JP2018537952A5 - - Google Patents

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JP2018537952A5
JP2018537952A5 JP2018517191A JP2018517191A JP2018537952A5 JP 2018537952 A5 JP2018537952 A5 JP 2018537952A5 JP 2018517191 A JP2018517191 A JP 2018517191A JP 2018517191 A JP2018517191 A JP 2018517191A JP 2018537952 A5 JP2018537952 A5 JP 2018537952A5
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composition
oligonucleotides
oligonucleotide
pattern
base sequence
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JP2018517191A
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JP2018537952A (ja
JP7511326B2 (ja
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Priority claimed from PCT/US2016/056123 external-priority patent/WO2017062862A2/en
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Priority to JP2022052547A priority Critical patent/JP7696310B2/ja
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Priority to JP2024207586A priority patent/JP2025029016A/ja
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JP2018517191A 2015-10-09 2016-10-07 オリゴヌクレオチド組成物およびその方法 Active JP7511326B2 (ja)

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JP2022052547A JP7696310B2 (ja) 2015-10-09 2022-03-28 オリゴヌクレオチド組成物およびその方法
JP2024207586A JP2025029016A (ja) 2015-10-09 2024-11-28 オリゴヌクレオチド組成物およびその方法

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US201562239839P 2015-10-09 2015-10-09
US62/239,839 2015-10-09
US201662331966P 2016-05-04 2016-05-04
US201662331961P 2016-05-04 2016-05-04
US62/331,961 2016-05-04
US62/331,966 2016-05-04
PCT/US2016/056123 WO2017062862A2 (en) 2015-10-09 2016-10-07 Oligonucleotide compositions and methods thereof

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JP2018537952A JP2018537952A (ja) 2018-12-27
JP2018537952A5 true JP2018537952A5 (https=) 2019-11-14
JP7511326B2 JP7511326B2 (ja) 2024-07-05

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JP2018517191A Active JP7511326B2 (ja) 2015-10-09 2016-10-07 オリゴヌクレオチド組成物およびその方法
JP2019518395A Pending JP2019530698A (ja) 2015-10-09 2017-10-06 オリゴヌクレオチド組成物およびその方法
JP2022052547A Active JP7696310B2 (ja) 2015-10-09 2022-03-28 オリゴヌクレオチド組成物およびその方法
JP2024207586A Pending JP2025029016A (ja) 2015-10-09 2024-11-28 オリゴヌクレオチド組成物およびその方法

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JP2022052547A Active JP7696310B2 (ja) 2015-10-09 2022-03-28 オリゴヌクレオチド組成物およびその方法
JP2024207586A Pending JP2025029016A (ja) 2015-10-09 2024-11-28 オリゴヌクレオチド組成物およびその方法

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US (4) US20190127733A1 (https=)
EP (2) EP3359523A4 (https=)
JP (4) JP7511326B2 (https=)
KR (1) KR102933365B1 (https=)
CN (3) CN108137492B (https=)
AU (4) AU2016334232B2 (https=)
BR (1) BR112018006636B1 (https=)
CA (2) CA2999772A1 (https=)
CL (1) CL2018000818A1 (https=)
CO (1) CO2018004758A2 (https=)
CR (1) CR20180233A (https=)
EC (1) ECSP18032914A (https=)
IL (2) IL295755A (https=)
MA (2) MA46427A (https=)
MX (2) MX2018003992A (https=)
NI (1) NI201800045A (https=)
PE (1) PE20181353A1 (https=)
PH (1) PH12018500719A1 (https=)
SG (1) SG10201912902TA (https=)
SV (1) SV2018005661A (https=)
TW (1) TW201722439A (https=)
WO (2) WO2017062862A2 (https=)

