JP2016518842A - マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法 - Google Patents

マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法 Download PDF

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JP2016518842A
JP2016518842A JP2016512010A JP2016512010A JP2016518842A JP 2016518842 A JP2016518842 A JP 2016518842A JP 2016512010 A JP2016512010 A JP 2016512010A JP 2016512010 A JP2016512010 A JP 2016512010A JP 2016518842 A JP2016518842 A JP 2016518842A
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nucleoside
compound
certain embodiments
modified
genotype
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JP2016518842A5 (enExample
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バルクリシェン バット
バルクリシェン バット
ダニエル ホーガン
ダニエル ホーガン
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レグルス セラピューティクス インコーポレイテッド
レグルス セラピューティクス インコーポレイテッド
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    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021531278A (ja) * 2018-07-20 2021-11-18 レグルス セラピューティクス インコーポレイテッド オリゴヌクレオチドの経口送達のための方法

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016518842A (ja) 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法
EP2992096B1 (en) * 2013-05-01 2021-03-03 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
DK3047160T3 (en) * 2013-09-16 2019-04-08 Vaelinge Innovation Ab TOTAL FURNITURE PRODUCT
WO2015061536A1 (en) 2013-10-25 2015-04-30 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
US10308937B2 (en) * 2014-11-10 2019-06-04 Glaxosmithkline Intellectual Property (No. 2) Limited Combination long acting compositions and methods for Hepatitis C
MA40844A (fr) * 2014-11-10 2017-09-20 Glaxosmithkline Ip No 2 Ltd Compositions pharmaceutiques à action prolongée pour l'hépatite c
AU2016349625B2 (en) 2015-11-06 2022-07-07 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
LT4119569T (lt) * 2015-11-06 2024-09-25 Ionis Pharmaceuticals, Inc. Konjuguoti priešprasmiai junginiai, skirti panaudoti terapijoje
JOP20170057B1 (ar) 2016-03-07 2022-03-14 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة كمركبات علاجية
JP7179721B2 (ja) 2016-06-21 2022-11-29 オリオン・オフサルモロジー・エルエルシー 複素環式プロリンアミド誘導体
JP7164521B2 (ja) 2016-06-21 2022-11-01 オリオン・オフサルモロジー・エルエルシー 炭素環式プロリンアミド誘導体
JOP20170056B1 (ar) 2016-09-02 2021-08-17 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
TWI854980B (zh) * 2018-05-08 2024-09-11 美商雷格勒斯治療公司 用於調節mir-122之微小rna化合物及方法
CN112843084A (zh) * 2019-11-27 2021-05-28 中国人民解放军第四军医大学 MiR-122-5p在制备促进创伤愈合药物中的应用
CN114057816B (zh) * 2020-07-30 2025-02-11 浙江柏拉阿图医药科技有限公司 富含腺嘌呤硫代磷酸酯化寡核苷酸及其抗肝炎病毒的应用
WO2023060237A1 (en) * 2021-10-08 2023-04-13 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
JP2024535445A (ja) * 2021-10-08 2024-09-30 レグルス セラピューティクス インコーポレイテッド オフターゲット効果を回避するための方法及び組成物
US20250049837A1 (en) * 2023-01-09 2025-02-13 Unity Health Toronto Microrna-based particle for the treatment of dysregulated immune response

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003524637A (ja) * 1999-12-23 2003-08-19 エクシコン エ／エス Ｌｎａ修飾オリゴヌクレオチドの治療上の使用
WO2008091703A2 (en) * 2007-01-24 2008-07-31 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro rna and uses thereof
JP2009506124A (ja) * 2005-08-29 2009-02-12 アイシス・ファーマシューティカルズ・インコーポレイテッド Ｍｉｒ−１２２ａをモジュレートする使用方法
JP2010539959A (ja) * 2007-10-04 2010-12-24 サンタリス ファーマ アー／エス マイクロｍｉｒ
JP2012524539A (ja) * 2009-04-24 2012-10-18 サンタリス ファーマ アー／エス インターフェロンに非応答性のｈｃｖ患者の治療のための医薬組成物
WO2012148952A1 (en) * 2011-04-25 2012-11-01 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-21 activity
WO2012173274A1 (ja) * 2011-06-16 2012-12-20 日鉄環境エンジニアリング株式会社 核酸測定用の核酸プローブ
WO2013033230A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use

