JPWO2019200185A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2019200185A5
JPWO2019200185A5 JP2020555234A JP2020555234A JPWO2019200185A5 JP WO2019200185 A5 JPWO2019200185 A5 JP WO2019200185A5 JP 2020555234 A JP2020555234 A JP 2020555234A JP 2020555234 A JP2020555234 A JP 2020555234A JP WO2019200185 A5 JPWO2019200185 A5 JP WO2019200185A5
Authority
JP
Japan
Prior art keywords
independently
heteroatoms
ring
optionally
oligonucleotide
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.)
Pending
Application number
JP2020555234A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021521140A (ja
JP2021521140A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/027109 external-priority patent/WO2019200185A1/en
Publication of JP2021521140A publication Critical patent/JP2021521140A/ja
Publication of JPWO2019200185A5 publication Critical patent/JPWO2019200185A5/ja
Publication of JP2021521140A5 publication Critical patent/JP2021521140A5/ja
Priority to JP2023110962A priority Critical patent/JP2023139036A/ja
Pending legal-status Critical Current

Links

JP2020555234A 2018-04-12 2019-04-11 オリゴヌクレオチド組成物及びその使用方法 Pending JP2021521140A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023110962A JP2023139036A (ja) 2018-04-12 2023-07-05 オリゴヌクレオチド組成物及びその使用方法

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201862656949P 2018-04-12 2018-04-12
US62/656,949 2018-04-12
US201862670709P 2018-05-11 2018-05-11
US62/670,709 2018-05-11
US201862715684P 2018-08-07 2018-08-07
US62/715,684 2018-08-07
US201862723375P 2018-08-27 2018-08-27
US62/723,375 2018-08-27
US201862776432P 2018-12-06 2018-12-06
US62/776,432 2018-12-06
PCT/US2019/027109 WO2019200185A1 (en) 2018-04-12 2019-04-11 Oligonucleotide compositions and methods of use thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023110962A Division JP2023139036A (ja) 2018-04-12 2023-07-05 オリゴヌクレオチド組成物及びその使用方法

Publications (3)

Publication Number Publication Date
JP2021521140A JP2021521140A (ja) 2021-08-26
JPWO2019200185A5 true JPWO2019200185A5 (https=) 2022-04-19
JP2021521140A5 JP2021521140A5 (https=) 2022-04-19

Family

ID=68163789

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020555234A Pending JP2021521140A (ja) 2018-04-12 2019-04-11 オリゴヌクレオチド組成物及びその使用方法
JP2023110962A Pending JP2023139036A (ja) 2018-04-12 2023-07-05 オリゴヌクレオチド組成物及びその使用方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023110962A Pending JP2023139036A (ja) 2018-04-12 2023-07-05 オリゴヌクレオチド組成物及びその使用方法

Country Status (12)

Country Link
US (1) US12552743B2 (https=)
EP (1) EP3775203A4 (https=)
JP (2) JP2021521140A (https=)
KR (1) KR20210005057A (https=)
CN (1) CN112004928A (https=)
AU (2) AU2019252680B2 (https=)
BR (1) BR112020020670A2 (https=)
CA (1) CA3096667A1 (https=)
IL (2) IL277889B2 (https=)
MX (1) MX2020010687A (https=)
SG (1) SG11202009877XA (https=)
WO (1) WO2019200185A1 (https=)