Families Citing this family (134)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8158768B2 (en) * 2002-12-23 2012-04-17 Dynavax Technologies Corporation Immunostimulatory sequence oligonucleotides and methods of using the same
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
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
AU2012284265B2 (en) 2011-07-19 2017-08-17 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
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
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
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
US10676737B2 (en) 2014-12-17 2020-06-09 Proqr Therapeutics Ii B.V. Targeted RNA editing
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
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
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
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
JP2019507579A (ja) 2015-10-28 2019-03-22 クリスパー セラピューティクス アーゲー デュシェンヌ型筋ジストロフィーの処置のための材料および方法
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
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
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
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
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated 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
SMT202200366T1 (it) 2016-12-19 2022-11-18 Sarepta Therapeutics Inc Coniugati di oligomeri per salto di esone per distrofia muscolare
IL297528A (en) 2016-12-19 2022-12-01 Sarepta Therapeutics Inc Exon-skipping oligomer conjugates for muscular dystrophy
SI3554554T1 (sl) 2016-12-19 2023-02-28 Sarepta Therapeutics, Inc. Oligomerni konjugati, ki preskakujejo ekson za mišično distrofijo
GB2574769A (en) 2017-03-03 2019-12-18 Univ California RNA Targeting of mutations via suppressor tRNAs and deaminases
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
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
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ 2テイル自己デリバリー型siRNAおよび関連方法
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
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
WO2019067979A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
WO2019067975A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
JP2020536058A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを処置するための併用療法
EP3694530A4 (en) * 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
CA3086485A1 (en) * 2017-12-21 2019-06-27 Alnylam Pharmaceuticals, Inc. Chirally-enriched double-stranded rna agents
JP6933768B2 (ja) 2018-03-15 2021-09-08 栄研化学株式会社 一塩基多型検出用オリゴヌクレオチドプローブ、及びシス型−トランス型判別方法
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
WO2019217459A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
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
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
TW202020153A (zh) 2018-07-27 2020-06-01 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
EP3829595A4 (en) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
JP7627042B2 (ja) * 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ O-メチルリッチ完全安定化オリゴヌクレオチド
IL282818B1 (en) 2018-11-02 2026-04-01 Biomarin Tech Bv Bispecific antisense oligonucleotides for dystrophin exon skipping
SG11202105626TA (en) * 2018-12-06 2021-06-29 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
MX2021006737A (es) 2018-12-13 2021-09-23 Sarepta Therapeutics Inc Conjugados de oligómeros de salto de exones para distrofia muscular.
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
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
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
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
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
JP2022545118A (ja) * 2019-08-23 2022-10-25 ユニバーシティー オブ マサチューセッツ O-メチルリッチ完全安定化オリゴヌクレオチド
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
CA3151789A1 (en) * 2019-09-19 2021-03-25 Arnay Sciences, Llc Compounds and methods useful for modulating gene splicing
WO2021062251A2 (en) * 2019-09-25 2021-04-01 University Of Massachusetts Targeting rlim to modulate body weight and obesity
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
KR20220070324A (ko) * 2019-10-06 2022-05-30 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 이용 방법
WO2021113270A1 (en) 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing
CA3158528A1 (en) 2019-12-23 2021-07-01 Jim SWILDENS Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease
CN118109468A (zh) 2019-12-26 2024-05-31 日本新药株式会社 诱导外显子50的跳读的反义核酸
US20230140736A1 (en) 2020-02-28 2023-05-04 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
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
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
EP4222262A1 (en) 2020-09-30 2023-08-09 BioMarin Technologies B.V. Antisense oligonucleotides targeting the exon 51 of dystrophin gene
CN117120456A (zh) * 2020-12-11 2023-11-24 卫材R&D管理有限公司 聚-吗啉代寡核苷酸缺口体
IL303886A (en) 2020-12-23 2023-08-01 Flagship Pioneering Inc Compositions of modified trems and uses thereof
MX2023012559A (es) 2021-04-30 2023-12-15 Sarepta Therapeutics Inc Métodos de tratamiento para la distrofia muscular.
AU2022299169A1 (en) 2021-06-23 2024-02-08 Beth Israel Deaconess Medical Center, Inc. Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
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
IL309936A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of dystrophinopathy
AU2022312170A1 (en) 2021-07-16 2024-02-08 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Oligonucleotide for inhibiting quaking activity
MX2024003690A (es) 2021-09-30 2024-06-19 Sarepta Therapeutics Inc Oligonucleótidos antisentido que tienen una o más unidades abásicas.
TWI866002B (zh) * 2021-12-29 2024-12-11 財團法人生物技術開發中心 反義寡核苷酸及其用途
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
JP2025509438A (ja) 2022-03-17 2025-04-11 サレプタ セラピューティクス, インコーポレイテッド ホスホロジアミデートモルホリノオリゴマーコンジュゲート
US20250339553A1 (en) 2022-04-15 2025-11-06 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
US20250250629A1 (en) * 2022-04-29 2025-08-07 Singular Genomics Systems, Inc. Nucleotide cyclic cleavable moieties and uses thereof
WO2023215781A1 (en) * 2022-05-05 2023-11-09 Biomarin Pharmaceutical Inc. Method of treating duchenne muscular dystrophy
PE20251277A1 (es) 2022-05-06 2025-05-14 Academisch Ziekenhuis Leiden Oligonucleotido
EP4282964A1 (en) * 2022-05-23 2023-11-29 Universitat de Valéncia Oligonucleotides conjugated to oleic acid and uses thereof
US20250179492A1 (en) 2022-06-22 2025-06-05 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
EP4555086A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucleotides for adar-mediated rna editing and use thereof
JP2025527208A (ja) 2022-07-25 2025-08-20 ヴィコ・セラピューティクス・ベー・フェー Appの異常なプロセシングに関連する疾患又は病態の治療用アンチセンスオリゴヌクレオチド
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
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
WO2024196804A1 (en) * 2023-03-17 2024-09-26 The Regents Of The University Of California Factor viii splice-modulating antisense oligonucleotides and methods of use
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
US20250066776A1 (en) 2023-04-27 2025-02-27 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of chronic kidney disease
CN121889504A (zh) * 2023-05-19 2026-04-17 瑞博威治疗公司 调节atxn2表达的组合物和方法
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
KR20260028817A (ko) * 2023-08-02 2026-03-04 가부시키가이샤 닛폰 쇼쿠바이 올리고뉴클레오티드의 제조 방법
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
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
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
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
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
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
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic 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 (99)

