JP2016529230A5 - - Google Patents

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JP2016529230A5
JP2016529230A5 JP2016525825A JP2016525825A JP2016529230A5 JP 2016529230 A5 JP2016529230 A5 JP 2016529230A5 JP 2016525825 A JP2016525825 A JP 2016525825A JP 2016525825 A JP2016525825 A JP 2016525825A JP 2016529230 A5 JP2016529230 A5 JP 2016529230A5
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oligonucleotide
ligand
linker
biologically active
conjugate
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JP2016525825A
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JP2016529230A (ja
JP6702862B2 (ja
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Priority claimed from PCT/US2014/046425 external-priority patent/WO2015006740A2/en
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Priority to JP2020081749A priority Critical patent/JP7296916B2/ja
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Publication of JP6702862B2 publication Critical patent/JP6702862B2/ja
Priority to JP2023060071A priority patent/JP7676460B2/ja
Priority to JP2025075070A priority patent/JP2025107295A/ja
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JP2016525825A 2013-07-11 2014-07-11 オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法 Active JP6702862B2 (ja)

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Application Number Priority Date Filing Date Title
JP2020081749A JP7296916B2 (ja) 2013-07-11 2020-05-07 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法
JP2023060071A JP7676460B2 (ja) 2013-07-11 2023-04-03 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法
JP2025075070A JP2025107295A (ja) 2013-07-11 2025-04-30 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法

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US201361845279P 2013-07-11 2013-07-11
US61/845,279 2013-07-11
PCT/US2014/046425 WO2015006740A2 (en) 2013-07-11 2014-07-11 Oligonucleotide-ligand conjugates and process for their preparation

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JP2020081749A Division JP7296916B2 (ja) 2013-07-11 2020-05-07 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法

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JP2016529230A JP2016529230A (ja) 2016-09-23
JP2016529230A5 true JP2016529230A5 (https=) 2017-08-24
JP6702862B2 JP6702862B2 (ja) 2020-06-03

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JP2016525825A Active JP6702862B2 (ja) 2013-07-11 2014-07-11 オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法
JP2020081749A Active JP7296916B2 (ja) 2013-07-11 2020-05-07 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法
JP2023060071A Active JP7676460B2 (ja) 2013-07-11 2023-04-03 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法
JP2025075070A Pending JP2025107295A (ja) 2013-07-11 2025-04-30 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法

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JP2020081749A Active JP7296916B2 (ja) 2013-07-11 2020-05-07 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法
JP2023060071A Active JP7676460B2 (ja) 2013-07-11 2023-04-03 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法
JP2025075070A Pending JP2025107295A (ja) 2013-07-11 2025-04-30 オリゴヌクレオチド-リガンドコンジュゲートおよびそれらの調製方法

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US (3) US10808246B2 (https=)
EP (3) EP3019200B1 (https=)
JP (4) JP6702862B2 (https=)
AU (4) AU2014287002A1 (https=)
CA (2) CA3177846A1 (https=)
DK (1) DK3019200T3 (https=)
WO (1) WO2015006740A2 (https=)

