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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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 (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
EP3234134B1 (en) 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Targeted rna editing
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
IL294124B2 (en) 2015-12-10 2024-02-01 Ptc Therapeutics Inc Methods for treating huntington's disease
CN108779132B (zh) 2016-03-13 2022-04-15 波涛生命科学有限公司 用于亚磷酰胺和寡核苷酸合成的组合物和方法
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
JP2019520339A (ja) 2016-06-03 2019-07-18 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、その組成物および方法
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
US11873316B2 (en) 2016-11-23 2024-01-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
KR20240006023A (ko) 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
PT3554552T (pt) 2016-12-19 2022-10-03 Sarepta Therapeutics Inc Conjugados de oligómero de skipping de exão para distrofia muscular
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
CA3065523A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP2020522510A (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
US11407753B2 (en) 2017-06-05 2022-08-09 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
US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
EP3664816A4 (en) 2017-08-08 2021-05-19 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES
CA3072110A1 (en) 2017-09-18 2019-03-21 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
WO2019067981A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
EP3687547A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular 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
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
MX2020009957A (es) 2018-03-27 2021-01-15 Ptc Therapeutics Inc Compuestos para el tratamiento de enfermedad de hungtinton.
SG11202009877XA (en) 2018-04-12 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
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
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
EP3814345B8 (en) 2018-06-27 2024-10-30 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
PL3814357T3 (pl) 2018-06-27 2024-09-16 Ptc Therapeutics, Inc. Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona
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
IL318474A (en) 2018-12-13 2025-03-01 Sarepta Therapeutics Inc Axon-skipping oligomer complexes for muscular dystrophy
CN113423385A (zh) * 2019-02-01 2021-09-21 波涛生命科学有限公司 寡核苷酸组合物及其方法
JP2022525541A (ja) 2019-03-20 2022-05-17 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調製に有用な技術
WO2020214763A1 (en) 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
SG11202111387YA (en) * 2019-04-25 2021-11-29 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
WO2020231977A1 (en) 2019-05-13 2020-11-19 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
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
MX2022014606A (es) 2020-05-22 2023-03-08 Wave Life Sciences Ltd Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas.
EP4165205A1 (en) * 2020-06-15 2023-04-19 CureVac SE 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
EP4396354A4 (en) * 2021-09-26 2025-12-03 Wave Life Sciences Ltd OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES
WO2023055774A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
JP2025524566A (ja) 2022-07-15 2025-07-30 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ Ａｄａｒ媒介ｒｎａ編集のためのオリゴヌクレオチドおよびその使用
US20260027237A1 (en) 2022-07-15 2026-01-29 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
AU2023390632A1 (en) 2022-12-09 2025-07-10 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
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
EP4689110A1 (en) 2023-03-24 2026-02-11 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
EP4688143A1 (en) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of liver disease
TW202442246A (zh) 2023-04-27 2024-11-01 美商薩羅塔治療公司 用於治療慢性腎病之反義寡聚物
TW202516003A (zh) 2023-06-16 2025-04-16 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療神經退化性疾病之反義寡核苷酸
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
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
WO2026060374A2 (en) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of 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
CA2176256A1 (en) 1993-11-16 1995-05-26 Lyle John Arnold, Jr. Synthetic oligomers having chirally pure phosphonate internucleosidyl linkages mixed with non-phosphonate internucleosidyl linkages
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
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
US7589189B2 (en) 2003-05-14 2009-09-15 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
JP4580870B2 (ja) 2003-09-02 2010-11-17 株式会社キラルジェン 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
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
DK2821085T3 (da) * 2003-09-12 2020-08-03 Univ Massachusetts Rna-interferens til behandling af "gain-of-function"-forstyrrelser
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2008005562A2 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
WO2005070859A1 (ja) 2004-01-27 2005-08-04 Takeshi Wada 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
WO2006121960A2 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing 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
JP5111385B2 (ja) * 2005-10-28 2013-01-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド ハンチンチン遺伝子の発現を抑制するための組成物および方法
EP1991677A2 (en) 2006-01-26 2008-11-19 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
ES2526295T5 (es) 2006-10-18 2021-05-04 Ionis Pharmaceuticals Inc Compuestos antisentido
EP2164865A2 (en) 2007-06-13 2010-03-24 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
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
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
WO2010118263A1 (en) 2009-04-08 2010-10-14 University Of Massachusetts Single-nucleotide polymorphism (snp) targeting therapies for the treatment of huntington's disease
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
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
NZ714887A (en) 2009-09-11 2019-07-26 Ionis Pharmaceuticals Inc Modulation of huntingtin expression
ES2586683T3 (es) 2009-09-16 2016-10-18 Wave Life Sciences Japan, Inc. Grupo protector novedoso para sintetizar ARN y derivados del mismo
CA2785727C (en) 2009-12-31 2020-01-07 Curna, Inc. Treatment of insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to irs2 and transcription factor e3 (tfe3)
US8957040B2 (en) 2010-02-08 2015-02-17 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
CA2789038A1 (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
JP5847700B2 (ja) 2010-03-05 2016-01-27 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ リボヌクレオシドホスホロチオエートの製造方法
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
CA2817256A1 (en) 2010-11-12 2012-05-18 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
EP2673361B1 (en) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2012109667A1 (en) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
US10202599B2 (en) 2011-08-11 2019-02-12 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US10066228B2 (en) 2011-11-30 2018-09-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
CA2879066C (en) 2012-07-13 2019-08-13 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
EP3459549B1 (en) 2012-10-12 2022-04-06 Ionis 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
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
WO2015070212A1 (en) 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 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
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 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
BR112016016400A2 (pt) * 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
WO2015168172A1 (en) 2014-04-28 2015-11-05 Isis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
WO2015171932A1 (en) 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
EP3146051B8 (en) 2014-05-20 2019-11-27 University of Iowa Research Foundation Huntington's disease therapeutic compounds
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
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
RU2708237C2 (ru) * 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
WO2016079183A1 (en) 2014-11-19 2016-05-26 Roche Innovation Center Copenhagen A/S Lna chiral phosphorothioates
HRP20190992T1 (hr) 2014-12-24 2019-09-20 Uniqure Ip B.V. Suprimiranje gena za huntingtin inducirano s rnai
WO2016127002A1 (en) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
CR20170406A (es) 2015-02-10 2017-11-14 Genzyme Corp Arni variante
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
CN108779132B (zh) 2016-03-13 2022-04-15 波涛生命科学有限公司 用于亚磷酰胺和寡核苷酸合成的组合物和方法
CN109462995A (zh) 2016-05-04 2019-03-12 波涛生命科学有限公司 生物活性剂的方法和组合物
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
JP2019520339A (ja) 2016-06-03 2019-07-18 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、その組成物および方法
WO2018022473A1 (en) 2016-07-25 2018-02-01 Wave Life Sciences Ltd. Phasing
US11873316B2 (en) 2016-11-23 2024-01-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
JP2020522510A (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
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
CA3065523A1 (en) 2017-06-02 2018-12-06 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
WO2019003260A1 (ja) 2017-06-26 2019-01-03 ヤマハ発動機株式会社 電動アシスト車いす、車いす用電動アシストユニット、電動アシスト車いすの制御装置、電動アシスト車いすの制御方法、及びプログラム
EP3664816A4 (en) 2017-08-08 2021-05-19 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES
CA3072110A1 (en) 2017-09-18 2019-03-21 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES

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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