Families Citing this family (73)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
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
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
RU2708237C2 (ru) 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
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
CA3015823A1 (en) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
CN109562122A (zh) 2016-06-03 2019-04-02 波涛生命科学有限公司 寡核苷酸、组合物及其方法
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11597927B2 (en) 2017-06-02 2023-03-07 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
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
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
EP3790596A4 (en) 2018-05-11 2022-04-06 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP3829595A4 (en) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN113748116A (zh) * 2019-03-20 2021-12-03 波涛生命科学有限公司 可用于寡核苷酸制备的技术
US12497615B2 (en) 2019-04-25 2025-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
EP4013767A4 (en) 2019-08-15 2023-10-25 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
JP7842395B2 (ja) * 2019-12-19 2026-04-08 日本新薬株式会社 エクソンスキッピングを可能にするアンチセンス核酸
CN114829599A (zh) * 2019-12-19 2022-07-29 豪夫迈·罗氏有限公司 Scamp3抑制剂用于治疗乙型肝炎病毒感染的用途
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
JP2023526975A (ja) * 2020-05-22 2023-06-26 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組成物及びその方法
CN112430645A (zh) * 2020-12-09 2021-03-02 北京华瑞康源生物科技发展有限公司 一种多重实时荧光pcr法检测人dmd基因拷贝数的相对定量方法及试剂盒
CN114763367B (zh) * 2021-01-14 2024-08-06 施能康生物科技有限公司 化合物、缀合物及其用途
WO2022270585A1 (ja) 2021-06-23 2022-12-29 日本新薬株式会社 アンチセンスオリゴマーの組み合わせ
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
IL309936A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of dystrophinopathy
CA3226457A1 (en) * 2021-07-30 2023-02-02 Amy WALKER Nanoparticles and peptides for the delivery of cargos to muscle cells
EP4124345A1 (en) * 2021-07-30 2023-02-01 4basebio UK Ltd Nanoparticles for muscle delivery
AU2022324471A1 (en) * 2021-08-05 2024-02-15 Sanegene Bio Usa Inc. 1'-alkyl modified ribose derivatives and methods of use
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
IL315089A (en) * 2022-03-02 2024-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof for exon skipping
CN114600803B (zh) * 2022-03-15 2023-05-09 北京协和建昊医药技术开发有限责任公司 一种斑马鱼cyp3a4代谢酶抑制模型、建立方法及应用
EP4536831A2 (en) * 2022-06-10 2025-04-16 Camp4 Therapeutics Corporation Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas
EP4555086A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
AR131145A1 (es) 2022-11-24 2025-02-19 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CA3276262A1 (en) 2022-12-09 2024-06-13 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
EP4669753A1 (en) 2023-02-20 2025-12-31 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
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024256620A1 (en) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurodegenerative disease
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025056973A2 (en) * 2023-09-13 2025-03-20 Haya Therapeutics Sa Compositions and methods for modulating wisp2 super-enhancer-associated rna
AU2024351126A1 (en) * 2023-09-28 2026-04-16 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
AU2024348542A1 (en) * 2023-09-28 2026-04-16 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
CN121909203A (zh) 2023-10-31 2026-04-21 科罗生物公司 包含氨基磷酸酯核苷酸间键的寡核苷酸
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
WO2025199428A1 (en) * 2024-03-21 2025-09-25 Adarx Pharmaceuticals, Inc. Mannose receptor ligand-tropomyosin receptor b ligand conjugates for drug delivery
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
WO2026050243A1 (en) 2024-08-26 2026-03-05 Korro Bio, Inc. Galnac conjugated oligonucleotides for rna editing
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