Families Citing this family (131)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
EA036496B1 (ru) * 2014-05-01 2020-11-17 Ионис Фармасьютикалз, Инк. Конъюгированные олигонуклеотиды для модулирования экспрессии фактора комплемента b
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3151839A4 (en) * 2014-06-06 2018-02-28 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
EP3303365A4 (en) * 2015-05-29 2019-04-24 Ascendis Pharma Inc. PRODRUGS WITH A PYROGLUTAMATE LINKER
WO2017010575A1 (ja) * 2015-07-16 2017-01-19 協和発酵キリン株式会社 β2GPI遺伝子発現抑制核酸複合体
CN105524124B (zh) * 2015-12-31 2018-10-30 天津英吉诺科技有限公司 一种n-乙酰氨基半乳糖的绿色合成方法
AU2017213404A1 (en) 2016-01-29 2018-09-20 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
CA3011946A1 (en) 2016-03-07 2017-09-14 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
WO2017178656A1 (en) 2016-04-14 2017-10-19 Roche Innovation Center Copenhagen A/S TRITYL-MONO-GalNAc COMPOUNDS AND THEIR USE
US11390867B2 (en) 2016-04-29 2022-07-19 Nanyang Technological University G-quadruplex-containing antisense oligonucleotides
TWI746590B (zh) * 2016-06-30 2021-11-21 日商協和麒麟股份有限公司 核酸複合體
WO2018013525A1 (en) 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
US10781175B2 (en) * 2016-07-15 2020-09-22 Am Chemicals Llc Solid supports and phosphoramidite building blocks for oligonucleotide conjugates
SG11201901841TA (en) 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
US11400161B2 (en) 2016-10-06 2022-08-02 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
EP3594346A4 (en) 2017-03-10 2020-12-16 National Center For Child Health And Development ANTISENS OLIGONUCLEOTIDE AND COMPOSITION FOR THE PREVENTION OR TREATMENT OF TYPE IA GLYCOGEN STORAGE DISEASE
KR20190123351A (ko) * 2017-03-22 2019-10-31 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 개질된 올리고뉴클레오티드 및 이의 치료 용도
DK3607069T3 (da) 2017-04-05 2022-11-21 Silence Therapeutics Gmbh Produkter og sammensætninger
KR102677783B1 (ko) 2017-04-28 2024-06-21 쿄와 기린 가부시키가이샤 올리고뉴클레오티드 유도체 또는 그 염
TW201920668A (zh) * 2017-08-02 2019-06-01 日商協和醱酵麒麟有限公司 核酸複合體
WO2019027015A1 (ja) * 2017-08-02 2019-02-07 協和発酵キリン株式会社 核酸複合体
EA202090720A1 (ru) * 2017-09-14 2020-07-07 Янссен Байофарма, Инк. ПРОИЗВОДНЫЕ GalNAc
WO2019105404A1 (zh) * 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110945131B (zh) * 2017-12-01 2024-05-28 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
EP3719126B1 (en) 2017-12-01 2025-01-01 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
DK3719128T3 (da) * 2017-12-01 2025-03-03 Suzhou Ribo Life Science Co Ltd Dobbelt-strenget oligonukleotid, sammensætning og konjugat bestaende dobbelt-strenget oligonukleotid, fremgangsmåde herfor og anvendelse heraf
CA3083970A1 (en) * 2017-12-01 2019-06-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
EP3719127A4 (en) 2017-12-01 2021-10-20 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE
CN110944675B9 (zh) * 2017-12-01 2024-08-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
AU2018394875B2 (en) 2017-12-29 2023-08-03 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
WO2019140102A1 (en) 2018-01-10 2019-07-18 Translate Bio Ma, Inc. Compositions and methods for facilitating delivery of synthetic nucleic acids to cells
KR102398295B1 (ko) * 2018-03-09 2022-05-17 다이이찌 산쿄 가부시키가이샤 당원병 Ia형 치료약
EP3775209A1 (en) * 2018-04-05 2021-02-17 Silence Therapeutics GmbH Sirnas with at least two ligands at different ends
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
WO2020038377A1 (zh) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
US11896674B2 (en) 2018-09-30 2024-02-13 Suzhou Ribo Life Science Co., Ltd. SiRNA conjugate, preparation method therefor and use thereof
GB2579253A (en) * 2018-11-28 2020-06-17 Pedanius Therapeutics Ltd Antibacterial antisense agents
AU2019390097A1 (en) 2018-11-30 2021-07-15 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
WO2020135673A1 (zh) 2018-12-28 2020-07-02 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN111377985B (zh) * 2018-12-29 2023-11-10 苏州瑞博生物技术股份有限公司 化合物和缀合物及其制备方法和用途
CN111377984B (zh) * 2018-12-29 2024-03-05 苏州瑞博生物技术股份有限公司 化合物和缀合物及其制备方法和用途
CN113330117B (zh) * 2019-01-18 2024-05-28 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
