JP2019516680A5 - - Google Patents
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- JP2019516680A5 JP2019516680A5 JP2018557087A JP2018557087A JP2019516680A5 JP 2019516680 A5 JP2019516680 A5 JP 2019516680A5 JP 2018557087 A JP2018557087 A JP 2018557087A JP 2018557087 A JP2018557087 A JP 2018557087A JP 2019516680 A5 JP2019516680 A5 JP 2019516680A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- pharmaceutically acceptable
- smg
- composition
- acceptable salt
- 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 40
- 239000000203 mixture Substances 0.000 claims 13
- 150000003839 salts Chemical class 0.000 claims 9
- 239000008194 pharmaceutical composition Substances 0.000 claims 8
- 159000000000 sodium salts Chemical group 0.000 claims 7
- 238000000034 method Methods 0.000 claims 6
- 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 4
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 4
- 238000012986 modification Methods 0.000 claims 4
- 230000004048 modification Effects 0.000 claims 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical group [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 4
- 229910019142 PO4 Chemical group 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 239000010452 phosphate Chemical group 0.000 claims 3
- 229910001415 sodium ion Inorganic materials 0.000 claims 3
- 208000023105 Huntington disease Diseases 0.000 claims 2
- 229920002521 macromolecule Polymers 0.000 claims 2
- 108700028369 Alleles Proteins 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000004615 ingredient Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 229920000642 polymer Polymers 0.000 claims 1
- 230000007017 scission Effects 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662331960P | 2016-05-04 | 2016-05-04 | |
| US62/331,960 | 2016-05-04 | ||
| USPCT/US2016/043542 | 2016-07-22 | ||
| PCT/US2016/043542 WO2017015555A1 (en) | 2015-07-22 | 2016-07-22 | Oligonucleotide compositions and methods thereof |
| US201762447832P | 2017-01-18 | 2017-01-18 | |
| US62/447,832 | 2017-01-18 | ||
| PCT/US2017/030753 WO2017192664A1 (en) | 2016-05-04 | 2017-05-03 | Oligonucleotide compositions and methods thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019516680A JP2019516680A (ja) | 2019-06-20 |
| JP2019516680A5 true JP2019516680A5 (OSRAM) | 2020-06-18 |
Family
ID=60203309
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018557087A Pending JP2019516680A (ja) | 2016-05-04 | 2017-05-03 | オリゴヌクレオチド組成物およびその方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US10724035B2 (OSRAM) |
| EP (1) | EP3452596A4 (OSRAM) |
| JP (1) | JP2019516680A (OSRAM) |
| CN (1) | CN109477108A (OSRAM) |
| MA (1) | MA45270A (OSRAM) |
| WO (1) | WO2017192664A1 (OSRAM) |
Families Citing this family (73)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| 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 |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| WO2016097212A1 (en) | 2014-12-17 | 2016-06-23 | Proqr Therapeutics Ii B.V. | Targeted rna editing |
| 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 |
| EA201800367A1 (ru) | 2015-12-10 | 2019-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы лечения болезни хантингтона |
| 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 |
| SG10202012839TA (en) | 2016-12-19 | 2021-01-28 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| BR112019012664A2 (pt) | 2016-12-19 | 2020-01-21 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
| MD3554554T2 (ro) | 2016-12-19 | 2022-12-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| WO2018134301A1 (en) | 2017-01-19 | 2018-07-26 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in rna editing |
| 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 |
| KR20200033249A (ko) | 2017-06-05 | 2020-03-27 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
| WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| US11395822B2 (en) | 2017-06-28 | 2022-07-26 | Ptc Therapeutics, Inc. | Methods for treating Huntington's disease |
| CA3067591A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | Methods for treating huntington's disease |