Family Cites Families (117)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5852188A (en) 1990-01-11 1998-12-22 Isis Pharmaceuticals, Inc. Oligonucleotides having chiral phosphorus linkages
EP0464638B1 (de) 1990-07-02 1997-04-02 Hoechst Aktiengesellschaft Oligonucleotid-analoge mit terminalen 3'-3'-bzw. 5'-5'-Internucleotidverknüpfungen
US5512668A (en) * 1991-03-06 1996-04-30 Polish Academy Of Sciences Solid phase oligonucleotide synthesis using phospholane intermediates
US5216168A (en) 1992-04-01 1993-06-01 G. D. Searle & Co. 2- and 3- amino and azido derivatives of 1,5-iminosugars
US6242589B1 (en) * 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
US20030138797A1 (en) 2001-06-29 2003-07-24 Micrologix Biotech Inc. Nucleic acid-based compounds
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
US8232383B2 (en) 2002-02-20 2012-07-31 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2004007718A2 (en) 2002-07-10 2004-01-22 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna-interference by single-stranded rna molecules
WO2004014933A1 (en) 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
AR040996A1 (es) 2002-08-19 2005-04-27 Coley Pharm Group Inc Acidos nucleicos inmunoestimuladores
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
WO2005028494A1 (ja) 2003-09-02 2005-03-31 Takeshi Wada 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
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
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
WO2006023880A2 (en) 2004-08-23 2006-03-02 Isis Pharmaceuticals, Inc. Compounds and methods for the characterization of oligonucleotides
JP2006248949A (ja) * 2005-03-09 2006-09-21 Univ Nagoya ヌクレオシド誘導体、ヌクレオチド誘導体及びそれらの製造方法
EP1801114B8 (de) 2005-11-23 2009-12-02 Roche Diagnostics GmbH Polynukleotid mit Phosphatmimetikum
EP2147010A1 (en) 2007-04-18 2010-01-27 Roche Diagnostics GmbH Nucleotide with an alpha-phosphate mimetic
AU2008317566B2 (en) * 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
DK2285819T3 (da) 2008-04-04 2013-12-02 Isis Pharmaceuticals Inc Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
US8324366B2 (en) 2008-04-29 2012-12-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for delivering RNAI using lipoproteins
WO2010048549A2 (en) 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
JP5786109B2 (ja) 2008-10-27 2015-09-30 プロセンサ テクノロジーズ ベー.フェー. Duchenne型筋ジストロフィーmRNA前駆体のエクソン45の効率的なスキッピングのための方法及び手段
RU2572826C2 (ru) * 2008-12-02 2016-01-20 Чиралджен, Лтд. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
CA2750561C (en) 2009-01-26 2017-10-10 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
EP2479182B8 (en) 2009-09-16 2016-07-13 Wave Life Sciences Japan, Inc. Novel protecting group for synthesizing rna and derivative thereof
US8859755B2 (en) 2010-03-05 2014-10-14 Chiralgen, Ltd. Method for preparing ribonucleoside phosphorothioate
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
WO2011139911A2 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Lipid formulated single stranded rna
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
JP6093924B2 (ja) 2010-11-30 2017-03-15 株式会社Wave Life Sciences Japan 2’−o−修飾rna
CN103649103A (zh) 2011-06-21 2014-03-19 阿尔尼拉姆医药品有限公司 用于抑制载脂蛋白c-iii(apoc3)基因表达的组合物与方法
US9416154B2 (en) 2011-07-13 2016-08-16 Merck Sharp & Dohme Corp. 5′-substituted nucleoside derivatives and methods of use thereof for the treatment of viral diseases
AU2012284265B2 (en) 2011-07-19 2017-08-17 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP2792746A4 (en) 2011-12-12 2015-09-16 Nat Cerebral & Cardiovascular Ct OLIGONUCLEOTIDE AND THERAPEUTIC FOR HYPERLIPIDEMIA WITH THIS AS AN ACTIVE SUBSTANCE
CN112251436A (zh) 2012-01-27 2021-01-22 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
EP2870246B1 (en) 2012-07-03 2019-09-11 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
CN104684923B (zh) 2012-07-13 2018-09-28 株式会社新日本科学 手性核酸佐剂
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
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
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
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
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
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
AU2015217301A1 (en) 2014-02-11 2016-08-25 Alnylam Pharmaceuticals, Inc. Ketohexokinase (KHK) iRNA compositions and methods of use thereof
SMT201900169T1 (it) 2014-03-12 2019-05-10 Nippon Shinyaku Co Ltd Acido nucleico antisenso
KR101443256B1 (ko) 2014-04-15 2014-09-22 강성덕 슬라이딩 도어형 밀폐 챔버장치
RU2708237C2 (ru) 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
HK1244843A1 (zh) 2014-11-17 2018-08-17 Alnylam Pharmaceuticals, Inc. 载脂蛋白c3(apoc3)irna组合物及其使用方法
WO2016130806A2 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
SMT202000454T1 (it) 2015-04-03 2020-11-10 Univ Massachusetts Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina
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
JP7511326B2 (ja) 2015-10-09 2024-07-05 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組成物およびその方法
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
CA3015823A1 (en) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
WO2017192679A1 (en) 2016-05-04 2017-11-09 Wave Life Sciences Ltd. Methods and compositions of biologically active agents
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
MA45183A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
CN109562122A (zh) 2016-06-03 2019-04-02 波涛生命科学有限公司 寡核苷酸、组合物及其方法
CA3029772A1 (en) 2016-07-05 2018-01-11 Biomarin Technologies B.V. Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
US20190330626A1 (en) 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
WO2018022473A1 (en) 2016-07-25 2018-02-01 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
US11597927B2 (en) 2017-06-02 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP2020522265A (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
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
WO2019002237A1 (en) 2017-06-28 2019-01-03 Roche Innovation Center Copenhagen A/S METHOD OF MULTIPLE COUPLING AND OXIDATION
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
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
WO2019071028A1 (en) 2017-10-04 2019-04-11 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
AR113490A1 (es) 2017-12-12 2020-05-06 Amgen Inc CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
EP3790596A4 (en) 2018-05-11 2022-04-06 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
TWI856973B (zh) 2018-09-19 2024-10-01 美商Ionis製藥公司 Pnpla3表現之調節劑
SG11202105626TA (en) 2018-12-06 2021-06-29 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
KR20210121199A (ko) 2019-02-01 2021-10-07 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법
CN113748116A (zh) 2019-03-20 2021-12-03 波涛生命科学有限公司 可用于寡核苷酸制备的技术
WO2020196662A1 (ja) 2019-03-25 2020-10-01 国立大学法人東京医科歯科大学 二本鎖核酸複合体及びその使用
EP3958872A4 (en) 2019-04-25 2024-07-03 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CA3137741A1 (en) 2019-04-25 2020-10-29 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
AU2020267775A1 (en) 2019-05-09 2021-12-02 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
KR20220070324A (ko) 2019-10-06 2022-05-30 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 이용 방법
CN114585737A (zh) 2019-10-06 2022-06-03 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類PATATIN磷脂酶結構域3(PNPLA3)iRNA組成物及其使用方法
AU2021230473A1 (en) 2020-03-01 2022-09-22 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
TW202202153A (zh) 2020-03-26 2022-01-16 美商艾羅海德製藥公司 用於抑制PNPLA3表現之RNAi劑、其醫藥組成物及使用方法
JP2023526975A (ja) 2020-05-22 2023-06-26 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組成物及びその方法
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
EP4200616A1 (en) 2020-08-24 2023-06-28 Wave Life Sciences Ltd. Immunoassays for detecting wild type huntingtin protein and methods of treatment employing such immunoassays
WO2022046667A1 (en) 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cells and non-human animals engineered to express adar1 and uses thereof
US20230416847A1 (en) 2020-09-24 2023-12-28 Mayo Foundation For Medical Education And Research Screening platforms
IL300882A (en) 2020-10-02 2023-04-01 Ionis Pharmaceuticals Inc Methods for reducing APOCIII expression
EP4240849A4 (en) 2020-11-08 2026-04-01 Wave Life Sciences Ltd OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES
CN116507724A (zh) 2020-11-08 2023-07-28 波涛生命科学有限公司 寡核苷酸组合物及其方法
JP2024536088A (ja) 2021-09-26 2024-10-04 ウェイブ ライフ サイエンシズ リミテッド Mecp2転写物を編集するための組成物及びその方法
AU2022352779A1 (en) 2021-09-26 2024-03-28 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP4422645A4 (en) 2021-10-27 2025-12-03 Wave Life Sciences Ltd OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODS OF USE
CA3236136A1 (en) 2021-10-27 2023-05-04 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
EP4476202A1 (en) 2022-02-11 2024-12-18 Wave Life Sciences Ltd. Stereoselective technologies for chiral compounds
IL315089A (en) 2022-03-02 2024-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof for exon skipping
IL316178A (en) 2022-04-15 2024-12-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods related thereto
CN119452084A (zh) 2022-05-12 2025-02-14 波涛生命科学有限公司 寡核苷酸组合物及其方法
JP2025526712A (ja) 2022-08-11 2025-08-15 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組成物及びその方法
WO2025151895A1 (en) 2024-01-12 2025-07-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Similar Documents