* 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
DE69232699T2 (de) * 1991-10-15 2003-02-06 Isis Pharmaceutical, Inc. Über chirale phosphoratome gebundene oligonukleotide
US5856465A (en) * 1996-05-24 1999-01-05 Polska Akademia Nauk Centrum Badan Molekularnych I Makromolekularnych Compositions and methods for the synthesis of chirally pure organophosphorus nucleoside derivatives
US5804384A (en) * 1996-12-06 1998-09-08 Vysis, Inc. Devices and methods for detecting multiple analytes in samples
US6242589B1 (en) * 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
US20020082227A1 (en) * 1999-09-30 2002-06-27 Scott Henry Use of oligonucleotides for inhibition of complement activation
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US6858720B2 (en) * 2001-10-31 2005-02-22 Agilent Technologies, Inc. Method of synthesizing polynucleotides using ionic liquids
WO2004044134A2 (en) * 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Phosphorous-linked oligomeric compounds and their use in gene modulation
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
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
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
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
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
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
MX2007002214A (es) 2004-09-03 2007-05-07 Yuhan Corp Derivados de pirrolo[3,2-c]piridina y procesos para su preparacion.
WO2006096963A1 (en) * 2005-03-15 2006-09-21 Mcgill University Ionic liquid supported synthesis
NZ563206A (en) 2005-04-22 2009-09-25 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mRNA by interfering with the binding or SR proteins and by interfering with secondary RNA structure
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
US20130129752A1 (en) 2006-04-25 2013-05-23 Immune Disease Institute, Inc. Targeted delivery to leukocytes using protein carriers
US8097596B2 (en) 2006-06-30 2012-01-17 Lakewood-Amedex, Inc. Compositions and methods for the treatment of muscle wasting
AU2008317566B2 (en) * 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US20110130440A1 (en) * 2008-03-26 2011-06-02 Alnylam Pharmaceuticals, Inc. Non-natural ribonucleotides, and methods of use thereof
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
US20100105054A1 (en) 2008-10-15 2010-04-29 Cincinnati Children's Hospital Medical Center Gene expression in duchenne muscular dystrophy
CN112574988A (zh) 2008-10-24 2021-03-30 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
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
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
US20120156138A1 (en) 2009-04-14 2012-06-21 Smith Larry J Methods and Compositions for the Treatment of Medical Conditions Involving Cellular Reprogramming
ES2618572T3 (es) 2009-05-08 2017-06-21 Curna, Inc. Tratamiento de enfermedades relacionadas con la familia de la distrofina mediante inhibición de un transcrito antisentido natural para la familia de dmd
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
EP2479182B8 (en) 2009-09-16 2016-07-13 Wave Life Sciences Japan, Inc. Novel protecting group for synthesizing rna and derivative thereof
LT2499249T (lt) 2009-11-12 2018-12-27 The University Of Western Australia Priešprasmės molekulės ir patologijų gydymo būdai
WO2011064552A1 (en) 2009-11-30 2011-06-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
WO2011078797A2 (en) 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
US8859755B2 (en) 2010-03-05 2014-10-14 Chiralgen, Ltd. Method for preparing ribonucleoside phosphorothioate
EP2582397A4 (en) * 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