| 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 |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536060A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| EP3687577A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| JP2020536057A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| WO2019075357A1 (en) | 2017-10-12 | 2019-04-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| SG11202006528XA (en) | 2018-01-12 | 2020-08-28 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| CN120310791A (zh) | 2018-01-12 | 2025-07-15 | 百时美施贵宝公司 | 靶向α-突触核蛋白的反义寡核苷酸及其用途 |
| US12103926B2 (en) | 2018-03-27 | 2024-10-01 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| 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 |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP3806868A4 (en) | 2018-06-13 | 2022-06-22 | Sarepta Therapeutics, Inc. | EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY |
| EA202092899A1 (ru) | 2018-06-27 | 2021-05-14 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Гетероарильные соединения для лечения болезни гентингтона |
| BR112020026545A2 (pt) | 2018-06-27 | 2021-03-23 | Ptc Therapeutics, Inc. | Compostos heterocíclicos e de heteroarila para o tratamento da doença de huntington |
| TW202020153A (zh) | 2018-07-27 | 2020-06-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| IL318474A (en) | 2018-12-13 | 2025-03-01 | Sarepta Therapeutics Inc | Axon-skipping oligomer complexes for muscular dystrophy |
| CA3126845A1 (en) * | 2019-02-01 | 2020-08-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| EP3955966A1 (en) | 2019-04-18 | 2022-02-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| MX2021012981A (es) * | 2019-04-25 | 2022-01-18 | Wave Life Sciences Ltd | Composiciones de oligonucleotido y metodos de uso de las mismas. |
| AU2020377204A1 (en) | 2019-11-01 | 2022-06-02 | Novartis Ag | The use of a splicing modulator for a treatment slowing progression of huntington's disease |
| 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 |
| WO2021254593A1 (en) * | 2020-06-15 | 2021-12-23 | Curevac Ag | Analysis of nucleic acid mixtures |
| TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
| EP4396354A4 (en) * | 2021-09-26 | 2025-12-03 | Wave Life Sciences Ltd | OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES |
| JP2024537711A (ja) | 2021-09-30 | 2024-10-16 | サレプタ セラピューティクス， インコーポレイテッド | １つ以上の脱塩基ユニットを有するアンチセンスオリゴヌクレオチド |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | 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 |
| EP4555085A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| WO2024064237A2 (en) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
| 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 |
| 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 |
| 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 |
| TW202442246A (zh) | 2023-04-27 | 2024-11-01 | 美商薩羅塔治療公司 | 用於治療慢性腎病之反義寡聚物 |
| 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 |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2025085810A2 (en) | 2023-10-18 | 2025-04-24 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of centronuclear myopathies |
| WO2025104239A1 (en) | 2023-11-16 | 2025-05-22 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of classic galactosemia |
| 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 (99)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5512668A (en) * | 1991-03-06 | 1996-04-30 | Polish Academy Of Sciences | Solid phase oligonucleotide synthesis using phospholane intermediates |
| US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
| WO1995014030A1 (en) | 1993-11-16 | 1995-05-26 | Genta Incorporated | Synthetic oligomers having chirally pure phosphonate internucleosidyl linkages mixed with non-phosphonate internucleosidyl linkages |
| US6172209B1 (en) | 1997-02-14 | 2001-01-09 | Isis Pharmaceuticals Inc. | Aminooxy-modified oligonucleotides and methods for making same |
| US6639062B2 (en) | 1997-02-14 | 2003-10-28 | Isis Pharmaceuticals, Inc. | Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom |
| US6242589B1 (en) * | 1998-07-14 | 2001-06-05 | Isis Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides having modified internucleoside linkages |