Publication Publication Date Title
JPWO2019200185A5 (https=)
JP2023139036A5 (https=)
JP2021521140A5 (https=)
JP4628437B2 (ja) ヌクレオチドアナログ組成物および合成方法
KR100858464B1 (ko) 콤브레타스타틴 a-4 인산염 프로드럭 모노- 및 디-유기아민 염, 모노- 및 디-아미노산 염, 그리고 모노- 및디-아미노산 에스테르 염
JP2024105256A5 (https=)
TWI630201B (zh) 化合物及其調節血紅素之用途
CN113214278B (zh) 新型Jak1选择性抑制剂及其用途
JPWO2020191252A5 (https=)
JP2004514663A5 (https=)
CN101885742A (zh) 一种手性双膦酰二胺化合物及其合成方法
BR112020021949A2 (pt) ligantes dirigidos à integrina e seus usos
CN101805369B (zh) 二硬脂酰磷酰乙醇胺及其氨基聚乙二醇化衍生物的制备方法
JP5850842B2 (ja) 骨代謝疾患を治療するためのビスホスホネート化合物
JPWO2022125790A5 (https=)
CN1617883A (zh) 苯并氮杂环庚三烯化合物的固体盐及其在制备药物化合物中的应用
JP7075681B2 (ja) 立体制御オリゴヌクレオチド合成用光学活性セグメントおよびその製造方法、ならびにそれを用いた立体制御オリゴヌクレオチドの合成方法
EP0112743A1 (fr) Nouveaux dérivés de tricyclophosphazènes, procédé pour leur préparation et leur application en tant que médicament
CN102161631B (zh) 一种手性α-二氟甲基取代的α-季碳氨基腈的合成方法
KR101548724B1 (ko) 고체 형태의 항바이러스제 및 이의 제조방법
JP7345167B2 (ja) ポリ(カルバメート)-核酸医薬コンジュゲート、ポリ(カルバメート)核酸医薬コンジュゲートの凝集粒子、及び凝集粒子の製造方法
JP2002529473A (ja) ポリヌクレオチド架橋成分
JP2002511504A (ja) イミダゾールから形成される化合物およびポリマー
CN109608492B (zh) 一种用于骨质疏松的二膦酸化合物及其制备方法
JPS59190998A (ja) 新規ビス(2,2−ジメチル−1−アジリジニル)ホスフインアミド類及びその腫瘍阻止組成物