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 (https=) | 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 (https=) |
| EP (3) | EP3628750A1 (https=) |
| JP (1) | JP6018506B2 (https=) |
| AU (1) | AU2011213563B2 (https=) |
| CA (1) | CA2789038A1 (https=) |
| IL (1) | IL221271A (https=) |
| WO (1) | WO2011097644A2 (https=) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| WO2011097643A1 (en) * | 2010-02-08 | 2011-08-11 | Isis 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 |
| US10017764B2 (en) | 2011-02-08 | 2018-07-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| 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 |
| WO2013159108A2 (en) | 2012-04-20 | 2013-10-24 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| 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 |
| WO2014059353A2 (en) | 2012-10-11 | 2014-04-17 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleosides and uses thereof |
| US20160138014A1 (en) * | 2012-10-12 | 2016-05-19 | Isis Pharmaceuticals, Inc. | Antisense compounds and uses thereof |
| EP3459549B1 (en) | 2012-10-12 | 2022-04-06 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| ES2817050T3 (es) * | 2013-02-04 | 2021-04-06 | Ionis Pharmaceuticals Inc | Compuestos antisentido selectivos y usos de los mismos |
| EP3008201B1 (en) | 2013-06-12 | 2019-08-07 | The General Hospital Corporation | Methods for multiplexed detection of target molecules and uses thereof |
| 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 |
| 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 |
| 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 |
| WO2016059239A1 (en) * | 2014-10-17 | 2016-04-21 | Nogra Pharma Limited | Methods and compositions for treating a subject with a smad7 antisense oligonucleotide |
| 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 |
| EA201800367A1 (ru) | 2015-12-10 | 2019-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы лечения болезни хантингтона |
| 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 |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes for delivery of therapeutic agents |
| EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
| 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 |
| EA202092001A1 (ru) | 2018-03-27 | 2021-01-29 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения болезни гентингтона |
| HRP20240935T1 (hr) | 2018-06-27 | 2024-11-22 | Ptc Therapeutics, Inc. | Heterociklični i heteroaril spojevi za liječenje huntingtonove bolesti |
| WO2020005877A1 (en) | 2018-06-27 | 2020-01-02 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| WO2020117706A1 (en) | 2018-12-03 | 2020-06-11 | Triplet Therapeutics, Inc. | Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity |
| KR20210121199A (ko) * | 2019-02-01 | 2021-10-07 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 방법 |
| 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 |
| 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 |
| 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 |
| US20240376469A1 (en) * | 2021-01-29 | 2024-11-14 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating huntingtin |
| 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 |
| IL322607A (en) * | 2023-02-17 | 2025-10-01 | Ionis Pharmaceuticals Inc | Allele-selective compounds and methods for modulating huntingtin expression |
| AU2024287308A1 (en) | 2023-07-13 | 2025-12-18 | 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 |
| EP0673559A1 (en) | 1992-12-14 | 1995-09-27 | Honeywell Inc. | Motor system with individually controlled redundant windings |
| 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 |
| US5898031A (en) * | 1996-06-06 | 1999-04-27 | Isis Pharmaceuticals, Inc. | Oligoribonucleotides for cleaving RNA |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| 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 |
| AU9063398A (en) | 1997-09-12 | 1999-04-05 | Exiqon A/S | Oligonucleotide analogues |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US20030228597A1 (en) | 1998-04-13 | 2003-12-11 | Cowsert Lex M. | Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation |
| US6753422B2 (en) * | 1999-03-01 | 2004-06-22 | O'brien Thomas G. | Odc allelic analysis method for assessing carcinogenic susceptibility |
