2022-02-23 | University of Utah Research Foundation | STAUFEN1 REGULATING AGENTS AND RELATED METHODS |
| KR20210102294A (ko) | 2018-12-06 | 2021-08-19 | 바이오젠 엠에이 인코포레이티드 | 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질 |
| CA3126845A1 (en) * | 2019-02-01 | 2020-08-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| EP3942049A4 (en) * | 2019-03-20 | 2023-10-18 | President And Fellows Of Harvard College | ANTISENSE OLIGONUCLEOTIDE BASED PROGRANULIN AUGMENTATION THERAPY FOR NEURODEGENERATIVE DISEASES |
| US11629347B2 (en) | 2019-05-06 | 2023-04-18 | 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 |
| US20220267776A1 (en) * | 2019-07-05 | 2022-08-25 | 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 |
| WO2021155067A1 (en) * | 2020-01-28 | 2021-08-05 | 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 |
| WO2021205005A2 (en) * | 2020-04-09 | 2021-10-14 | Association Institut De Myologie | Antisense sequences for treating amyotrophic lateral sclerosis |
| KR20230016201A (ko) | 2020-05-22 | 2023-02-01 | 웨이브 라이프 사이언시스 리미티드 | 이중 가닥 올리고뉴클레오티드 조성물 및 이와 관련된 방법 |
| JP2024513237A (ja) | 2021-04-06 | 2024-03-22 | メイズ セラピューティクス， インコーポレイテッド | Ｔｄｐ－４３プロテイノパチーを処置するための組成物及び方法 |
| CN117561334A (zh) * | 2021-06-04 | 2024-02-13 | 阿尔尼拉姆医药品有限公司 | 人染色体9开放阅读框72（C9ORF72）iRNA药剂组合物和其使用方法 |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| US20250304957A1 (en) | 2022-05-13 | 2025-10-02 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | 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 |
| EP4599062A1 (en) | 2022-10-05 | 2025-08-13 | Trace Neuroscience, Inc. | Unc13a antisense oligonucleotides and uses thereof |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| EP4623084A1 (en) | 2022-11-24 | 2025-10-01 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| CA3276262A1 (en) | 2022-12-09 | 2024-06-13 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of cardiovascular disease |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| WO2024175550A1 (en) | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| KR102599078B1 (ko) | 2023-03-21 | 2023-11-06 | 고아라 | 큐티클 케어 세트 및 이를 이용한 큐티클 케어 방법 |
| WO2024206175A1 (en) | 2023-03-24 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| AU2024246572A1 (en) | 2023-03-27 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| WO2024238385A2 (en) | 2023-05-12 | 2024-11-21 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
| AR132964A1 (es) | 2023-06-16 | 2025-08-13 | Proqr Therapeutics Ii Bv | Oligonucleótidos antisentido para el tratamiento de enfermedades neurodegenerativas |
| 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 |
Family Cites Families (71)
| 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 |
| EP2333062A1 (en) | 2002-07-10 | 2011-06-15 | Max-Planck-Gesellschaft zur Förderung 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 |
| 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 |
| WO2005028494A1 (ja) | 2003-09-02 | 2005-03-31 | Takeshi Wada | 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 |
| JP4865544B2 (ja) | 2004-03-25 | 2012-02-01 | 株式会社キラルジェン | 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 |
| JP5665317B2 (ja) | 2006-10-18 | 2015-02-04 | アイシス ファーマシューティカルズ， インコーポレーテッド | アンチセンス化合物 |
| RU2015151857A (ru) | 2008-12-02 | 2019-01-15 | Уэйв Лайф Сайенсес Джапан, Инк. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| 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 |
| CA2789038A1 (en) * | 2010-02-08 | 2011-08-11 | Isis 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 |
| 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 |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| JP6093924B2 (ja) | 2010-11-30 | 2017-03-15 | 株式会社Wave Life Sciences Japan | ２’−ｏ−修飾ｒｎａ |
| EP3248982A1 (en) | 2011-07-19 | 2017-11-29 | Wave Life Sciences Ltd. | Thiosulfonate reagents for the synthesis of functionalized nucleic acids |
| CA2846307C (en) | 2011-08-31 | 2020-03-10 | Hospital District Of Helsinki And Uusimaa | Method for diagnosing a neurodegenerative disease |
| EP2812433A4 (en) | 2012-02-08 | 2016-01-20 | Isis Pharmaceuticals Inc | METHOD AND COMPOSITIONS FOR MODULATING THE FACTOR VII EXPRESSION |
| EP2839006B1 (en) * | 2012-04-20 | 2018-01-03 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
| CA2879066C (en) | 2012-07-13 | 2019-08-13 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| FI2906696T4 (fi) | 2012-10-15 | 2023-03-18 | Menetelmiä c9orf72:n ilmentymisen moduloimiseksi | |
| MY192689A (en) * | 2013-10-11 | 2022-09-01 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
| 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 |
| 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 |
| 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 |
| MX2016009290A (es) * | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| 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 |
| BR112017017178A2 (pt) | 2015-02-26 | 2018-04-03 | Ionis Pharmaceuticals Inc | moduladores específicos de alelo de rodopsina de p23h |
| JP6606557B2 (ja) | 2015-04-16 | 2019-11-13 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | Ｃ９ｏｒｆ７２発現を調節するための組成物 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| SG10201912902TA (en) | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| US11260073B2 (en) * | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| CN114685589A (zh) | 2016-03-13 | 2022-07-01 | 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 |
