TW202320809A - 經修飾之短干擾核酸（ｓｉＮＡ）分子及其用途 - Google Patents

經修飾之短干擾核酸（ｓｉＮＡ）分子及其用途 Download PDF

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TW202320809A
TW202320809A TW111133970A TW111133970A TW202320809A TW 202320809 A TW202320809 A TW 202320809A TW 111133970 A TW111133970 A TW 111133970A TW 111133970 A TW111133970 A TW 111133970A TW 202320809 A TW202320809 A TW 202320809A
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nucleotides
nucleotide
sina
nucleotide sequence
antisense strand
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TW111133970A
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Chinese (zh)
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里歐尼德 畢戈曼
維韋克 庫瑪爾 拉萬施
馬可斯 賀司巴
瑞珍卓拉 Ｋ 潘迪
勁 洪
樂思曼 雅特布
索爾 馬丁尼茲 蒙太羅
柯斯塔，Ｎ 提力尼 Ｓ 迪
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美商艾利格斯醫療公司
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TW111133970A 2021-09-08 2022-09-07 經修飾之短干擾核酸（ｓｉＮＡ）分子及其用途 TW202320809A (zh)

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US202163241935P 2021-09-08 2021-09-08
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US (2) US12454691B2 (https=)
EP (1) EP4399217A2 (https=)
JP (1) JP2024533371A (https=)
KR (1) KR20240099159A (https=)
CN (1) CN118201942A (https=)
AU (1) AU2022344131A1 (https=)
CA (1) CA3231695A1 (https=)
TW (1) TW202320809A (https=)
WO (1) WO2023039005A2 (https=)

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CN118271378A (zh) * 2022-12-29 2024-07-02 中国科学院深圳先进技术研究院 一种糖环修饰的核苷亚磷酰胺单体及其制备方法和应用
CN119836430A (zh) * 2023-05-12 2025-04-15 北京炫景瑞医药科技有限公司 修饰的核苷类似物及其用途与含核苷酸类似物的双链寡核苷酸及其用途
WO2025029913A2 (en) * 2023-07-31 2025-02-06 Basecure Therapeutics Llc Conjugates of n-acetyl-galactosamine (galnac) and oligonucleotides and uses thereof
CN117534717A (zh) * 2024-01-09 2024-02-09 凯莱英生命科学技术(天津)有限公司 5′-(e)-乙烯基磷酸酯的合成方法

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2008306455C1 (en) * 2007-10-03 2014-04-17 Quark Pharmaceuticals, Inc. Novel siRNA structures
WO2011094953A1 (en) 2010-02-08 2011-08-11 F. Hoffmann-La Roche Ag Compounds for the treatment and prevention of influenza
KR101869570B1 (ko) 2010-04-28 2018-06-20 아이오니스 파마수티컬즈, 인코포레이티드 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물
WO2011139699A2 (en) * 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
HK1224229A1 (zh) 2013-06-26 2017-08-18 Alios Biopharma, Inc. 取代的核苷，核苷酸及其类似物
IL291323B2 (en) 2016-07-15 2023-09-01 Am Chemicals Llc Solid non-nucleoside supports and phosphoramidite building blocks for oligonucleotide synthesis
KR102350647B1 (ko) 2016-09-02 2022-01-14 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
MX2018009853A (es) 2016-09-02 2018-11-09 Arrowhead Pharmaceuticals Inc Ligandos de direccion.
MA51583A (fr) * 2018-01-04 2020-11-11 Avidity Biosciences Inc Molécules d'acide nucléique hétéroduplex et leurs utilisations
WO2019217397A2 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for improving strand biased
PE20211783A1 (es) 2018-11-08 2021-09-08 Aligos Therapeutics Inc Polimeros oligonucleotidos que inhiben el transporte de antigenos y metodos
US11033556B2 (en) 2019-02-14 2021-06-15 Aligos Therapeutics, Inc. Bicyclic sulfonamides
CN121108187A (zh) 2019-04-03 2025-12-12 阿里戈斯治疗公司 吡咯化合物
KR102641566B1 (ko) 2019-04-17 2024-02-28 알리고스 테라퓨틱스 인코포레이티드 바이사이클릭 및 트라이사이클릭 화합물
US20240352464A1 (en) * 2023-03-07 2024-10-24 Aligos Therapeutics, Inc. Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof

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US20230159929A1 (en) 2023-05-25
AU2022344131A1 (en) 2024-03-28
WO2023039005A2 (en) 2023-03-16
KR20240099159A (ko) 2024-06-28
JP2024533371A (ja) 2024-09-12
EP4399217A2 (en) 2024-07-17
WO2023039005A3 (en) 2023-07-20
CN118201942A (zh) 2024-06-14
US12454691B2 (en) 2025-10-28
CA3231695A1 (en) 2023-03-16
US20260002157A1 (en) 2026-01-01

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