JP2018525357A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018525357A5 JP2018525357A5 JP2018502647A JP2018502647A JP2018525357A5 JP 2018525357 A5 JP2018525357 A5 JP 2018525357A5 JP 2018502647 A JP2018502647 A JP 2018502647A JP 2018502647 A JP2018502647 A JP 2018502647A JP 2018525357 A5 JP2018525357 A5 JP 2018525357A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- oligonucleotide
- composition according
- pattern
- backbone
- 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
Links
- 239000000203 mixture Substances 0.000 claims 57
- 108091034117 Oligonucleotide Proteins 0.000 claims 40
- 238000000034 method Methods 0.000 claims 8
- 238000012986 modification Methods 0.000 claims 8
- 239000002773 nucleotide Substances 0.000 claims 7
- 125000003729 nucleotide group Chemical group 0.000 claims 7
- 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 6
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 150000007523 nucleic acids Chemical class 0.000 claims 6
- 102000039446 nucleic acids Human genes 0.000 claims 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 5
- 230000004048 modification Effects 0.000 claims 4
- 238000003776 cleavage reaction Methods 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 3
- 230000007017 scission Effects 0.000 claims 3
- 229910019142 PO4 Inorganic materials 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 239000002777 nucleoside Substances 0.000 claims 2
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 2
- 239000010452 phosphate Substances 0.000 claims 2
- 229920000642 polymer Polymers 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- YIMATHOGWXZHFX-WCTZXXKLSA-N (2r,3r,4r,5r)-5-(hydroxymethyl)-3-(2-methoxyethoxy)oxolane-2,4-diol Chemical group COCCO[C@H]1[C@H](O)O[C@H](CO)[C@H]1O YIMATHOGWXZHFX-WCTZXXKLSA-N 0.000 claims 1
- 108700028369 Alleles Proteins 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 102100034343 Integrase Human genes 0.000 claims 1
- 101710203526 Integrase Proteins 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000002619 bicyclic group Chemical group 0.000 claims 1
- 210000001175 cerebrospinal fluid Anatomy 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
- 230000001404 mediated effect Effects 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 159000000000 sodium salts Chemical class 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021170541A JP7606954B2 (ja) | 2015-07-22 | 2021-10-18 | オリゴヌクレオチド組成物およびその方法 |
| JP2024219444A JP2025038091A (ja) | 2015-07-22 | 2024-12-13 | オリゴヌクレオチド組成物およびその方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562195779P | 2015-07-22 | 2015-07-22 | |
| US62/195,779 | 2015-07-22 | ||
| US201562236847P | 2015-10-02 | 2015-10-02 | |
| US62/236,847 | 2015-10-02 | ||
| US201662331960P | 2016-05-04 | 2016-05-04 | |
| US62/331,960 | 2016-05-04 | ||
| PCT/US2016/043542 WO2017015555A1 (en) | 2015-07-22 | 2016-07-22 | Oligonucleotide compositions and methods thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021170541A Division JP7606954B2 (ja) | 2015-07-22 | 2021-10-18 | オリゴヌクレオチド組成物およびその方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018525357A JP2018525357A (ja) | 2018-09-06 |
| JP2018525357A5 true JP2018525357A5 (https=) | 2019-09-05 |
Family
ID=57835219
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018502637A Active JP7296724B2 (ja) | 2015-07-22 | 2016-07-22 | オリゴヌクレオチド組成物およびその方法 |
| JP2018502647A Pending JP2018525357A (ja) | 2015-07-22 | 2016-07-22 | オリゴヌクレオチド組成物およびその方法 |
| JP2021134582A Active JP7441816B2 (ja) | 2015-07-22 | 2021-08-20 | オリゴヌクレオチド組成物およびその方法 |
| JP2021170541A Active JP7606954B2 (ja) | 2015-07-22 | 2021-10-18 | オリゴヌクレオチド組成物およびその方法 |
| JP2023207761A Active JP7818563B2 (ja) | 2015-07-22 | 2023-12-08 | オリゴヌクレオチド組成物およびその方法 |
| JP2024219444A Pending JP2025038091A (ja) | 2015-07-22 | 2024-12-13 | オリゴヌクレオチド組成物およびその方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018502637A Active JP7296724B2 (ja) | 2015-07-22 | 2016-07-22 | オリゴヌクレオチド組成物およびその方法 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021134582A Active JP7441816B2 (ja) | 2015-07-22 | 2021-08-20 | オリゴヌクレオチド組成物およびその方法 |
| JP2021170541A Active JP7606954B2 (ja) | 2015-07-22 | 2021-10-18 | オリゴヌクレオチド組成物およびその方法 |
| JP2023207761A Active JP7818563B2 (ja) | 2015-07-22 | 2023-12-08 | オリゴヌクレオチド組成物およびその方法 |
| JP2024219444A Pending JP2025038091A (ja) | 2015-07-22 | 2024-12-13 | オリゴヌクレオチド組成物およびその方法 |
Country Status (23)
| Country | Link |
|---|---|
| US (6) | US10479995B2 (https=) |
| EP (3) | EP3325017A4 (https=) |
| JP (6) | JP7296724B2 (https=) |
| KR (1) | KR20180028516A (https=) |
| CN (3) | CN108135921B (https=) |
| AU (3) | AU2016297155B2 (https=) |
| BR (1) | BR112017028636A2 (https=) |
| CA (1) | CA2989682A1 (https=) |
| CL (1) | CL2018000145A1 (https=) |
| CO (1) | CO2018001484A2 (https=) |
| CR (1) | CR20180107A (https=) |
| EC (1) | ECSP18010463A (https=) |
| HK (2) | HK1255369A1 (https=) |
| IL (2) | IL320434A (https=) |
| MA (1) | MA43072A (https=) |
| MX (2) | MX2018000796A (https=) |
| NI (1) | NI201800009A (https=) |
| PE (1) | PE20181174A1 (https=) |
| PH (1) | PH12018500145A1 (https=) |
| SG (1) | SG10201912900XA (https=) |
| SV (1) | SV2018005619A (https=) |
| TW (1) | TW201707711A (https=) |
| WO (2) | WO2017015555A1 (https=) |
Families Citing this family (160)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8158768B2 (en) * | 2002-12-23 | 2012-04-17 | Dynavax Technologies Corporation | Immunostimulatory sequence oligonucleotides and methods of using the same |