US12590304B2 (en) 2019-05-22 2026-03-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
AU2020280439B2 (en) * 2019-05-22 2025-08-14 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
KR20220011689A (ko) * 2019-05-22 2022-01-28 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 약제학적 조성물, 접합체, 제조 방법, 및 용도
JP7606758B2 (ja) 2019-05-22 2024-12-26 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
CN111973619B (zh) * 2019-05-23 2024-01-30 苏州瑞博生物技术股份有限公司 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
CN111973618B (zh) * 2019-05-23 2024-02-02 苏州瑞博生物技术股份有限公司 核酸、药物组合物与siRNA缀合物及制备方法和用途
US20220315929A1 (en) 2019-05-24 2022-10-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate, preparation method therefor and use thereof
CN113795280B (zh) * 2019-05-24 2024-04-05 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN113795582B (zh) 2019-05-24 2024-05-24 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN112007040B (zh) * 2019-05-31 2024-01-30 苏州瑞博生物技术股份有限公司 用于治疗乙型病毒性肝炎的联合用药物
US20230076803A1 (en) 2019-08-29 2023-03-09 Suzhou Ribo Life Science Co., Ltd. Compound and drug conjugate, and preparation method and use thereof
IL291251B1 (en) 2019-09-10 2026-02-01 Daiichi Sankyo Co Ltd Oligonucleotide-galnac conjugate for targeted delivery to the liver and method for its production
CN115003330A (zh) 2019-09-23 2022-09-02 科纳克斯资本 作为疫苗和诊断工具的新糖缀合物
BR112022007795A2 (pt) * 2019-11-06 2022-07-05 Alnylam Pharmaceuticals Inc Administração extra-hepática
IL293341B1 (en) * 2019-11-26 2026-04-01 Dtx Pharma Inc A compound comprising a nucleic acid and a half-life extension motif
CN112876534B (zh) * 2019-11-29 2024-02-09 苏州瑞博生物技术股份有限公司 肝靶向化合物及缀合物
EP4098655A4 (en) * 2020-01-29 2024-02-28 Sumitomo Chemical Company, Limited METHOD FOR PRODUCING NUCLEIC ACID OLIGOMERS
CN111269279B (zh) * 2020-02-24 2022-08-05 陕西师范大学 一种在温和条件下制备卤代糖的方法
JP2023520763A (ja) 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー ゴーシェ治療薬を発現するための非ウイルス性dnaベクター及びその使用
US20230138409A1 (en) 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
CN113683651A (zh) * 2020-05-19 2021-11-23 上海京新生物医药有限公司 一种GalNAc中间体的制备方法
MX2022014606A (es) 2020-05-22 2023-03-08 Wave Life Sciences Ltd Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas.
TWI879977B (zh) 2020-06-10 2025-04-11 大陸商正大天晴藥業集團股份有限公司 雙鏈siRNA類似物的共軛物
IL303886A (en) 2020-12-23 2023-08-01 Flagship Pioneering Inc Compositions of modified trems and uses thereof
EP4273245A4 (en) 2020-12-29 2024-12-11 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND SIRNA CONJUGATE WITH THE NUCLEIC ACID, MANUFACTURING PROCESS THEREOF AND USE THEREOF
WO2022189861A1 (en) 2021-03-08 2022-09-15 Tollys Carbohydrate conjugates of tlr3 ligands and uses thereof
US20240216535A1 (en) 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
AU2022264509A1 (en) 2021-04-27 2023-12-14 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
US20240335551A1 (en) * 2021-05-20 2024-10-10 Olix Us, Inc. Functional moieties and their uses and synthetic preparation
CN115572241B (zh) * 2021-06-21 2024-08-30 润佳(苏州)医药科技有限公司 双价化合物、偶联物及其用途
CA3225573A1 (en) 2021-07-08 2023-01-12 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
TW202308664A (zh) 2021-07-08 2023-03-01 日商日本新藥股份有限公司 腎毒性減輕劑
EP4372085A4 (en) 2021-07-16 2026-01-21 Suzhou Ribo Life Science Co Ltd Nucleic acid, composition and conjugate, container, preparation process and use
WO2023284763A1 (zh) 2021-07-16 2023-01-19 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
WO2023015223A2 (en) * 2021-08-03 2023-02-09 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
US12465647B2 (en) 2021-08-05 2025-11-11 Sanegene Bio Usa Inc. 1′-alkyl modified ribose derivatives and methods of use
EP4405368A1 (en) 2021-09-22 2024-07-31 Sanegene Bio USA Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
EP4413015A1 (en) 2021-10-05 2024-08-14 Sanegene Bio USA Inc. Polyhydroxylated cyclopentane derivatives and methods of use
WO2023097251A1 (en) * 2021-11-23 2023-06-01 Yale University Oligonucleotide-containing transcription factor targeting chimeras
US20230346819A1 (en) 2022-02-22 2023-11-02 Sanegene Bio Usa Inc. 5'-modified carbocyclic ribonucleotide derivatives and methods of use