Family Cites Families (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH10503364A (ja) 1994-05-10 1998-03-31 ザ ジェネラル ホスピタル コーポレーション Ｃ型肝炎ウイルスのアンチセンス阻害
TW520293B (en) 1995-11-22 2003-02-11 Univ Johns Hopkins Med Delivery system to enhance cellular uptake of biomolecules
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
AU7406700A (en) 1999-10-04 2001-05-10 Exiqon A/S Design of high affinity rnase h recruiting oligonucleotide
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
CN100406065C (zh) 2000-12-01 2008-07-30 细胞工厂治疗公司 糖基化/半乳糖基化肽、双官能接头和核苷酸单体/多聚体的缀合物以及相关的组合物和使用方法
CN1344803A (zh) 2001-09-27 2002-04-17 吴昌 核酸靶分子选择及扩增方法
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP2669377A3 (en) 2003-04-17 2015-10-14 Alnylam Pharmaceuticals Inc. Modified iRNA agents
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
JP4989225B2 (ja) 2003-09-25 2012-08-01 コーリー ファーマシューティカル グループ，インコーポレイテッド 核酸親油性接合体
ES2344566T3 (es) 2003-12-23 2010-08-31 Santaris Pharma A/S Compuestos oligomericos para la modulacion de bcl-2.
AU2005325262B2 (en) 2004-04-27 2011-08-11 Alnylam Pharmaceuticals, Inc. Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
PT1747023E (pt) 2004-05-04 2011-04-11 Univ Leland Stanford Junior Métodos e composições para reduzir quantidades de genoma viral numa célula alvo
EP1913011B1 (en) 2004-08-04 2016-11-02 Alnylam Pharmaceuticals Inc. Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
WO2006031461A2 (en) 2004-09-09 2006-03-23 Isis Pharmaceuticals, Inc. Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
US20070099196A1 (en) 2004-12-29 2007-05-03 Sakari Kauppinen Novel oligonucleotide compositions and probe sequences useful for detection and analysis of micrornas and their target mRNAs
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
EP1841867A1 (en) 2005-01-24 2007-10-10 Avaris AB COMPLEX CONTAINING SiRNA, ShRNA OR ANTISENSE MOLECULE AND FUNCTIONAL ENTITY, FOR IMPROVED SPECIFICITY AND DELIVERY
AU2006279906B2 (en) 2005-08-10 2012-05-10 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
WO2007027894A2 (en) 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Antisense compounds having enhanced anti-microrna activity
EP2388328A1 (en) 2006-01-27 2011-11-23 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
CA3042781C (en) 2006-04-03 2021-10-19 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
EA201100811A1 (ru) 2006-04-03 2012-06-29 Сантарис Фарма А/С Фармацевтическая композиция
CA3144493A1 (en) 2006-10-03 2008-04-10 Arbutus Biopharma Corporation Lipid containing formulations
DK2152879T3 (da) 2007-05-01 2013-02-18 Santaris Pharma As RNA antagonistforbindelser til modulering af beta-catenin
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
NZ585327A (en) 2007-11-26 2012-10-26 Santaris Pharma As Lna antagonists targeting the androgen receptor
EP2229186A2 (en) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
WO2009091972A2 (en) 2008-01-18 2009-07-23 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and small molecules for use in reducing micro rna activity levels and uses thereof