| RU2572826C2 (ru) | 2008-12-02 | 2016-01-20 | Чиралджен, Лтд. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| AU2012284265B2 (en) | 2011-07-19 | 2017-08-17 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| AU2013203395A1 (en) | 2012-03-30 | 2013-10-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| JP6453212B2 (ja) | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | キラル制御 |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ma公司 | 用於調節τ蛋白表現之組合物 |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| 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 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| CN107208092B (zh) * | 2014-12-16 | 2021-09-10 | 罗氏创新中心哥本哈根有限公司 | 手性毒性筛选方法 |
| US10676737B2 (en) | 2014-12-17 | 2020-06-09 | Proqr Therapeutics Ii B.V. | Targeted RNA editing |
| US11129844B2 (en) | 2015-03-03 | 2021-09-28 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating MECP2 expression |
| SMT202000454T1 (it) | 2015-04-03 | 2020-11-10 | Univ Massachusetts | Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina |
| EP3277811B1 (en) | 2015-04-03 | 2020-12-23 | University of Massachusetts | Fully stabilized asymmetric sirna |
| CN108135921B (zh) | 2015-07-22 | 2023-10-17 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| JP7511326B2 (ja) | 2015-10-09 | 2024-07-05 | 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およびその方法 |
| EA201800367A1 (ru) | 2015-12-10 | 2019-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы лечения болезни хантингтона |
| WO2017132669A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| CA3015823A1 (en) | 2016-03-13 | 2017-09-21 | 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 |
| CN109562122A (zh) | 2016-06-03 | 2019-04-02 | 波涛生命科学有限公司 | 寡核苷酸、组合物及其方法 |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| CA3033368A1 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| US10941402B2 (en) | 2016-09-01 | 2021-03-09 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded RNA-editing oligonucleotides |
| ES2963428T3 (es) | 2016-09-29 | 2024-03-27 | Biogen Ma Inc | Compuestos y métodos para reducir la expresión de Tau |
| JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
| MA46905A (fr) | 2016-11-23 | 2019-10-02 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides |
| SMT202200366T1 (it) | 2016-12-19 | 2022-11-18 | Sarepta Therapeutics Inc | Coniugati di oligomeri per salto di esone per distrofia muscolare |
| SI3554554T1 (sl) | 2016-12-19 | 2023-02-28 | Sarepta Therapeutics, Inc. | Oligomerni konjugati, ki preskakujejo ekson za mišično distrofijo |
| IL297528A (en) | 2016-12-19 | 2022-12-01 | Sarepta Therapeutics Inc | Exon-skipping oligomer conjugates for muscular dystrophy |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| WO2018145009A1 (en) * | 2017-02-06 | 2018-08-09 | University Of Tennessee Research Foundation | Dna-zyme based methods & compositions for treating huntington's disease |
| GB2574769A (en) | 2017-03-03 | 2019-12-18 | Univ California | RNA Targeting of mutations via suppressor tRNAs and deaminases |
| US11597927B2 (en) | 2017-06-02 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
| US11718638B2 (en) | 2017-06-21 | 2023-08-08 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| JP7406793B2 (ja) | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | 2テイル自己デリバリー型siRNAおよび関連方法 |
| CN111182898B (zh) | 2017-06-28 | 2024-04-16 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| 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 |
| CN111108096A (zh) | 2017-09-18 | 2020-05-05 | 波涛生命科学有限公司 | 寡核苷酸制备技术 |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536058A (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 |
| WO2019067975A1 (en) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| WO2019067979A1 (en) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| EP3694530A4 (en) | 2017-10-12 | 2021-06-30 | Wave Life Sciences Ltd. | COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES |
| TWI809004B (zh) | 2017-11-09 | 2023-07-21 | 美商Ionis製藥公司 | 用於降低snca表現之化合物及方法 |
| EA202091693A1 (ru) | 2018-01-12 | 2021-04-14 | Бристол-Маерс Сквибб Компани | Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения |
| BR112020013994A2 (pt) | 2018-01-12 | 2020-12-08 | Bristol-Myers Squibb Company | Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos |
| BR112020014425A2 (pt) | 2018-01-15 | 2020-12-29 | Ionis Pharmaceuticals, Inc. | Moduladores de expressão de dnm2 |
| AU2019218987B2 (en) | 2018-02-12 | 2025-04-24 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| US11661601B2 (en) | 2018-03-22 | 2023-05-30 | Ionis Pharmaceuticals, Inc. | Methods for modulating FMR1 expression |
| EA202092001A1 (ru) | 2018-03-27 | 2021-01-29 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения болезни гентингтона |
| IL277889B2 (en) | 2018-04-12 | 2025-01-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods of using them |
| WO2019217459A1 (en) | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| SG11202010215TA (en) | 2018-05-09 | 2020-11-27 | Ionis Pharmaceuticals Inc | Compounds and methods for reducing atxn3 expression |
| JP7438135B2 (ja) | 2018-05-09 | 2024-02-26 | 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 | Fxiの発現を低下させるための化合物及び方法 |
| EP3790596A4 (en) * | 2018-05-11 | 2022-04-06 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE |
| GB201808146D0 (en) * | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| EP3802821A4 (en) * | 2018-05-25 | 2022-06-08 | New York Institute of Technology | DIRECT NUCLEIC ACID SEQUENCING METHOD |
| 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 |
| CA3103429A1 (en) * | 2018-06-14 | 2019-12-19 | Don W. Cleveland | Compounds and methods for increasing stmn2 expression |
| WO2020005877A1 (en) | 2018-06-27 | 2020-01-02 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| HRP20240935T1 (hr) | 2018-06-27 | 2024-11-22 | Ptc Therapeutics, Inc. | Heterociklični i heteroaril spojevi za liječenje huntingtonove bolesti |
| CN109030440B (zh) * | 2018-07-18 | 2020-12-04 | 西北农林科技大学 | 一种基于三氧化钼量子点检测单宁酸含量的方法 |
| BR112021000308A2 (pt) | 2018-07-25 | 2021-04-13 | Ionis Pharmaceuticals, Inc. | Compostos e métodos para redução da expressão de atxn2 |
| 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 |
| WO2020033899A1 (en) | 2018-08-10 | 2020-02-13 | University Of Massachusetts | Modified oligonucleotides targeting snps |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | O-メチルリッチ完全安定化オリゴヌクレオチド |
| TWI856973B (zh) | 2018-09-19 | 2024-10-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
| WO2020072883A1 (en) * | 2018-10-05 | 2020-04-09 | Ionis Pharmaceuticals, Inc. | Chirally enriched oligomeric compounds |
| BR112021008967A2 (pt) | 2018-11-15 | 2021-08-17 | Ionis Pharmaceuticals, Inc. | moduladores da expressão de irf5 |
| SG11202103794QA (en) | 2018-11-21 | 2021-05-28 | Ionis Pharmaceuticals Inc | Compounds and methods for reducing prion expression |
| WO2020117706A1 (en) | 2018-12-03 | 2020-06-11 | Triplet Therapeutics, Inc. | Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity |
| MX2021006737A (es) | 2018-12-13 | 2021-09-23 | Sarepta Therapeutics Inc | Conjugados de oligómeros de salto de exones para distrofia muscular. |
| EP3899024A4 (en) | 2018-12-21 | 2023-05-31 | Ionis Pharmaceuticals, Inc. | Modulators of hsd17b13 expression |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| KR20210121199A (ko) * | 2019-02-01 | 2021-10-07 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 방법 |
| TW202540426A (zh) | 2019-02-27 | 2025-10-16 | 美商Ionis製藥公司 | Malat1表現之調節劑 |
| CN113748116A (zh) | 2019-03-20 | 2021-12-03 | 波涛生命科学有限公司 | 可用于寡核苷酸制备的技术 |
| FI3947684T3 (fi) | 2019-03-29 | 2025-05-27 | Ionis Pharmaceuticals Inc | Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi |
| EP3955966A1 (en) | 2019-04-18 | 2022-02-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| CA3137741A1 (en) * | 2019-04-25 | 2020-10-29 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP3958872A4 (en) * | 2019-04-25 | 2024-07-03 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| JP7649257B2 (ja) | 2019-05-13 | 2025-03-19 | ピーティーシー セラピューティクス, インコーポレイテッド | ハンチントン病を処置するための化合物 |
| WO2020243292A1 (en) | 2019-05-28 | 2020-12-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing fus expression |
| EP3956450B1 (en) | 2019-07-26 | 2025-08-13 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating gfap |
| MX2022001710A (es) | 2019-08-09 | 2022-05-10 | Univ Massachusetts | Oligonucleótidos modificados químicamente dirigidos a los snp. |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| EP4045062A1 (en) | 2019-10-14 | 2022-08-24 | Astrazeneca AB | Modulators of pnpla3 expression |
| TW202131920A (zh) | 2019-11-01 | 2021-09-01 | 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 |
| WO2021113270A1 (en) | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Therapeutic editing |
| BR112022016370A2 (pt) * | 2020-02-21 | 2022-10-04 | Ionis Pharmaceuticals Inc | Métodos para reduzir a expressão de htt |
| CR20220485A (es) | 2020-02-28 | 2022-11-10 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular smn2 |
| AU2021246024A1 (en) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals, Inc. | Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof |
| AU2021260934A1 (en) | 2020-04-21 | 2022-11-24 | Flagship Pioneering, Inc. | Bifunctional molecules and methods of using thereof |
| 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 |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| AR122534A1 (es) | 2020-06-03 | 2022-09-21 | Triplet Therapeutics Inc | Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3 |
| TW202227102A (zh) | 2020-09-22 | 2022-07-16 | 瑞典商阿斯特捷利康公司 | 治療脂肪肝病之方法 |
| EP4136092B1 (en) | 2020-11-18 | 2024-07-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating angiotensinogen expression |
| GB2603454A (en) | 2020-12-09 | 2022-08-10 | Ucl Business Ltd | Novel therapeutics for the treatment of neurodegenerative disorders |
| PE20240696A1 (es) | 2021-03-08 | 2024-04-10 | Servier Lab | Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina |
| 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 |
| CN113122615B (zh) * | 2021-05-24 | 2024-07-09 | 湖南赛哲智造科技有限公司 | 一种应用于绝对定量高通量测序的多重pcr扩增的单分子标签引物及其应用 |
| IL309445A (en) | 2021-06-22 | 2024-02-01 | Acurastem Inc | PIKFYVE antisense oligonucleotides |
| AU2022299169A1 (en) | 2021-06-23 | 2024-02-08 | Beth Israel Deaconess Medical Center, Inc. | Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders |
| CA3227115A1 (en) | 2021-07-21 | 2023-01-26 | Wen-Hsuan Chang | Unc13a antisense oligonucleotides |
| MX2024003690A (es) | 2021-09-30 | 2024-06-19 | Sarepta Therapeutics Inc | Oligonucleótidos antisentido que tienen una o más unidades abásicas. |
