JP2020522265A5 - - Google Patents
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- JP2020522265A5 JP2020522265A5 JP2019566837A JP2019566837A JP2020522265A5 JP 2020522265 A5 JP2020522265 A5 JP 2020522265A5 JP 2019566837 A JP2019566837 A JP 2019566837A JP 2019566837 A JP2019566837 A JP 2019566837A JP 2020522265 A5 JP2020522265 A5 JP 2020522265A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- oligonucleotides
- composition according
- base sequence
- share
- 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
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- 108091034117 Oligonucleotide Proteins 0.000 claims 20
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 13
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 claims 5
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 claims 5
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims 3
- 208000019425 cirrhosis of liver Diseases 0.000 claims 3
- 229910052698 phosphorus Inorganic materials 0.000 claims 3
- 239000011574 phosphorus Substances 0.000 claims 3
- 102100030970 Apolipoprotein C-III Human genes 0.000 claims 2
- 206010016654 Fibrosis Diseases 0.000 claims 2
- 101000793223 Homo sapiens Apolipoprotein C-III Proteins 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-N Phosphoric acid Chemical compound OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 claims 2
- 230000007882 cirrhosis Effects 0.000 claims 2
- 208000004930 Fatty Liver Diseases 0.000 claims 1
- 206010019708 Hepatic steatosis Diseases 0.000 claims 1
- 229910000147 aluminium phosphate Inorganic materials 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 239000000470 constituent Substances 0.000 claims 1
- 208000010706 fatty liver disease Diseases 0.000 claims 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 claims 1
- 231100000844 hepatocellular carcinoma Toxicity 0.000 claims 1
- 239000007788 liquid Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 231100000240 steatosis hepatitis Toxicity 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023125286A JP2023153920A (ja) | 2017-06-02 | 2023-08-01 | オリゴヌクレオチド組成物及びその使用方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762514769P | 2017-06-02 | 2017-06-02 | |
| US62/514,769 | 2017-06-02 | ||
| US201862670702P | 2018-05-11 | 2018-05-11 | |
| US62/670,702 | 2018-05-11 | ||
| PCT/US2018/035712 WO2018223073A1 (en) | 2017-06-02 | 2018-06-01 | Oligonucleotide compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023125286A Division JP2023153920A (ja) | 2017-06-02 | 2023-08-01 | オリゴヌクレオチド組成物及びその使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020522265A JP2020522265A (ja) | 2020-07-30 |
| JP2020522265A5 true JP2020522265A5 (enExample) | 2021-08-19 |
Family
ID=64455646
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019566837A Pending JP2020522265A (ja) | 2017-06-02 | 2018-06-01 | オリゴヌクレオチド組成物及びその使用方法 |
| JP2023125286A Pending JP2023153920A (ja) | 2017-06-02 | 2023-08-01 | オリゴヌクレオチド組成物及びその使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023125286A Pending JP2023153920A (ja) | 2017-06-02 | 2023-08-01 | オリゴヌクレオチド組成物及びその使用方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US20210198305A1 (enExample) |
| EP (1) | EP3630788A4 (enExample) |
| JP (2) | JP2020522265A (enExample) |
| CN (2) | CN111164091B (enExample) |
| TW (1) | TW201908483A (enExample) |
| WO (1) | WO2018223073A1 (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. |
| 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 |
| SG11201906728TA (en) | 2017-02-06 | 2019-08-27 | Mpeg La Llc | Multimeric oligonucleotides having decreased kidney clearance |
| 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 |
| 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 |
| KR102834361B1 (ko) | 2017-12-01 | 2025-07-17 | 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 | 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도 |
| WO2019105418A1 (zh) | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途 |
| US11414665B2 (en) | 2017-12-01 | 2022-08-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| JP7365052B2 (ja) | 2017-12-01 | 2023-10-19 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用 |
| US11414661B2 (en) | 2017-12-01 | 2022-08-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof |
