JP2019516680A - オリゴヌクレオチド組成物およびその方法 - Google Patents

オリゴヌクレオチド組成物およびその方法 Download PDF

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JP2019516680A
JP2019516680A JP2018557087A JP2018557087A JP2019516680A JP 2019516680 A JP2019516680 A JP 2019516680A JP 2018557087 A JP2018557087 A JP 2018557087A JP 2018557087 A JP2018557087 A JP 2018557087A JP 2019516680 A JP2019516680 A JP 2019516680A
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oligonucleotide
composition
sequence
pattern
backbone
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JP2019516680A5 (enExample
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チャンドラ ヴァージェシー
チャンドラ ヴァージェシー
ミーナ
ネナド シビルジカパ
ネナド シビルジカパ
スソヴァン モハパトラ
スソヴァン モハパトラ
クリストファー ジェイ フランシス
クリストファー ジェイ フランシス
グレゴリー エル ヴァーダイン
グレゴリー エル ヴァーダイン
アンナ ソコロヴスカ
アンナ ソコロヴスカ
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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JP2018557087A 2016-05-04 2017-05-03 オリゴヌクレオチド組成物およびその方法 Pending JP2019516680A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662331960P 2016-05-04 2016-05-04
US62/331,960 2016-05-04
USPCT/US2016/043542 2016-07-22
PCT/US2016/043542 WO2017015555A1 (en) 2015-07-22 2016-07-22 Oligonucleotide compositions and methods thereof
US201762447832P 2017-01-18 2017-01-18
US62/447,832 2017-01-18
PCT/US2017/030753 WO2017192664A1 (en) 2016-05-04 2017-05-03 Oligonucleotide compositions and methods thereof

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JP2019516680A true JP2019516680A (ja) 2019-06-20
JP2019516680A5 JP2019516680A5 (enExample) 2020-06-18

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US (2) US10724035B2 (enExample)
EP (1) EP3452596A4 (enExample)
JP (1) JP2019516680A (enExample)
CN (1) CN109477108A (enExample)
MA (1) MA45270A (enExample)
WO (1) WO2017192664A1 (enExample)