EP2268811A1 (en) 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
WO2010076248A1 (en) 2008-12-31 2010-07-08 Santaris Pharma A/S Use of lna apob antisense oligomers for the treatment of acute coronary syndromes
MA33488B1 (fr) 2009-06-08 2012-08-01 Miragen Therapeutics Motifs de modification chimique pour des inhibiteurs et mimétiques de miarn
ES2651308T3 (es) 2009-10-16 2018-01-25 Glaxo Group Limited Inhibidores antisentido de HBV
PH12012501596A1 (en) 2010-02-24 2017-02-03 Arrowhead Res Corporation Compositions for targeted delivery of sirna
US8349308B2 (en) 2010-03-26 2013-01-08 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
WO2011130458A2 (en) 2010-04-13 2011-10-20 John Rossi Rna aptamers against baff-r as cell-type specific delivery agents and methods for their use
WO2012007477A1 (en) 2010-07-12 2012-01-19 Santaris Pharma A/S Anti hcv oligomers
US9290760B2 (en) * 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
RU2013117288A (ru) 2010-12-17 2015-01-27 Эрроухэд Рисерч Корпорейшн СОДЕРЖАЩАЯ ГАЛАКТОЗНЫЙ КЛАСТЕР НАЦЕЛИВАЮЩАЯ ГРУППА ДЛЯ миРНК, МОДУЛИРУЮЩАЯ ФОРМАКОКИНЕТИЧЕСКИЕ СВОЙСТВА
RU2582235C2 (ru) 2010-12-29 2016-04-20 Ф.Хоффманн-Ля Рош Аг Низкомолекулярные конъюгаты для внутриклеточной доставки нуклеиновых кислот
WO2012175733A1 (en) 2011-06-23 2012-12-27 Santaris Pharma A/S Hcv combination therapy
KR20140058536A (ko) 2011-06-30 2014-05-14 스텔라 에이피에스 Hcv 조합 치료
EP2726610A1 (en) 2011-06-30 2014-05-07 Stella ApS Hcv combination therapy
WO2013068348A1 (en) 2011-11-07 2013-05-16 Santaris Pharma A/S Lna oligomers for improvement in hepatic function
EP2776590B1 (en) 2011-11-07 2016-10-26 Roche Innovation Center Copenhagen A/S Prognostic method for checking efficacy of micro rna-122 inhibitors in hcv+ patients
EP2864482A4 (en) 2012-06-21 2016-02-10 Miragen Therapeutics Inc OLIGONUCLEOTIDE INHIBITORS COMPRISING A BLOCKED NUCLEIC ACID PATTERN
CN104955950B (zh) 2012-09-26 2019-04-26 米尔克斯治疗学公司 具有改善的脱靶特征谱的寡核苷酸
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
CA2893801A1 (en) 2013-01-30 2014-08-07 F. Hoffmann-La Roche Ag Lna oligonucleotide carbohydrate conjugates
EP2992096B1 (en) 2013-05-01 2021-03-03 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
JP2016518842A (ja) 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003524637A (ja) * 1999-12-23 2003-08-19 エクシコン エ／エス Ｌｎａ修飾オリゴヌクレオチドの治療上の使用
JP2009506124A (ja) * 2005-08-29 2009-02-12 アイシス・ファーマシューティカルズ・インコーポレイテッド Ｍｉｒ−１２２ａをモジュレートする使用方法
WO2008091703A2 (en) * 2007-01-24 2008-07-31 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro rna and uses thereof
JP2010539959A (ja) * 2007-10-04 2010-12-24 サンタリス ファーマ アー／エス マイクロｍｉｒ
JP2012524539A (ja) * 2009-04-24 2012-10-18 サンタリス ファーマ アー／エス インターフェロンに非応答性のｈｃｖ患者の治療のための医薬組成物
WO2012148952A1 (en) * 2011-04-25 2012-11-01 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-21 activity
WO2012173274A1 (ja) * 2011-06-16 2012-12-20 日鉄環境エンジニアリング株式会社 核酸測定用の核酸プローブ
WO2013033230A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021531278A (ja) * 2018-07-20 2021-11-18 レグルス セラピューティクス インコーポレイテッド オリゴヌクレオチドの経口送達のための方法

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