| US6867294B1 (en) | 1998-07-14 | 2005-03-15 | Isis Pharmaceuticals, Inc. | Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages |
| US20020082227A1 (en) | 1999-09-30 | 2002-06-27 | Scott Henry | Use of oligonucleotides for inhibition of complement activation |
| DE10019756A1 (de) | 2000-04-20 | 2001-10-25 | Bayer Ag | Verfahren zur Herstellung von superabsorbierenden Polymeren aus Polyacrylnitrilen |
| US20050277133A1 (en) | 2001-05-18 | 2005-12-15 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| 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 |
| US20050096284A1 (en) | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US7045306B2 (en) | 2003-04-28 | 2006-05-16 | The General Hospital Corporation | Method for identifying compounds in vitro that modulate the dysregulation of transcription of transcription mediated by mutant huntingtin protein |
| US7589189B2 (en) | 2003-05-14 | 2009-09-15 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| 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 |
| 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 |
| 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 |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| ES2969371T3 (es) * | 2003-09-12 | 2024-05-17 | Univ Massachusetts | Interferencia por ARN para el tratamiento de trastornos de ganancia de función |
| 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 |
| US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
| WO2006121960A2 (en) | 2005-05-06 | 2006-11-16 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
| US9133517B2 (en) | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| ATE522626T1 (de) | 2005-06-28 | 2011-09-15 | Medtronic Inc | Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken |
| EP2325315B1 (en) | 2005-10-28 | 2014-05-07 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of huntingtin gene |
| WO2007089611A2 (en) | 2006-01-26 | 2007-08-09 | Isis Pharmaceuticals Inc. | Compositions and their uses directed to huntingtin |
| EP2046993A4 (en) | 2006-07-07 | 2010-11-17 | Univ Massachusetts | RNA SILENCING COMPOSITIONS, AND METHODS OF TREATING HUNTINGTON CHOREA |
| EP2410053B2 (en) | 2006-10-18 | 2020-07-15 | Ionis Pharmaceuticals, Inc. | Antisense compounds |
| WO2008151833A2 (en) | 2007-06-13 | 2008-12-18 | Hochschule Mannheim | Compounds for the modulation of huntingtin aggregation, methods and means for identifying such compounds |
| EP2014769B1 (en) | 2007-06-18 | 2010-03-31 | Commissariat à l'Energie Atomique | Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease |
| EP2240768A1 (en) | 2008-02-04 | 2010-10-20 | Galapagos N.V. | Target sequences and methods to identify the same, useful in treatment of neurodegenerative diseases |
| CA2726866A1 (en) * | 2008-05-09 | 2009-11-12 | The University Of British Columbia | Methods and compositions for the treatment of huntington's disease |
| US8679750B2 (en) | 2008-05-09 | 2014-03-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington'S disease |
| WO2009143391A2 (en) | 2008-05-22 | 2009-11-26 | Isis Pharmaceuticals, Inc | Methods for modulation expression of creb |
| WO2009148605A2 (en) | 2008-06-04 | 2009-12-10 | Isis Pharmaceuticals, Inc. | Methods for treating hypercholesterolemia |
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| US8987222B2 (en) | 2009-04-08 | 2015-03-24 | University Of Massachusetts | Single nucleotide polymorphism (SNP) targeting therapies for the treatment of huntington'S disease |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| WO2011015572A1 (en) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases |
| WO2011015573A1 (en) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases |
| US8927553B2 (en) | 2009-08-10 | 2015-01-06 | Daljit Singh Dhanoa | Deuterium-enriched alkyl sulfonamides and uses thereof |
| EP3626823A1 (en) | 2009-09-11 | 2020-03-25 | Ionis Pharmaceuticals, Inc. | Modulation of huntingtin expression |
| ES2586683T3 (es) | 2009-09-16 | 2016-10-18 | Wave Life Sciences Japan, Inc. | Grupo protector novedoso para sintetizar ARN y derivados del mismo |
| CN102791862B (zh) | 2009-12-31 | 2017-04-05 | 库尔纳公司 | 通过抑制胰岛素受体底物2(irs2)和转录因子e3(tfe3)的天然反义转录物而治疗irs2相关疾病 |