| MX2024004011A (es) | 2021-10-01 | 2024-07-01 | Adarx Pharmaceuticals Inc | Composiciones moduladoras de precalicreína y métodos de uso de estas. |
| GB202117758D0 (en) | 2021-12-09 | 2022-01-26 | Ucl Business Ltd | Therapeutics for the treatment of neurodegenerative disorders |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2023212625A1 (en) | 2022-04-28 | 2023-11-02 | AcuraStem Incorporated | Syf2 antisense oligonucleotides |
| EP4555086A1 (en) | 2022-07-15 | 2025-05-21 | 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 |
| JP2025526712A (ja) * | 2022-08-11 | 2025-08-15 | 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 |
| WO2024064237A2 (en) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
| JP2025532127A (ja) | 2022-09-23 | 2025-09-29 | 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 | Mecp2発現を低減する化合物及び方法 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| AR131145A1 (es) | 2022-11-24 | 2025-02-19 | Proqr Therapeutics Ii Bv | Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria |
| 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 B.V. | Antisense oligonucleotides for the treatment of cardiovascular disease |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| WO2024155986A2 (en) | 2023-01-20 | 2024-07-25 | AcuraStem Incorporated | Unc13a antisense oligonucleotides |
| AU2024215901A1 (en) | 2023-01-31 | 2025-07-24 | AcuraStem Incorporated | Syf2 antisense oligonucleotides |
| KR20250143312A (ko) | 2023-02-06 | 2025-10-01 | 아큐라스템 인코포레이티드 | Pikfyve 안티센스 올리고뉴클레오타이드 |
| EP4669753A1 (en) | 2023-02-20 | 2025-12-31 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| WO2024206175A1 (en) | 2023-03-24 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| AU2024246572A1 (en) | 2023-03-27 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| WO2024220930A2 (en) | 2023-04-20 | 2024-10-24 | Adarx Pharmaceuticals, Inc. | Mapt-modulating compositions and methods of use thereof |
| US20250066776A1 (en) | 2023-04-27 | 2025-02-27 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of chronic kidney disease |
| CN121398851A (zh) | 2023-05-12 | 2026-01-23 | 阿达尔克斯制药有限公司 | Nmda配体缀合的化合物及其用途 |
| IL324720A (en) | 2023-05-26 | 2026-01-01 | Adarx Pharmaceuticals Inc | SOD1 modulators and methods of using them |
| WO2024256620A1 (en) | 2023-06-16 | 2024-12-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurodegenerative disease |
| CN121712891A (zh) | 2023-06-20 | 2026-03-20 | 阿达尔克斯制药有限公司 | Lrrk2调节组合物及其使用方法 |
| AU2024287308A1 (en) | 2023-07-13 | 2025-12-18 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| WO2025036916A1 (en) | 2023-08-16 | 2025-02-20 | Les Laboratoires Servier | Oligonucleotides for modulating kcnt1 expression |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| TW202526017A (zh) | 2023-09-14 | 2025-07-01 | 美商Ionis製藥公司 | 用於減少apociii表現的化合物及方法 |
| WO2025064821A2 (en) | 2023-09-21 | 2025-03-27 | Ionis Pharmaceuticals, Inc. | Compounds and methods for inhibiting lpa |
| GB202314724D0 (en) | 2023-09-26 | 2023-11-08 | Astrazeneca Ab | compounds and methods for reducing psd3 expression |
| WO2025085810A2 (en) | 2023-10-18 | 2025-04-24 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of centronuclear myopathies |
| CN121909203A (zh) | 2023-10-31 | 2026-04-21 | 科罗生物公司 | 包含氨基磷酸酯核苷酸间键的寡核苷酸 |
| 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 |
| 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 |
| WO2026062247A1 (en) | 2024-09-20 | 2026-03-26 | Astrazeneca Ab | Liquid-phase oligonucleotide synthesis using 2-(2-nitrophenyl)propyloxycarbonyl (nppoc) as a protecting group |
| WO2026068781A1 (en) | 2024-09-30 | 2026-04-02 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
Family Cites Families (211)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US513030A (en) | 1894-01-16 | Machine for waxing or coating paper | ||
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
| US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
| US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
| US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
| DE3329892A1 (de) | 1983-08-18 | 1985-03-07 | Köster, Hubert, Prof. Dr., 2000 Hamburg | Verfahren zur herstellung von oligonucleotiden |
| US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
| US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
| FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
| US4659774A (en) | 1985-11-01 | 1987-04-21 | American Hoechst Corporation | Support for solid-phase oligonucleotide synthesis |
| US5130300A (en) | 1986-03-07 | 1992-07-14 | Monsanto Company | Method for enhancing growth of mammary parenchyma |
| AU598946B2 (en) | 1987-06-24 | 1990-07-05 | Howard Florey Institute Of Experimental Physiology And Medicine | Nucleoside derivatives |
| US4923901A (en) | 1987-09-04 | 1990-05-08 | Millipore Corporation | Membranes with bound oligonucleotides and peptides |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| US5047524A (en) | 1988-12-21 | 1991-09-10 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5262530A (en) | 1988-12-21 | 1993-11-16 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5141813A (en) | 1989-08-28 | 1992-08-25 | Clontech Laboratories, Inc. | Multifunctional controlled pore glass reagent for solid phase oligonucleotide synthesis |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| CA2029273A1 (en) | 1989-12-04 | 1991-06-05 | Christine L. Brakel | Modified nucleotide compounds |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5212295A (en) | 1990-01-11 | 1993-05-18 | Isis Pharmaceuticals | Monomers for preparation of oligonucleotides having chiral phosphorus linkages |