Families Citing this family (73)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011005761A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc Novel nucleic acid prodrugs and methods use thereof
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
AU2013288048A1 (en) 2012-07-13 2015-01-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
PL3234134T3 (pl) 2014-12-17 2020-12-28 Proqr Therapeutics Ii B.V. Ukierunkowana edycja rna
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017062862A2 (en) 2015-10-09 2017-04-13 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
KR102488323B1 (ko) 2015-12-10 2023-01-12 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
AU2017234150B2 (en) 2016-03-13 2021-09-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45188A (fr) 2016-06-03 2019-04-10 Wave Life Sciences Ltd Oligonucléotides, compositions et méthodes associées
AU2017281497B2 (en) 2016-06-22 2023-04-06 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
PL3507366T3 (pl) 2016-09-01 2021-05-04 Proqr Therapeutics Ii B.V. Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
MY195801A (en) 2016-12-19 2023-02-22 Sarepta Therapeutics Inc Exon Skipping Oligomer Conjugates for Muscular Dystrophy
RS63705B1 (sr) 2016-12-19 2022-11-30 Sarepta Therapeutics Inc Oligomerni konjugati za preskakanje egzona za mišićnu distrofiju
PT3554552T (pt) 2016-12-19 2022-10-03 Sarepta Therapeutics Inc Conjugados de oligómero de skipping de exão para distrofia muscular
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
EP3630789A4 (en) 2017-06-02 2021-06-16 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR THEIR USE
CN111050806A (zh) 2017-06-02 2020-04-21 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
JP7376471B2 (ja) 2017-06-05 2023-11-08 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
JP7402696B2 (ja) 2017-06-21 2023-12-21 ウェイブ ライフ サイエンシズ リミテッド 合成のための化合物、組成物、及び方法
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
BR112019027719A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
WO2019032607A1 (en) 2017-08-08 2019-02-14 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
KR20250123934A (ko) 2017-09-18 2025-08-18 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 제조 기술
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
EP3687577A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
US20200248178A1 (en) 2017-09-28 2020-08-06 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
US20200254002A1 (en) 2017-09-28 2020-08-13 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
US11596646B2 (en) 2017-10-12 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3737387A1 (en) 2018-01-12 2020-11-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
BR112020014011A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso dirigidos à alfa-sinucleína e usos dos mesmos
KR20210005559A (ko) 2018-03-27 2021-01-14 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
US12391942B2 (en) 2018-05-11 2025-08-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP4219717A3 (en) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
SMT202400286T1 (it) 2018-06-27 2024-09-16 Ptc Therapeutics Inc Composti eterociclici ed eteroarilici per il trattamento della corea di huntington
AU2019294482B2 (en) 2018-06-27 2022-09-01 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
TW202449155A (zh) 2018-07-27 2024-12-16 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
CA3122281A1 (en) 2018-12-13 2020-06-18 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
CA3126845A1 (en) * 2019-02-01 2020-08-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
MX2021012981A (es) * 2019-04-25 2022-01-18 Wave Life Sciences Ltd Composiciones de oligonucleotido y metodos de uso de las mismas.
CN114650822A (zh) 2019-11-01 2022-06-21 诺华股份有限公司 剪接调节子用于减慢亨廷顿氏病进展的治疗的用途
BR112022023465A2 (pt) 2020-05-22 2023-01-10 Wave Life Sciences Ltd Agente de rnai de fita dupla (dsrnai), composição de oligonucleotídeo quiralmente controlada, oligonucleotídeo de fita dupla, método para reduzir o nível e/ou a atividade de um transcrito ou uma proteína codificada pelo mesmo, e método para supressão específica quanto ao alelo de um transcrito de uma sequência de ácidos nucleicos
WO2021254593A1 (en) * 2020-06-15 2021-12-23 Curevac Ag Analysis of nucleic acid mixtures
TW202304446A (zh) 2021-03-29 2023-02-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
CA3232070A1 (en) * 2021-09-26 2023-03-30 Prashant MONIAN Oligonucleotide compositions and methods thereof
CA3233242A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
EP4555085A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Chemically modified oligonucleotides for adar-mediated rna editing
JP2025533454A (ja) 2022-09-21 2025-10-07 サレプタ セラピューティクス， インコーポレイテッド Ｄｍｄアンチセンスオリゴヌクレオチド介在性エクソンスキッピング効率
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CN120322556A (zh) 2022-12-09 2025-07-15 ProQR治疗上市公司Ⅱ 用于治疗心血管疾病的反义寡核苷酸
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
KR20250167580A (ko) 2023-03-27 2025-12-01 프로큐알 테라퓨틱스 Ⅱ 비.브이. 간 질환 치료용 안티센스 올리고뉴클레오타이드
WO2024227040A1 (en) 2023-04-27 2024-10-31 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of chronic kidney disease
WO2024256620A1 (en) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurodegenerative disease
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH06220083A (ja) * 1991-03-06 1994-08-09 Polish Acad Of Sci オリゴヌクレオチドとオリゴヌクレオチド類似体の固相合成のための方法と化合物
JP2002520420A (ja) * 1998-07-14 2002-07-09 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 部位特異的キラルホスホロチオエート・ヌクレオシド間結合を有するオリゴヌクレオチド
WO2014012081A2 (en) * 2012-07-13 2014-01-16 Ontorii, Inc. Chiral control
US20140256578A1 (en) * 2008-05-09 2014-09-11 The University Of British Columbia Methods and compositions for the treatment of huntington's disease