| CA3087106A1 (en) | 2017-12-29 | 2019-07-04 | Suzhou Ribo Life Science Co., Ltd. | Conjugates and preparation and use 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 |
| JP2021533800A (ja) | 2018-08-21 | 2021-12-09 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッドSuzhou Ribo Life Science Co., Ltd. | 核酸、当該核酸を含む薬物組成物及び複合体ならびにその使用 |
| CN111655297A (zh) | 2018-09-30 | 2020-09-11 | 苏州瑞博生物技术有限公司 | 一种siRNA缀合物及其制备方法和用途 |
| JP2022523226A (ja) * | 2019-03-04 | 2022-04-21 | エムペグ エルエイ リミテッド ライアビリティ カンパニー | 増強された生理活性を有する多量体オリゴヌクレオチド |
| MX2022001460A (es) * | 2019-08-05 | 2022-02-22 | Arrowhead Pharmaceuticals Inc | Metodos para el tratamiento de enfermedades y trastornos relacionados con apoc3. |
| TW202122093A (zh) * | 2019-08-29 | 2021-06-16 | 大陸商蘇州瑞博生物技術股份有限公司 | 化合物、藥物綴合物、試劑盒及其用途 |
| IL294515A (en) | 2020-01-31 | 2022-09-01 | Avilar Therapeutics Inc | Compounds bind asgpr to degrade extracellular proteins |
| AR121446A1 (es) * | 2020-02-28 | 2022-06-08 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular smn2 |
| US11478533B2 (en) | 2020-04-27 | 2022-10-25 | Novo Nordisk A/S | Semaglutide for use in medicine |
| KR20230016201A (ko) * | 2020-05-22 | 2023-02-01 | 웨이브 라이프 사이언시스 리미티드 | 이중 가닥 올리고뉴클레오티드 조성물 및 이와 관련된 방법 |
| AU2021306628A1 (en) * | 2020-07-09 | 2022-11-24 | F. Hoffmann-La Roche Ag | Process for the preparation of oligonucleotides using modified oxidation protocol |
| CN111978179A (zh) * | 2020-08-27 | 2020-11-24 | 青岛科技大学 | 一种6-羟基己酸酯的合成方法 |
| AU2022268925A1 (en) | 2021-05-03 | 2023-11-16 | Avilar Therapeutics, Inc. | Potent asgpr-binding compounds for the degradation of immunoglobulins and other proteins |
| CA3225015A1 (en) * | 2021-07-16 | 2023-01-19 | Zicai Liang | Nucleic acid, composition and conjugate containing same, and preparation method and use |
| WO2023083442A1 (en) | 2021-11-10 | 2023-05-19 | Norchem Limited | Process for producing a triazine ring containing compound, addition product, triazine ring containing polyol, use of the addition product, use of the triazine ring containing polyol and pu/pir foam obtained by using the triazine ring containing polyol |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2024013361A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| 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 |
| 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 |
| 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 |
| 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 |
| WO2025199466A1 (en) | 2024-03-22 | 2025-09-25 | Purdue Research Foundation | Liver-specific asialoglycoprotein receptor targeting ligands, conjugates comprising same, and related compositions and methods of use |
| 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 (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8324366B2 (en) * | 2008-04-29 | 2012-12-04 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for delivering RNAI using lipoproteins |
| CN104661664B (zh) * | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
| SG11201503821YA (en) * | 2012-11-15 | 2015-06-29 | Roche Innovation Ct Copenhagen As | Oligonucleotide conjugates |
| KR102482890B1 (ko) * | 2013-05-01 | 2022-12-30 | 아이오니스 파마수티컬즈, 인코포레이티드 | 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법 |
| MX2016009290A (es) * | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| EP3221451A1 (en) * | 2014-11-17 | 2017-09-27 | Alnylam Pharmaceuticals, Inc. | Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof |
| SG10201912902TA (en) * | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
-
2018
- 2018-06-01 WO PCT/US2018/035712 patent/WO2018223073A1/en not_active Ceased
- 2018-06-01 CN CN201880049562.1A patent/CN111164091B/zh active Active
- 2018-06-01 JP JP2019566837A patent/JP2020522265A/ja active Pending
- 2018-06-01 TW TW107119085A patent/TW201908483A/zh unknown
- 2018-06-01 US US16/618,003 patent/US20210198305A1/en not_active Abandoned
- 2018-06-01 EP EP18809059.1A patent/EP3630788A4/en active Pending
- 2018-06-01 CN CN202411896095.XA patent/CN120330183A/zh active Pending
-
2022
- 2022-10-04 US US17/960,090 patent/US20240109931A1/en not_active Abandoned
-
2023
- 2023-08-01 JP JP2023125286A patent/JP2023153920A/ja active Pending