| BR9106702A (pt) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals Inc | Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| 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 |
| US20020183502A1 (en) | 1991-05-21 | 2002-12-05 | Mesmaeker Alain De | Backbone-modified oligonucleotide analogs and methods for using same |
| DE69232699T2 (de) | 1991-10-15 | 2003-02-06 | Isis Pharmaceutical, Inc. | Über chirale phosphoratome gebundene oligonukleotide |
| EP0538194B1 (de) | 1991-10-17 | 1997-06-04 | Novartis AG | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
| US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| US6235887B1 (en) | 1991-11-26 | 2001-05-22 | Isis Pharmaceuticals, Inc. | Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines |
| US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
| KR940703846A (ko) | 1991-12-24 | 1994-12-12 | 비. 린네 파샬 | 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides) |
| US6015886A (en) | 1993-05-24 | 2000-01-18 | Chemgenes Corporation | Oligonucleotide phosphate esters |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| AU678085B2 (en) | 1993-11-16 | 1997-05-15 | Genta Incorporated | Synthetic oligomers having chirally pure phosphonate internucleosidyl linkages mixed with non-phosphonate internucleosidyl linkages |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| US6166197A (en) | 1995-03-06 | 2000-12-26 | Isis Pharmaceuticals, Inc. | Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions |
| AU5359496A (en) | 1995-03-06 | 1996-09-23 | Isis Pharmaceuticals, Inc. | Improved process for the synthesis of 2'-o-substituted pyrimidines and oligomeric compounds therefrom |
| 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 |
| AU8512598A (en) | 1997-07-25 | 1999-02-16 | Hybridon, Inc. | Oligonuclotides having 3' terminal stereospecific phosphorothioates |
| US6617438B1 (en) | 1997-11-05 | 2003-09-09 | Sirna Therapeutics, Inc. | Oligoribonucleotides with enzymatic activity |
| US6528640B1 (en) | 1997-11-05 | 2003-03-04 | Ribozyme Pharmaceuticals, Incorporated | Synthetic ribonucleic acids with RNAse activity |
| BR9909220A (pt) * | 1998-02-20 | 2000-11-21 | Tanox Inc | Inibidores de ativação de complemento |
| US7045610B2 (en) | 1998-04-03 | 2006-05-16 | Epoch Biosciences, Inc. | Modified oligonucleotides for mismatch discrimination |
| US6867294B1 (en) | 1998-07-14 | 2005-03-15 | Isis Pharmaceuticals, Inc. | Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages |
| US6242589B1 (en) * | 1998-07-14 | 2001-06-05 | Isis Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides having modified internucleoside linkages |
| US6066500A (en) | 1999-06-25 | 2000-05-23 | Isis Pharmaceuticals Inc. | Antisense modulation of Beta catenin expression |
| US6147200A (en) | 1999-08-19 | 2000-11-14 | Isis Pharmaceuticals, Inc. | 2'-O-acetamido modified monomers and oligomers |
| US6949520B1 (en) | 1999-09-27 | 2005-09-27 | Coley Pharmaceutical Group, Inc. | Methods related to immunostimulatory nucleic acid-induced interferon |
| US20020082227A1 (en) | 1999-09-30 | 2002-06-27 | Scott Henry | Use of oligonucleotides for inhibition of complement activation |
| US20010055761A1 (en) | 1999-10-29 | 2001-12-27 | Agilent Technologies | Small scale dna synthesis using polymeric solid support with functionalized regions |
| DE10019756A1 (de) | 2000-04-20 | 2001-10-25 | Bayer Ag | Verfahren zur Herstellung von superabsorbierenden Polymeren aus Polyacrylnitrilen |
| US6492171B2 (en) | 2000-05-16 | 2002-12-10 | Isis Pharmaceuticals, Inc. | Antisense modulation of TERT expression |
| GB0024752D0 (en) | 2000-10-10 | 2000-11-22 | Univ Belfast | Oxidative halogenation of aromatic compounds |
| 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) |
| US6440739B1 (en) | 2001-07-17 | 2002-08-27 | Isis Pharmaceuticals, Inc. | Antisense modulation of glioma-associated oncogene-2 expression |
| 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) |
| US8232383B2 (en) | 2002-02-20 | 2012-07-31 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| WO2003085110A2 (en) | 2002-04-05 | 2003-10-16 | Santaris Pharma A/S | Oligomeric compounds for the modulation hif-1alpha expression |
| WO2004007718A2 (en) | 2002-07-10 | 2004-01-22 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Rna-interference by single-stranded rna molecules |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion 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 |
| WO2004101787A1 (ja) | 2003-05-14 | 2004-11-25 | Japan Science And Technology Agency | ハンチンチン遺伝子の発現抑制 |
| 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 |
| EP1661905B9 (en) | 2003-08-28 | 2012-12-19 | IMANISHI, Takeshi | Novel artificial nucleic acids of n-o bond crosslinkage type |
| 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 |
| 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 |
| WO2005027980A1 (en) | 2003-09-12 | 2005-03-31 | University Of Massachusetts | Rna interference for the treatment of gain-of-function disorders |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| 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 |
| EP1730281A2 (en) | 2004-04-02 | 2006-12-13 | Coley Pharmaceutical Group, Inc. | Immunostimulatory nucleic acids for inducing il-10 responses |