Family Cites Families (95)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
JPH09507836A (ja) 1993-11-16 1997-08-12 ジンタ・インコーポレイテッド 非ホスホネート・ヌクレオシジル間結合と混合したキラリティー的に純粋なホスホネート・ヌクレオシジル間結合を有する合成オリゴマー
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US20020082227A1 (en) 1999-09-30 2002-06-27 Scott Henry Use of oligonucleotides for inhibition of complement activation
DE10019756A1 (de) 2000-04-20 2001-10-25 Bayer Ag Verfahren zur Herstellung von superabsorbierenden Polymeren aus Polyacrylnitrilen
US20050277133A1 (en) 2001-05-18 2005-12-15 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
JP4348044B2 (ja) 2002-02-12 2009-10-21 株式会社キラルジェン 立体規則性の高いジヌクレオシドホスホロチオエートの製造法
US20050096284A1 (en) 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
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
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
JP2005089441A (ja) 2003-08-08 2005-04-07 Toudai Tlo Ltd 立体規則性の高いリン原子修飾ヌクレオチド類縁体の製造法
JP4616175B2 (ja) 2003-09-02 2011-01-19 株式会社キラルジェン ５’−ホスフィチル化モノマーおよびｈ−ホスホネートオリゴヌクレオチド誘導体の製造方法
WO2005023828A1 (ja) 2003-09-02 2005-03-17 Takeshi Wada リボヌクレオチド又はリボヌクレオチド誘導体の製造方法
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
ES2808561T3 (es) 2003-09-12 2021-03-01 Univ Massachusetts Interferencia por ARN para el tratamiento de trastornos de ganancia de función
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
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
US20060257912A1 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
US20070161590A1 (en) 2005-06-28 2007-07-12 Medtronic, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
CN101365801B (zh) * 2005-10-28 2013-03-27 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
DK2161038T3 (da) 2006-01-26 2014-03-03 Isis Pharmaceuticals Inc Sammensætninger og deres anvendelser rettet mod Huntington
EP2046993A4 (en) 2006-07-07 2010-11-17 Univ Massachusetts RNA SILENCING COMPOSITIONS, AND METHODS OF TREATING HUNTINGTON CHOREA
ES2620472T5 (es) 2006-10-18 2020-07-09 Ionis Pharmaceuticals Inc Compuestos antisentido
WO2008151833A2 (en) 2007-06-13 2008-12-18 Hochschule Mannheim Compounds for the modulation of huntingtin aggregation, methods and means for identifying such compounds
EP2014769B1 (en) 2007-06-18 2010-03-31 Commissariat à l'Energie Atomique Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease
WO2009098197A1 (en) 2008-02-04 2009-08-13 Biofocus Dpi B.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
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
CN102282155B (zh) 2008-12-02 2017-06-09 日本波涛生命科学公司 磷原子修饰的核酸的合成方法
US8987222B2 (en) * 2009-04-08 2015-03-24 University Of Massachusetts Single nucleotide polymorphism (SNP) targeting therapies for the treatment of huntington'S disease
WO2011005761A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc Novel nucleic acid prodrugs and methods use thereof
WO2011015572A1 (en) 2009-08-03 2011-02-10 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
WO2011015573A1 (en) 2009-08-03 2011-02-10 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
US8927553B2 (en) 2009-08-10 2015-01-06 Daljit Singh Dhanoa Deuterium-enriched alkyl sulfonamides and uses thereof
RU2751847C9 (ru) 2009-09-11 2021-08-20 Ионис Фармасьютикалз, Инк. Модуляция экспрессии гентингтина
JP5878758B2 (ja) 2009-09-16 2016-03-08 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ Ｒｎａ及びその誘導体合成のための新規保護基
CN102791862B (zh) 2009-12-31 2017-04-05 库尔纳公司 通过抑制胰岛素受体底物2(irs2)和转录因子e3(tfe3)的天然反义转录物而治疗irs2相关疾病
US9006198B2 (en) 2010-02-08 2015-04-14 Isis 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
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
EP2638163B1 (en) 2010-11-12 2017-05-17 The General Hospital Corporation Polycomb-associated non-coding rnas
US8822671B2 (en) 2010-11-30 2014-09-02 The University Of Tokyo 2'-O-modified RNA
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
CA2827380C (en) 2011-02-12 2020-08-25 University Of Iowa Research Foundation Therapeutic nucleic acids
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