| WO2017180835A1 (en) | 2016-04-13 | 2017-10-19 | Ionis Pharmaceuticals, Inc. | Methods for reducing c9orf72 expression |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| MA45290A (fr) | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
| US11013757B2 (en) | 2016-06-03 | 2021-05-25 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| US20190264267A1 (en) | 2016-07-25 | 2019-08-29 | Wave Life Sciences Ltd. | Phasing |
| WO2018098264A1 (en) | 2016-11-23 | 2018-05-31 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| WO2018223081A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2018223056A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2018223073A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| JP7402696B2 (ja) | 2017-06-21 | 2023-12-21 | ウェイブ ライフ サイエンシズ リミテッド | 合成のための化合物、組成物、及び方法 |
| JP7492829B2 (ja) | 2017-06-28 | 2024-05-30 | ロシュ イノベーション センター コペンハーゲン エーエス | 多重カップリングおよび酸化の方法 |
| EP3664816A4 (en) | 2017-08-08 | 2021-05-19 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES |
| AU2018333065B2 (en) | 2017-09-18 | 2024-04-18 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
| WO2019075357A1 (en) | 2017-10-12 | 2019-04-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| AU2019252680B2 (en) | 2018-04-12 | 2025-11-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CA3098624A1 (en) | 2018-05-11 | 2019-11-14 | 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 |
| CA3126845A1 (en) | 2019-02-01 | 2020-08-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| US20230089442A1 (en) | 2019-03-20 | 2023-03-23 | Pachamuthu Kandasamy | Technologies useful for oligonucleotide preparation |
| JPWO2020196662A1 (enExample) | 2019-03-25 | 2020-10-01 | ||
| MX2021012981A (es) | 2019-04-25 | 2022-01-18 | Wave Life Sciences Ltd | Composiciones de oligonucleotido y metodos de uso de las mismas. |
| CA3137740A1 (en) | 2019-04-25 | 2020-10-29 | 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 |
| US20230220384A1 (en) | 2019-10-06 | 2023-07-13 | Prashant Monian | 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 |
| CN115210377A (zh) | 2020-03-01 | 2022-10-18 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| US20230203087A1 (en) | 2020-05-22 | 2023-06-29 | Pachamuthu Kandasamy | Oligonucleotide compositions and methods 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 |
| US20230329201A1 (en) | 2020-08-24 | 2023-10-19 | Wave Life Sciences Ltd. | Cells and non-human animals engineered to express adar1 and uses thereof |
| KR20230118716A (ko) | 2020-11-08 | 2023-08-11 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 방법 |
-
2018
- 2018-08-07 EP EP18844660.3A patent/EP3664816A4/en not_active Withdrawn
- 2018-08-07 WO PCT/US2018/045653 patent/WO2019032607A1/en not_active Ceased
- 2018-08-07 WO PCT/US2018/045659 patent/WO2019032612A1/en not_active Ceased
- 2018-08-07 CN CN201880052699.2A patent/CN111032057A/zh active Pending
- 2018-08-07 CN CN201880051313.6A patent/CN110996968A/zh active Pending
- 2018-08-07 SG SG11202000274RA patent/SG11202000274RA/en unknown
- 2018-08-07 TW TW107127371A patent/TW201920672A/zh unknown
- 2018-08-07 US US16/636,902 patent/US11739325B2/en active Active
- 2018-08-07 US US16/636,900 patent/US20200362337A1/en not_active Abandoned
- 2018-08-07 KR KR1020207006702A patent/KR20200035301A/ko not_active Ceased
- 2018-08-07 AU AU2018316159A patent/AU2018316159A1/en not_active Abandoned
- 2018-08-07 JP JP2020506814A patent/JP7422068B2/ja active Active
- 2018-08-07 AR ARP180102248A patent/AR112779A1/es unknown
- 2018-08-07 JP JP2020506776A patent/JP2020534253A/ja active Pending
- 2018-08-07 EP EP18842985.6A patent/EP3664815A4/en active Pending
- 2018-08-07 CA CA3072076A patent/CA3072076A1/en active Pending
-
2023
- 2023-09-25 JP JP2023161596A patent/JP2024009824A/ja active Pending
-
2024
- 2024-03-21 US US18/613,034 patent/US20250066775A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US11739325B2 (en) | 2023-08-29 |
| US20250066775A1 (en) | 2025-02-27 |
| JP2020534254A (ja) | 2020-11-26 |
| EP3664815A1 (en) | 2020-06-17 |
| KR20200035301A (ko) | 2020-04-02 |
| CN110996968A (zh) | 2020-04-10 |
| EP3664815A4 (en) | 2021-08-11 |
| EP3664816A4 (en) | 2021-05-19 |
| WO2019032612A1 (en) | 2019-02-14 |
| JP2020534253A (ja) | 2020-11-26 |
| AR112779A1 (es) | 2019-12-11 |
| CN111032057A (zh) | 2020-04-17 |
| JP2024009824A (ja) | 2024-01-23 |
| CA3072076A1 (en) | 2019-02-14 |
| WO2019032607A1 (en) | 2019-02-14 |
| US20200362337A1 (en) | 2020-11-19 |
| US20210032620A1 (en) | 2021-02-04 |
| AU2018316159A1 (en) | 2020-02-06 |
| SG11202000274RA (en) | 2020-02-27 |
| JP7422068B2 (ja) | 2024-01-25 |
| EP3664816A1 (en) | 2020-06-17 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| TW201920672A (zh) | 寡核苷酸組合物及其使用方法 | |
| 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 | |
| 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 | |
| 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 | |
| CA3185488A1 (en) | Treatment of neurological diseases using modulators of gene transcripts | |
| RU2847121C2 (ru) | Композиции на основе олигонуклеотидов и способы, связанные с ними | |
| 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 | |
| JP2025179843A (ja) | オリゴヌクレオチド組成物及びその使用方法 | |
| HK40019007A (en) | Oligonucleotide compositions and methods thereof |