| EP2397563A3 (en) | 2004-09-17 | 2012-07-18 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| WO2006066260A2 (en) | 2004-12-17 | 2006-06-22 | Thiosense, Inc. | Compositions of and methods for producing phosphorus-chiral monomers and oligomers |
| US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
| EP1885854B1 (en) | 2005-05-06 | 2012-10-17 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
| WO2007002904A2 (en) | 2005-06-28 | 2007-01-04 | Medtronic, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| US9133517B2 (en) | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| WO2007028065A2 (en) | 2005-08-30 | 2007-03-08 | Isis Pharmaceuticals, Inc. | Chimeric oligomeric compounds for modulation of splicing |
| WO2007051045A2 (en) * | 2005-10-28 | 2007-05-03 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of huntingtin gene |
| PT2161038E (pt) | 2006-01-26 | 2014-03-10 | Isis Pharmaceuticals Inc | Composições e suas utilizações dirigidas à huntingtina |
| JP5342881B2 (ja) | 2006-01-27 | 2013-11-13 | 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 | 6−修飾された二環式核酸類似体 |
| EP2023940B1 (en) | 2006-05-05 | 2011-06-22 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression of sglt2 |
| AU2007249349B2 (en) | 2006-05-11 | 2012-03-08 | Isis Pharmaceuticals, Inc. | 5'-Modified bicyclic nucleic acid analogs |
| EP2034015B1 (en) | 2006-05-31 | 2012-07-11 | Toray Industries, Inc. | Immunostimulatory oligonucleotide and pharmaceutical application thereof |
| EP2046993A4 (en) | 2006-07-07 | 2010-11-17 | Univ Massachusetts | RNA SILENCING COMPOSITIONS, AND METHODS OF TREATING HUNTINGTON CHOREA |
| WO2008049085A1 (en) | 2006-10-18 | 2008-04-24 | Isis 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 |
| ATE462787T1 (de) | 2007-06-18 | 2010-04-15 | Commissariat Energie Atomique | Reversibles sirna-silencing eines mutierten und endogenen huntington-wildtypgens und dessen anwendung zur behandlung von morbus huntington |
| CA2692161C (en) | 2007-07-09 | 2015-09-29 | Idera Pharmaceuticals, Inc. | Stabilized immune modulatory rna (simra) compounds |
| WO2009086558A1 (en) * | 2008-01-02 | 2009-07-09 | Tekmira Pharmaceuticals Corporation | Improved compositions and methods for the delivery of nucleic acids |
| 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 |
| PL2320925T3 (pl) | 2008-07-10 | 2016-06-30 | Regenesance B V | Antagoniści dopełniacza i ich zastosowania |
| RU2572826C2 (ru) | 2008-12-02 | 2016-01-20 | Чиралджен, Лтд. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| WO2010091301A1 (en) | 2009-02-06 | 2010-08-12 | Isis Pharmaceuticals, Inc. | Oligomeric compounds and excipients |
| WO2010118263A1 (en) | 2009-04-08 | 2010-10-14 | University Of Massachusetts | Single-nucleotide polymorphism (snp) targeting therapies for the treatment of huntington's disease |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| 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 |
| 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 |
| 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 |
| EP2479182B8 (en) | 2009-09-16 | 2016-07-13 | Wave Life Sciences Japan, Inc. | Novel protecting group for synthesizing rna and derivative thereof |
| DK2519632T3 (en) | 2009-12-31 | 2018-07-23 | Curna Inc | TREATMENT OF INSULIN RECEPTOR SUBSTRATE 2- (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPTION TO IRS2 AND TRANSCRIPTION FACTOR E3 (TFE3) |
| EP3628750A1 (en) | 2010-02-08 | 2020-04-01 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| WO2011097643A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| 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 |
| US8859755B2 (en) | 2010-03-05 | 2014-10-14 | Chiralgen, Ltd. | Method for preparing ribonucleoside phosphorothioate |
| RU2664452C2 (ru) | 2010-04-19 | 2018-08-17 | Нлифе Терапеутикс, С.Л. | Конъюгат, медицинское средство и способы лечения и/или профилактики депрессии и болезни, связанной с отложением телец Леви |
| EA201390145A1 (ru) * | 2010-08-20 | 2013-11-29 | Серулин Фарма Инк. | Конъюгаты, частицы, композиции и связанные с ними способы |
| CN103221549A (zh) | 2010-08-31 | 2013-07-24 | 默沙东公司 | 用于递送寡核苷酸的新型简单化学实体和方法 |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| EP3260540A1 (en) | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| JP6093924B2 (ja) | 2010-11-30 | 2017-03-15 | 株式会社Wave Life Sciences Japan | 2’−o−修飾rna |
| US10017764B2 (en) | 2011-02-08 | 2018-07-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| ES2739804T3 (es) | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
| RU2013151301A (ru) * | 2011-04-20 | 2015-05-27 | Ларри Дж. СМИТ | Способы и композиции для модулирования экспрессии генов с использованием компонентов, которые обладают способностью к самосборке в клетках и обусловливают рнкi-активность |
| AU2012284265B2 (en) | 2011-07-19 | 2017-08-17 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| ES2651514T3 (es) | 2011-08-11 | 2018-01-26 | Ionis Pharmaceuticals, Inc. | Compuestos oligoméricos gapados que comprenden deoxirribonucleósidos modificados en 5 ' -en la brecha y usos de ellos |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| CN112251436A (zh) * | 2012-01-27 | 2021-01-22 | 比奥马林技术公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| JP6453212B2 (ja) | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | キラル制御 |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CN104684923B (zh) * | 2012-07-13 | 2018-09-28 | 株式会社新日本科学 | 手性核酸佐剂 |