WO2023171804A1 (ja) 2022-03-10 2023-09-14 日本新薬株式会社 抗ウイルスアンチセンスオリゴマー
CN119072330A (zh) 2022-03-14 2024-12-03 世代生物公司 异源初免加强疫苗组合物和使用方法
EP4495240A1 (en) 2022-03-16 2025-01-22 Daiichi Sankyo Company, Limited Sirna for suppressing expression of transferrin receptor-2
US20250188477A1 (en) 2022-03-16 2025-06-12 Daiichi Sankyo Company, Limited CHEMICALLY MODIFIED OLIGONUCLEOTIDE HAVING RNAi ACTIVITY
WO2023207615A1 (zh) * 2022-04-26 2023-11-02 南京明德新药研发有限公司 一类含由天然核苷酸组成突出端的双链RNAi化合物
WO2023212134A2 (en) * 2022-04-28 2023-11-02 Dicerna Pharmaceuticals, Inc. Methods for synthesis of peracetylgalactosamine-1-pentanoic acid
EP4522742A2 (en) 2022-05-13 2025-03-19 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
US20250179492A1 (en) 2022-06-22 2025-06-05 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
CN119654412A (zh) * 2022-06-27 2025-03-18 中美瑞康核酸技术(南通)研究院有限公司 激活补体因子h表达的寡核苷酸调节剂
JP2025522880A (ja) 2022-06-30 2025-07-17 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 環状ジスルフィド修飾リン酸ベースのオリゴヌクレオチドプロドラッグ
EP4555088A1 (en) 2022-07-11 2025-05-21 Sanegene Bio USA Inc. Optimized 2'- modified ribose derivatives and methods of use
KR20250046303A (ko) 2022-08-05 2025-04-02 사네진 바이오 유에스에이 인크. 안지오텐시노겐 (agt)을 표적화하는 이중 가닥 rna 및 그의 사용 방법
US20260055426A1 (en) 2022-08-19 2026-02-26 Generation Bio Co. CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF
KR20250088573A (ko) 2022-10-14 2025-06-17 사네진 바이오 유에스에이 인크. C3을 표적화하는 소형 간섭 rna 및 그의 용도
CR20250257A (es) 2022-12-02 2025-08-28 Shanghai Argo Biopharmaceutical Co Ltd Análogos de ácidos nucleicos bicíclicos abásicos y compuestos oligoméricos preparados a partir de estos
WO2024137590A2 (en) 2022-12-19 2024-06-27 Sanegene Bio Usa Inc. Small interfering rna targeting cfb and uses thereof
CN116178458A (zh) * 2022-12-26 2023-05-30 成都药明康德新药开发有限公司 一种糖苷化合物的合成
WO2024160204A1 (en) * 2023-01-30 2024-08-08 Primelink Biotherapeutics (Suzhou) Co., Ltd. Polymer-oligonucleotide conjugates
US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
WO2024186673A2 (en) 2023-03-03 2024-09-12 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
WO2024188174A1 (zh) 2023-03-10 2024-09-19 苏州瑞博生物技术股份有限公司 一种药物组合物及其用途
EP4709855A2 (en) 2023-05-12 2026-03-18 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
AU2024280172A1 (en) 2023-06-01 2025-12-04 Sunshine Lake Pharma Co., Ltd. Novel double-stranded sirna, conjugate thereof, and use thereof
WO2024263915A2 (en) 2023-06-21 2024-12-26 Sanegene Bio Usa Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
CN119219716A (zh) * 2023-06-30 2024-12-31 上海维申医药有限公司 新型GalNAc的靶向递送片段及其制备和应用
WO2025016441A1 (zh) * 2023-07-19 2025-01-23 上海维申医药有限公司 新型GalNAc的靶向递送片段及其制备和应用
US20250064939A1 (en) 2023-07-24 2025-02-27 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
AU2024321710A1 (en) 2023-08-04 2026-03-05 Chia Tai Tianqing Pharmaceutical Group Co., Ltd. Double-stranded ribonucleic acid targeting pd-l1
CN119464281A (zh) 2023-08-09 2025-02-18 北京福元医药股份有限公司 抑制DGAT2基因表达的siRNA、siRNA缀合物或其前药和药物组合物及用途
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
CN121729496A (zh) 2023-08-31 2026-03-24 正大天晴药业集团股份有限公司 靶向凝血因子xi的双链核糖核酸
CN117384226B (zh) * 2023-09-14 2025-12-26 苏州盛诺维生物科技有限公司 一种GalNAc类化合物的合成工艺
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
CN120209036A (zh) * 2023-12-26 2025-06-27 广东东阳光药业股份有限公司 一种新型的化合物及其用途
WO2025155911A1 (en) 2024-01-18 2025-07-24 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hurler syndrome
TW202602932A (zh) 2024-03-28 2026-01-16 美商萊西亞醫療公司 用於降解甲狀腺刺激荷爾蒙受體自體抗體之胞溶體靶向雙功能分子
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
CN118146284B (zh) * 2024-05-08 2024-07-26 北京悦康科创医药科技股份有限公司 一种GalNAc化合物、其与寡核苷酸缀合物及制备方法
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2026017140A2 (en) 2024-07-18 2026-01-22 Sanegene Bio Usa Inc. Small interfering rna targeting fxi and uses thereof
WO2026041067A2 (en) 2024-08-20 2026-02-26 Sanegene Bio Usa Inc. Small interfering rna targeting myostatin (mstn) and uses thereof
WO2026046411A1 (en) 2024-09-02 2026-03-05 Sanegene Bio Usa Inc. Small interfering rna targeting plasminogen (plg) and uses thereof
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor
CN121081660A (zh) * 2025-02-21 2025-12-09 上海辉棵生物医药有限公司 一种靶向递送系统及其核酸缀合物的制备方法与用途