| EP3208347B1 (en) | 2010-02-08 | 2019-08-14 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| EP3321361B1 (en) | 2010-02-08 | 2019-03-27 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| US8859755B2 (en) | 2010-03-05 | 2014-10-14 | Chiralgen, Ltd. | Method for preparing ribonucleoside phosphorothioate |
| JP2011184318A (ja) | 2010-03-05 | 2011-09-22 | Univ Of Tokyo | リボヌクレシドｈ−ボラノホスホネート |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| EP2638163B1 (en) | 2010-11-12 | 2017-05-17 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| WO2012073857A1 (ja) | 2010-11-30 | 2012-06-07 | 株式会社キラルジェン | ２'－ｏ－修飾ｒｎａ |
| WO2012109395A1 (en) | 2011-02-08 | 2012-08-16 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| EP2673286B1 (en) | 2011-02-12 | 2019-07-03 | University of Iowa Research Foundation | Therapeutic compounds |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| EP3205725B1 (en) | 2011-08-11 | 2019-03-27 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| WO2013082548A1 (en) | 2011-11-30 | 2013-06-06 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| 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 |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| US20150275208A1 (en) | 2012-10-12 | 2015-10-01 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| EP3778618A1 (en) | 2013-02-04 | 2021-02-17 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| BR112015027322A8 (pt) | 2013-05-01 | 2018-01-02 | Isis Pharmaceuticals Inc | Compostos antissenso conjugados e sua utilização |
| US10323076B2 (en) | 2013-10-03 | 2019-06-18 | Modernatx, Inc. | Polynucleotides encoding low density lipoprotein receptor |
| EP3068905A4 (en) | 2013-11-11 | 2017-07-05 | Sangamo BioSciences, Inc. | Methods and compositions for treating huntington's disease |
| 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 |
| 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 |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| US9926556B2 (en) | 2014-04-28 | 2018-03-27 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
| AU2015255877B2 (en) | 2014-05-08 | 2020-03-26 | Chdi Foundation, Inc. | Methods and compositions for treating huntington's disease |
| RU2711147C2 (ru) | 2014-05-20 | 2020-01-15 | Юниверсити Оф Айова Рисерч Фаундейшн | Терапевтические соединения для лечения болезни хантингтона |
| US20160017327A1 (en) | 2014-07-11 | 2016-01-21 | The Johns Hopkins University | Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity |
| WO2016011226A1 (en) | 2014-07-16 | 2016-01-21 | Moderna Therapeutics, Inc. | Chimeric polynucleotides |
| RU2708237C2 (ru) * | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| JP2017536366A (ja) | 2014-11-19 | 2017-12-07 | 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 | Ｌｎａキラルホスホロチオエート |
| EA037696B1 (ru) | 2014-12-24 | 2021-05-12 | ЮНИКЕР АйПи Б.В. | Супрессия гена гентингтина, индуцированная рнк-интерференцией |
| WO2016127002A1 (en) | 2015-02-04 | 2016-08-11 | Bristol-Myers Squibb Company | Lna oligonucleotides with alternating flanks |
| MA40819B1 (fr) | 2015-02-10 | 2020-04-30 | Genzyme Corp | Variant d'arni |
| PL3277814T3 (pl) | 2015-04-03 | 2020-11-30 | University Of Massachusetts | Związki oligonukleotydowe ukierunkowane na mrna huntingtyny |
| 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 |
| 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 |
| EP3646836B1 (en) | 2017-06-26 | 2021-10-06 | Yamaha Hatsudoki Kabushiki Kaisha | Power assist wheelchair, power assist unit for wheelchair, control device for power assist wheelchair, control method for power assist wheelchair, and program |
| 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 |
-
0
- MA MA045270A patent/MA45270A/fr unknown
-
2017
- 2017-05-03 EP EP17793215.9A patent/EP3452596A4/en not_active Withdrawn
- 2017-05-03 WO PCT/US2017/030753 patent/WO2017192664A1/en not_active Ceased
- 2017-05-03 US US16/098,836 patent/US10724035B2/en not_active Expired - Fee Related
- 2017-05-03 CN CN201780038913.4A patent/CN109477108A/zh active Pending
- 2017-05-03 JP JP2018557087A patent/JP2019516680A/ja active Pending
-
2020
- 2020-05-07 US US16/869,126 patent/US20220162598A1/en not_active Abandoned
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