| EP3459549B1 (en) | 2012-10-12 | 2022-04-06 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| MX2015005328A (es) | 2012-10-26 | 2015-09-25 | Nlife Therapeutics S L | Composiciones y metodos para la administracion selectiva de moleculas de oligonucleotidos a tipos de celulas. |
| AU2013336581A1 (en) | 2012-10-26 | 2015-06-11 | Nlife Therapeutics, S.L. | Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
| CN104955952A (zh) | 2013-01-30 | 2015-09-30 | 弗·哈夫曼-拉罗切有限公司 | Lna寡核苷酸碳水化合物缀合物 |
| ES2817050T3 (es) | 2013-02-04 | 2021-04-06 | Ionis Pharmaceuticals Inc | Compuestos antisentido selectivos y usos de los mismos |
| DK2991656T3 (da) | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| JP2016538829A (ja) | 2013-10-03 | 2016-12-15 | モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. | 低密度リポタンパク質受容体をコードするポリヌクレオチド |
| WO2015070212A1 (en) | 2013-11-11 | 2015-05-14 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| 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 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| DK3137476T3 (da) | 2014-04-28 | 2019-11-18 | Ionis Pharmaceuticals Inc | Linker-modificerede oligomerforbindelser |
| RU2691102C2 (ru) | 2014-05-08 | 2019-06-11 | Сангамо Байосайенсиз, Инк. | Способы и композиции для лечения болезни хантингтона |
| JP6663859B2 (ja) | 2014-05-20 | 2020-03-13 | ユニバーシティー オブ アイオワ リサーチ ファウンデーション | ハンチントン病の治療化合物 |
| 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 |
| EP3169693B1 (en) * | 2014-07-16 | 2022-03-09 | ModernaTX, Inc. | Chimeric polynucleotides |
| 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 |
| CN107208092B (zh) | 2014-12-16 | 2021-09-10 | 罗氏创新中心哥本哈根有限公司 | 手性毒性筛选方法 |
| AU2015370903B2 (en) | 2014-12-24 | 2021-06-17 | Uniqure Ip B.V. | RNAi induced huntingtin gene suppression |
| WO2016127002A1 (en) | 2015-02-04 | 2016-08-11 | Bristol-Myers Squibb Company | Lna oligonucleotides with alternating flanks |
| EP3254104B1 (en) | 2015-02-04 | 2021-08-25 | Bristol-Myers Squibb Company | Methods of selecting therapeutic molecules |
| TN2017000354A1 (en) | 2015-02-10 | 2019-01-16 | Genzyme Corp | VARIANT RNAi |
| SMT202000454T1 (it) | 2015-04-03 | 2020-11-10 | Univ Massachusetts | Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina |
| CN108135921B (zh) | 2015-07-22 | 2023-10-17 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| JP7511326B2 (ja) | 2015-10-09 | 2024-07-05 | 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およびその方法 |
| EP3394258B1 (en) | 2015-10-22 | 2021-09-22 | Roche Innovation Center Copenhagen A/S | In vitro toxicity screening assay |
| CA3015823A1 (en) | 2016-03-13 | 2017-09-21 | 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 |
| WO2017192679A1 (en) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd. | Methods and compositions of biologically active agents |
| CN109562122A (zh) | 2016-06-03 | 2019-04-02 | 波涛生命科学有限公司 | 寡核苷酸、组合物及其方法 |
| WO2018022473A1 (en) | 2016-07-25 | 2018-02-01 | 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 |
| 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 |
| US11597927B2 (en) | 2017-06-02 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US11718638B2 (en) | 2017-06-21 | 2023-08-08 | 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 |
| CN111108096A (zh) | 2017-09-18 | 2020-05-05 | 波涛生命科学有限公司 | 寡核苷酸制备技术 |
| EP3694530A4 (en) | 2017-10-12 | 2021-06-30 | Wave Life Sciences Ltd. | COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES |
| IL277889B2 (en) | 2018-04-12 | 2025-01-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods of using them |
| EP3790596A4 (en) | 2018-05-11 | 2022-04-06 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE |
| SG11202105626TA (en) | 2018-12-06 | 2021-06-29 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| KR20210121199A (ko) | 2019-02-01 | 2021-10-07 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 방법 |
| CN113748116A (zh) | 2019-03-20 | 2021-12-03 | 波涛生命科学有限公司 | 可用于寡核苷酸制备的技术 |
| WO2020196662A1 (ja) | 2019-03-25 | 2020-10-01 | 国立大学法人東京医科歯科大学 | 二本鎖核酸複合体及びその使用 |
| EP3958872A4 (en) | 2019-04-25 | 2024-07-03 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CA3137741A1 (en) | 2019-04-25 | 2020-10-29 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| AU2020267775A1 (en) | 2019-05-09 | 2021-12-02 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| KR20220070324A (ko) | 2019-10-06 | 2022-05-30 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 이용 방법 |
| CN114585737A (zh) | 2019-10-06 | 2022-06-03 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| AU2021230473A1 (en) | 2020-03-01 | 2022-09-22 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| JP2023526975A (ja) | 2020-05-22 | 2023-06-26 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物及びその方法 |
| 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 |
| 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 |
| WO2022046667A1 (en) | 2020-08-24 | 2022-03-03 | Wave Life Sciences Ltd. | Cells and non-human animals engineered to express adar1 and uses thereof |
| US20230416847A1 (en) | 2020-09-24 | 2023-12-28 | Mayo Foundation For Medical Education And Research | Screening platforms |
| CN116507724A (zh) | 2020-11-08 | 2023-07-28 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| EP4240849A4 (en) | 2020-11-08 | 2026-04-01 | Wave Life Sciences Ltd | OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES |
| AU2022352779A1 (en) | 2021-09-26 | 2024-03-28 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| JP2024536088A (ja) | 2021-09-26 | 2024-10-04 | ウェイブ ライフ サイエンシズ リミテッド | Mecp2転写物を編集するための組成物及びその方法 |
| EP4422645A4 (en) | 2021-10-27 | 2025-12-03 | Wave Life Sciences Ltd | OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODS OF USE |
| CA3236136A1 (en) | 2021-10-27 | 2023-05-04 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP4476202A1 (en) | 2022-02-11 | 2024-12-18 | Wave Life Sciences Ltd. | Stereoselective technologies for chiral compounds |
| IL315089A (en) | 2022-03-02 | 2024-10-01 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof for exon skipping |
| IL316178A (en) | 2022-04-15 | 2024-12-01 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods related thereto |
| CN119452084A (zh) | 2022-05-12 | 2025-02-14 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| JP2025526712A (ja) | 2022-08-11 | 2025-08-15 | 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 |
| WO2025030155A1 (en) | 2023-08-03 | 2025-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
-
2016
- 2016-07-22 CN CN201680042413.3A patent/CN108135921B/zh active Active
- 2016-07-22 HK HK18114508.6A patent/HK1255369A1/zh unknown
- 2016-07-22 US US15/746,220 patent/US10479995B2/en active Active
- 2016-07-22 CA CA2989682A patent/CA2989682A1/en active Pending
- 2016-07-22 JP JP2018502637A patent/JP7296724B2/ja active Active
- 2016-07-22 US US15/746,199 patent/US20180216107A1/en not_active Abandoned
- 2016-07-22 CR CR20180107A patent/CR20180107A/es unknown
- 2016-07-22 MA MA43072A patent/MA43072A/fr unknown
- 2016-07-22 IL IL320434A patent/IL320434A/en unknown
- 2016-07-22 EP EP16828604.5A patent/EP3325017A4/en not_active Withdrawn
- 2016-07-22 MX MX2018000796A patent/MX2018000796A/es unknown
- 2016-07-22 EP EP23206565.6A patent/EP4344744A3/en active Pending
- 2016-07-22 WO PCT/US2016/043542 patent/WO2017015555A1/en not_active Ceased
- 2016-07-22 SG SG10201912900XA patent/SG10201912900XA/en unknown
- 2016-07-22 AU AU2016297155A patent/AU2016297155B2/en active Active
- 2016-07-22 HK HK18114509.5A patent/HK1255370A1/zh unknown
- 2016-07-22 JP JP2018502647A patent/JP2018525357A/ja active Pending
- 2016-07-22 TW TW105123348A patent/TW201707711A/zh unknown
- 2016-07-22 CN CN201680043299.6A patent/CN108025089A/zh active Pending
- 2016-07-22 KR KR1020187004483A patent/KR20180028516A/ko not_active Withdrawn
- 2016-07-22 WO PCT/US2016/043598 patent/WO2017015575A1/en not_active Ceased
- 2016-07-22 BR BR112017028636A patent/BR112017028636A2/pt not_active IP Right Cessation
- 2016-07-22 EP EP16828617.7A patent/EP3324978A4/en active Pending
- 2016-07-22 CN CN202311216358.3A patent/CN117965536A/zh active Pending
- 2016-07-22 PE PE2018000099A patent/PE20181174A1/es unknown
-
2017
- 2017-12-26 IL IL256603A patent/IL256603A/en unknown
-
2018
- 2018-01-15 NI NI201800009A patent/NI201800009A/es unknown
- 2018-01-18 MX MX2025000894A patent/MX2025000894A/es unknown
- 2018-01-18 PH PH12018500145A patent/PH12018500145A1/en unknown
- 2018-01-18 CL CL2018000145A patent/CL2018000145A1/es unknown
- 2018-01-22 SV SV2018005619A patent/SV2018005619A/es unknown
- 2018-02-09 EC ECIEPI201810463A patent/ECSP18010463A/es unknown
- 2018-02-15 CO CONC2018/0001484A patent/CO2018001484A2/es unknown
-
2019
- 2019-08-26 US US16/551,503 patent/US20200080083A1/en not_active Abandoned
-
2020
- 2020-02-04 US US16/782,021 patent/US11634710B2/en active Active
-
2021
- 2021-07-14 US US17/375,658 patent/US20220195429A1/en not_active Abandoned
- 2021-08-20 JP JP2021134582A patent/JP7441816B2/ja active Active
- 2021-10-18 JP JP2021170541A patent/JP7606954B2/ja active Active
-
2023
- 2023-02-14 AU AU2023200807A patent/AU2023200807A1/en not_active Abandoned
- 2023-04-21 US US18/305,195 patent/US12486505B2/en active Active
- 2023-12-08 JP JP2023207761A patent/JP7818563B2/ja active Active
-
2024
- 2024-12-13 JP JP2024219444A patent/JP2025038091A/ja active Pending
-
2025
- 2025-08-25 AU AU2025220872A patent/AU2025220872A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018525357A5 (https=) | ||
| US10337005B2 (en) | Oligonucleotide-based inhibitors comprising locked nucleic acid motif | |
| JP2023179428A (ja) | メッセンジャーリボ核酸(mRNA)をコードする安定化された核酸 | |
| EP4605531A1 (en) | Heteroduplex rna editing oligonucleotide complexes | |
| JP2020188771A (ja) | 二本鎖RNAによるα−1アンチトリプシンの特異的阻害のための方法及び組成物 | |
| US20140066491A1 (en) | Chemical modification motifs for mirna inhibitors and mimetics | |
| TWI816066B (zh) | 用於降低PAPD5及PAPD7 mRNA以治療B型肝炎感染之核酸分子 | |
| JP2018512155A5 (https=) | ||
| JP2016513976A5 (https=) | ||
| TW202219273A (zh) | 靶向rna結合蛋白位點之寡核苷酸 | |
| CN104540527A (zh) | Mir-15微小rna家族的抑制剂 | |
| JP2019520839A (ja) | 単分子配列決定のための一本鎖環状dnaライブラリーを生成するための方法 | |
| WO2024200472A1 (en) | Antisense oligonucleotides for the treatment of liver disease | |
| JPWO2020171149A1 (ja) | ヘテロ核酸の最適ps修飾パターン | |
| JP2008541729A (ja) | LMW−PTPase発現の調節 | |
| TW202136510A (zh) | 用於抑制scn9a表現之增強型寡核苷酸 | |
| JPWO2023109938A5 (https=) | ||
| HK40116036A (zh) | 靶向rna结合蛋白位点的改进的寡核苷酸 | |
| JP2026068029A (ja) | 一本鎖核酸及びその用途 | |
| RU2017146349A (ru) | Композиции олигонуклеотидов и способы с ними | |
| HK1259394A1 (en) | Oligonucleotide-based inhibitors comprising locked nucleic acid motif | |
| HK40033074A (en) | Nucleic acid molecule for reduction of papd5 and papd7 mrna for treating hepatitis b infection | |
| HK40029231A (en) | Stabilized nucleic acids encoding messenger ribonucleic acid (mrna) |