JP2013518605A5 - - Google Patents
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- Publication number
- JP2013518605A5 JP2013518605A5 JP2012552933A JP2012552933A JP2013518605A5 JP 2013518605 A5 JP2013518605 A5 JP 2013518605A5 JP 2012552933 A JP2012552933 A JP 2012552933A JP 2012552933 A JP2012552933 A JP 2012552933A JP 2013518605 A5 JP2013518605 A5 JP 2013518605A5
- Authority
- JP
- Japan
- Prior art keywords
- modified
- sugar
- nucleoside
- modified oligonucleotide
- wing
- 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.)
- Granted
Links
- 238000000034 method Methods 0.000 claims 31
- 108091034117 Oligonucleotide Proteins 0.000 claims 16
- 239000002777 nucleoside Substances 0.000 claims 16
- 108700028369 Alleles Proteins 0.000 claims 12
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 8
- 230000000295 complement effect Effects 0.000 claims 6
- 125000003835 nucleoside group Chemical group 0.000 claims 5
- 125000002619 bicyclic group Chemical group 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- -1 bicyclic nucleoside Chemical class 0.000 claims 3
- 150000001875 compounds Chemical class 0.000 claims 3
- 238000012986 modification Methods 0.000 claims 3
- 230000004048 modification Effects 0.000 claims 3
- 235000021092 sugar substitutes Nutrition 0.000 claims 3
- 239000003765 sweetening agent Substances 0.000 claims 3
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 108091027967 Small hairpin RNA Proteins 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- 239000004055 small Interfering RNA Substances 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US30245810P | 2010-02-08 | 2010-02-08 | |
| US61/302,458 | 2010-02-08 | ||
| US37164010P | 2010-08-06 | 2010-08-06 | |
| US61/371,640 | 2010-08-06 | ||
| PCT/US2011/024104 WO2011097644A2 (en) | 2010-02-08 | 2011-02-08 | Selective reduction of allelic variants |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013518605A JP2013518605A (ja) | 2013-05-23 |
| JP2013518605A5 true JP2013518605A5 (enExample) | 2014-03-27 |
| JP6018506B2 JP6018506B2 (ja) | 2016-11-02 |
Family
ID=44356119
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012552933A Active JP6018506B2 (ja) | 2010-02-08 | 2011-02-08 | 対立遺伝子多様体の選択的低減 |
Country Status (7)
| Country | Link |
|---|---|
| US (5) | US9006198B2 (enExample) |
| EP (3) | EP3208347B1 (enExample) |
| JP (1) | JP6018506B2 (enExample) |
| AU (1) | AU2011213563B2 (enExample) |
| CA (1) | CA2789038A1 (enExample) |
| IL (1) | IL221271A (enExample) |
| WO (1) | WO2011097644A2 (enExample) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
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| EP3321361B1 (en) * | 2010-02-08 | 2019-03-27 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| WO2012109395A1 (en) * | 2011-02-08 | 2012-08-16 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| 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 |
| WO2013159108A2 (en) | 2012-04-20 | 2013-10-24 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| BR112015000723A2 (pt) | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
| US9695418B2 (en) | 2012-10-11 | 2017-07-04 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleosides and uses thereof |
| EP2906255B1 (en) * | 2012-10-12 | 2023-02-22 | Ionis Pharmaceuticals, Inc. | Antisense compounds and uses thereof |
| 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 |
| ES2754177T3 (es) | 2013-06-12 | 2020-04-16 | Massachusetts Gen Hospital | Métodos para la detección multiplexada de moléculas diana y sus usos |
| 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 |
| 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 |
| EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| MA43331A (fr) * | 2014-10-17 | 2017-08-23 | Nogra Pharma Ltd | Procédés et compositions pour le traitement d'un sujet au moyen d'un oligonucléotide antisens de smad7 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| EA201800367A1 (ru) | 2015-12-10 | 2019-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы лечения болезни хантингтона |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| KR20200033249A (ko) | 2017-06-05 | 2020-03-27 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
| CA3067591A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | Methods for treating huntington's disease |