US9752142B2 (en) 2011-08-11 2017-09-05 Ionis Pharmaceuticals, Inc. Gapped oligomeric compounds comprising 5′-modified deoxyribonucleosides in the gap and uses thereof
JP6317675B2 (ja) 2011-11-30 2018-04-25 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
AU2013288048A1 (en) 2012-07-13 2015-01-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
AU2013287630B2 (en) 2012-07-13 2017-05-25 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
WO2014059356A2 (en) 2012-10-12 2014-04-17 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2014121287A2 (en) 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
IL296543B2 (en) 2013-05-01 2025-02-01 Ionis Pharmaceuticals Inc Compositions and methods for modulating HBV and TTR expression
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
EP3068905A4 (en) 2013-11-11 2017-07-05 Sangamo BioSciences, Inc. Methods and compositions for treating huntington's disease
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
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
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
EP3137476B1 (en) 2014-04-28 2019-10-09 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
CN106413760B (zh) 2014-05-08 2020-01-14 桑格摩生物科学股份有限公司 用于治疗亨廷顿病的方法和组合物
RU2711147C2 (ru) 2014-05-20 2020-01-15 Юниверсити Оф Айова Рисерч Фаундейшн Терапевтические соединения для лечения болезни хантингтона
US20160017327A1 (en) 2014-07-11 2016-01-21 The Johns Hopkins University Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity
CA2955250A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
RU2708237C2 (ru) * 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
JP2017536366A (ja) 2014-11-19 2017-12-07 ロシュ イノベーション センター コペンハーゲン エーエス Ｌｎａキラルホスホロチオエート
HUE043842T2 (hu) 2014-12-24 2019-09-30 Uniqure Ip Bv RNAi-indukált huntingtin gén szuppresszió
US20180023081A1 (en) 2015-02-04 2018-01-25 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
EA201791805A1 (ru) 2015-02-10 2018-05-31 Джензим Корпорейшн ВАРИАНТ СРЕДСТВА ДЛЯ RNAi
CA2980337A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017062862A2 (en) 2015-10-09 2017-04-13 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
AU2017234150B2 (en) 2016-03-13 2021-09-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
MA45188A (fr) 2016-06-03 2019-04-10 Wave Life Sciences Ltd Oligonucléotides, compositions et méthodes associées
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
CN120330183A (zh) 2017-06-02 2025-07-18 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
CN111050806A (zh) 2017-06-02 2020-04-21 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
EP3630789A4 (en) 2017-06-02 2021-06-16 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR THEIR USE
JP7402696B2 (ja) 2017-06-21 2023-12-21 ウェイブ ライフ サイエンシズ リミテッド 合成のための化合物、組成物、及び方法
US11432977B2 (en) 2017-06-26 2022-09-06 Yamaha Hatsudoki Kabushiki Kaisha Power assist wheelchair, power assist unit for wheelchair, control device for power assist wheelchair, control method for power assist wheelchair, and program
WO2019032607A1 (en) 2017-08-08 2019-02-14 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
KR20250123934A (ko) 2017-09-18 2025-08-18 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 제조 기술
US11596646B2 (en) 2017-10-12 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH06220083A (ja) * 1991-03-06 1994-08-09 Polish Acad Of Sci オリゴヌクレオチドとオリゴヌクレオチド類似体の固相合成のための方法と化合物
JP2002520420A (ja) * 1998-07-14 2002-07-09 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 部位特異的キラルホスホロチオエート・ヌクレオシド間結合を有するオリゴヌクレオチド
US20140256578A1 (en) * 2008-05-09 2014-09-11 The University Of British Columbia Methods and compositions for the treatment of huntington's disease
WO2014012081A2 (en) * 2012-07-13 2014-01-16 Ontorii, Inc. Chiral control

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
FEBS LETTERS, 2002, VOL. 528, PP. 48-52, JPN6017026959, ISSN: 0004475816 *
WAN, W. BRAD ET AL., NUCLEIC ACIDS RESEARCH, vol. 42, no. 22, JPN6020023028, 14 November 2014 (2014-11-14), pages 13456 - 13468, ISSN: 0004475817 *

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