| JP2003503008A (ja) | 1999-03-01 | 2003-01-28 | バリアジェニックス インコーポレーテッド | Ｒｎａ分子をターゲティングする方法 |
| EP1178999B1 (en) | 1999-05-04 | 2007-03-14 | Santaris Pharma A/S | L-ribo-lna analogues |
| US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
| AU4819101A (en) | 2000-04-13 | 2001-10-30 | University Of British Columbia, The | Modulating cell survival by modulating huntingtin function |
| JP2005504020A (ja) | 2001-07-03 | 2005-02-10 | アイシス・ファーマシューティカルス・インコーポレーテッド | ヌクレアーゼ耐性キメラオリゴヌクレオチド |
| AU2002326589B2 (en) | 2001-08-07 | 2008-06-05 | University Of Delaware | Compositions and methods for the prevention and treatment of Huntington's disease |
| US8604183B2 (en) | 2002-11-05 | 2013-12-10 | Isis Pharmaceuticals, Inc. | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
| CA2504694C (en) | 2002-11-05 | 2013-10-01 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| EP2305812A3 (en) | 2002-11-14 | 2012-06-06 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| WO2004106356A1 (en) | 2003-05-27 | 2004-12-09 | Syddansk Universitet | Functionalized nucleotide derivatives |
| EP1661905B9 (en) | 2003-08-28 | 2012-12-19 | IMANISHI, Takeshi | Novel artificial nucleic acids of n-o bond crosslinkage type |
| WO2005027962A1 (en) | 2003-09-18 | 2005-03-31 | Isis Pharmaceuticals, Inc. | 4’-thionucleosides and oligomeric compounds |
| US20050176045A1 (en) * | 2004-02-06 | 2005-08-11 | Dharmacon, Inc. | SNP discriminatory siRNA |
| AU2005252663B2 (en) * | 2004-06-03 | 2011-07-07 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
| EP2397563A3 (en) * | 2004-09-17 | 2012-07-18 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| WO2007002904A2 (en) | 2005-06-28 | 2007-01-04 | Medtronic, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| 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 |
| US9273356B2 (en) | 2006-05-24 | 2016-03-01 | Medtronic, Inc. | Methods and kits for linking polymorphic sequences to expanded repeat mutations |
| 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 |
| JP2010510807A (ja) | 2006-11-27 | 2010-04-08 | 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 | 高コレステロール血症を治療するための方法 |
| US8093222B2 (en) | 2006-11-27 | 2012-01-10 | Isis Pharmaceuticals, Inc. | Methods for treating hypercholesterolemia |
| US20100190837A1 (en) | 2007-02-15 | 2010-07-29 | Isis Pharmaceuticals, Inc. | 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom |
| WO2008143774A2 (en) | 2007-05-01 | 2008-11-27 | University Of Massachusetts | Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy |
| EP2170917B1 (en) | 2007-05-30 | 2012-06-27 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| EP2173760B2 (en) | 2007-06-08 | 2015-11-04 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| 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 |
| CA2692579C (en) | 2007-07-05 | 2016-05-03 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| CA2726866A1 (en) | 2008-05-09 | 2009-11-12 | The University Of British Columbia | Methods and compositions for the treatment of huntington's disease |
| AT507215B1 (de) | 2009-01-14 | 2010-03-15 | Boehler Edelstahl Gmbh & Co Kg | Verschleissbeständiger werkstoff |
| US9778706B2 (en) | 2012-02-24 | 2017-10-03 | Blackberry Limited | Peekable user interface on a portable electronic device |
| US9984408B1 (en) | 2012-05-30 | 2018-05-29 | Amazon Technologies, Inc. | Method, medium, and system for live video cooperative shopping |
-
2011
- 2011-02-08 EP EP19191293.0A patent/EP3628750A1/en active Pending
- 2011-02-08 AU AU2011213563A patent/AU2011213563B2/en active Active
- 2011-02-08 JP JP2012552933A patent/JP6018506B2/ja active Active
- 2011-02-08 EP EP16203872.3A patent/EP3208347B1/en not_active Revoked
- 2011-02-08 WO PCT/US2011/024104 patent/WO2011097644A2/en not_active Ceased
- 2011-02-08 US US13/577,622 patent/US9006198B2/en active Active
- 2011-02-08 CA CA2789038A patent/CA2789038A1/en active Pending
- 2011-02-08 EP EP11740543.1A patent/EP2534262B1/en active Active
-
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
-
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
-
2023
- 2023-10-16 US US18/487,664 patent/US20240309452A1/en active Pending
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