| US11395822B2 (en) | 2017-06-28 | 2022-07-26 | 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 |
| US12103926B2 (en) | 2018-03-27 | 2024-10-01 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| BR112020026545A2 (pt) | 2018-06-27 | 2021-03-23 | Ptc Therapeutics, Inc. | Compostos heterocíclicos e de heteroarila para o tratamento da doença de huntington |
| EA202092899A1 (ru) | 2018-06-27 | 2021-05-14 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Гетероарильные соединения для лечения болезни гентингтона |
| WO2020117706A1 (en) | 2018-12-03 | 2020-06-11 | Triplet Therapeutics, Inc. | Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity |
| CA3126845A1 (en) * | 2019-02-01 | 2020-08-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| 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 |
| US20220251557A1 (en) * | 2019-11-27 | 2022-08-11 | Tokyo Institute Of Technology | Method for reducing toxicity of antisense nucleic acids |
| 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 |
| 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 |
| TW202241462A (zh) * | 2021-01-29 | 2022-11-01 | 美商Ionis製藥公司 | 調節杭丁頓蛋白（ｈｕｎｔｉｎｇｔｉｎ）之化合物及方法 |
| 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 |
| EP4665359A2 (en) * | 2023-02-17 | 2025-12-24 | Ionis Pharmaceuticals, Inc. | Allele-selective compounds and methods for modulating huntingtin expression |
| WO2025015335A1 (en) | 2023-07-13 | 2025-01-16 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| WO2025128799A1 (en) | 2023-12-12 | 2025-06-19 | Korro Bio, Inc. | Double-stranded rna-editing oligonucleotides and uses thereof |
Family Cites Families (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US2699808A (en) | 1944-10-06 | 1955-01-18 | Mark W Lowe | Apparatus for peeling tomatoes |
| US2699508A (en) | 1951-12-21 | 1955-01-11 | Selectronics Inc | Method of mounting and construction of mounting for low frequency piezoelectric crystals |
| US6582908B2 (en) | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
| JPH08504559A (ja) | 1992-12-14 | 1996-05-14 | ハネウエル・インコーポレーテッド | 個別に制御される冗長巻線を有するモータシステム |
| US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
| US5656408A (en) | 1996-04-29 | 1997-08-12 | Xerox Corporation | Coated carrier particles |
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| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
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| US6753422B2 (en) * | 1999-03-01 | 2004-06-22 | O'brien Thomas G. | Odc allelic analysis method for assessing carcinogenic susceptibility |
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| WO2001079283A1 (en) | 2000-04-13 | 2001-10-25 | University Of British Columbia | Modulating cell survival by modulating huntingtin function |
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| AU2002326589B2 (en) | 2001-08-07 | 2008-06-05 | University Of Delaware | Compositions and methods for the prevention and treatment of Huntington's disease |
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| WO2004106356A1 (en) | 2003-05-27 | 2004-12-09 | Syddansk Universitet | Functionalized nucleotide derivatives |
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-
2011
- 2011-02-08 EP EP16203872.3A patent/EP3208347B1/en not_active Revoked
- 2011-02-08 EP EP19191293.0A patent/EP3628750A1/en active Pending
- 2011-02-08 WO PCT/US2011/024104 patent/WO2011097644A2/en not_active Ceased
- 2011-02-08 AU AU2011213563A patent/AU2011213563B2/en active Active
- 2011-02-08 JP JP2012552933A patent/JP6018506B2/ja active Active
- 2011-02-08 EP EP11740543.1A patent/EP2534262B1/en active Active
- 2011-02-08 US US13/577,622 patent/US9006198B2/en active Active
- 2011-02-08 CA CA2789038A patent/CA2789038A1/en active Pending
-
2012
- 2012-08-02 IL IL221271A patent/IL221271A/en active IP Right Grant
-
2015
- 2015-03-03 US US14/636,960 patent/US20150292015A1/en not_active Abandoned
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2017
- 2017-12-18 US US15/846,039 patent/US20180291453A1/en not_active Abandoned
-
2020
- 2020-01-14 US US16/742,320 patent/US20200377946A1/en not_active Abandoned
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2023
- 2023-10-16 US US18/487,664 patent/US20240309452A1/en active Pending