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
AU637800B2 (en) 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
ES2061416T3 (es) 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
US5574142A (en) * 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
WO1996011266A2 (en) 1994-10-05 1996-04-18 Amgen Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
US5681824A (en) 1995-06-05 1997-10-28 Eisai Co., Ltd. Substituted liposaccharides useful in the treatment and prevention of endotoxemia
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US6300319B1 (en) * 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
AU2006279906B2 (en) 2005-08-10 2012-05-10 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
EP1989307B1 (en) * 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
WO2009073809A2 (en) * 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2721183C (en) * 2008-04-11 2019-07-16 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
US9198972B2 (en) * 2010-01-28 2015-12-01 Alnylam Pharmaceuticals, Inc. Monomers and oligonucleotides comprising cycloaddition adduct(s)
US8501930B2 (en) * 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
EP2594575A1 (en) * 2011-11-15 2013-05-22 Université de Strasbourg Mannosylated compounds useful for the prevention and the treatment of infectious diseases
EP3730618A1 (en) * 2011-11-18 2020-10-28 Alnylam Pharmaceuticals, Inc. Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
US8577565B2 (en) 2011-12-16 2013-11-05 GM Global Technology Operations LLC Limiting branch pressure to a solenoid valve in a fluid circuit
TW201808342A (zh) 2012-05-02 2018-03-16 喜納製藥公司 包含四galnac之新穎結合物及傳送寡核苷酸之方法
SE536942C2 (sv) * 2012-07-24 2014-11-11 Reiz Ismet Flexibel dragnyckel
AU2013299717B2 (en) * 2012-08-06 2018-06-28 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated RNA agents and process for their preparation
IL292159B1 (en) * 2012-12-05 2026-04-01 Alnylam Pharmaceuticals Inc PCSK9 iRNA compositions and methods of using them
DK2992098T3 (da) * 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
EP3564374A1 (en) * 2013-06-21 2019-11-06 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
EP3151839A4 (en) * 2014-06-06 2018-02-28 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds

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