JP6093924B2 (ja) 2010-11-30 2017-03-15 株式会社Wave Life Sciences Japan 2’−o−修飾rna
EP2672977A1 (en) 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
DE102011005259A1 (de) * 2011-03-08 2012-09-13 Olympus Winter & Ibe Gmbh Verfahren und System zur Darstellung videoendoskopischer Bilddaten eines Videoendoskops
AU2012284265B2 (en) 2011-07-19 2017-08-17 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP3453761A1 (en) * 2011-08-29 2019-03-13 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
US20140080896A1 (en) * 2011-08-30 2014-03-20 The Regents Of The University Of California Identification of small molecules that facilitate therapeutic exon skipping
JP2015502365A (ja) * 2011-12-12 2015-01-22 オンコイミューニン,インコーポレイティド オリゴヌクレオチドのイン−ビボ送達
CN117721110A (zh) 2011-12-28 2024-03-19 日本新药株式会社 反义核酸
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
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
CN104684923B (zh) 2012-07-13 2018-09-28 株式会社新日本科学 手性核酸佐剂
US10077443B2 (en) 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
NZ731587A (en) 2013-03-14 2021-07-30 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
ES2770667T3 (es) 2013-06-27 2020-07-02 Roche Innovation Ct Copenhagen As Oligómeros antisentido y conjugados que se dirigen a PCSK9
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ma公司 用於調節τ蛋白表現之組合物
KR20160083876A (ko) * 2013-11-14 2016-07-12 로슈 이노베이션 센터 코펜하겐 에이/에스 ApoB 안티센스 접합체 화합물
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
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
SMT201900169T1 (it) 2014-03-12 2019-05-10 Nippon Shinyaku Co Ltd Acido nucleico antisenso
PT3137605T (pt) 2014-05-01 2020-12-18 Ionis Pharmaceuticals Inc Composições e métodos para modulação da expressão de angiopoietina de tipo 3
RU2695430C2 (ru) 2014-06-17 2019-07-23 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
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
SI3351633T1 (sl) 2015-09-15 2020-09-30 Nippon Shinyaku Co., Ltd. Protismiselna nukleinska kislina
JP6875684B2 (ja) 2015-09-16 2021-05-26 日本新薬株式会社 筋萎縮症治療用アンチセンス核酸
US10373069B2 (en) 2015-09-26 2019-08-06 Intel Corporation Technologies for platform-targeted machine learning
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
US10108542B2 (en) * 2016-01-04 2018-10-23 Avalanche Technology, Inc. Serial link storage interface (SLSI) hybrid block storage
US20170210647A1 (en) * 2016-01-21 2017-07-27 Veolia Water Solutions & Technologies Support Process for reducing selenium from an ion-exchange or adsorption media brine
CA3015823A1 (en) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
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
WO2017192679A1 (en) * 2016-05-04 2017-11-09 Wave Life Sciences Ltd. Methods and compositions of biologically active agents
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
RU2016138168A (ru) 2016-09-26 2018-03-29 Оксфордский Университет Аналоги олигонуклеотидов как средства коррекции сплайсинга для лечения мышечной дистрофии дюшена
US9763168B1 (en) * 2016-09-30 2017-09-12 T-Mobile Usa, Inc. Blocked device checking in roaming scenarios
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
RU2740501C2 (ru) 2017-02-21 2021-01-14 Федеральное государственное бюджетное учреждение науки Институт химической биологии и фундаментальной медицины Сибирского отделения Российской академии наук (ИХБФМ СО РАН) МОДИФИЦИРОВАННЫЕ ОЛИГОНУКЛЕОТИДЫ, АКТИВИРУЮЩИЕ РНКазу Н
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
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
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES

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