KR102426487B1 - 5'-시클로-포스포네이트 변형된 뉴클레오티드 - Google Patents

5'-시클로-포스포네이트 변형된 뉴클레오티드 Download PDF

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KR102426487B1
KR102426487B1 KR1020187034959A KR20187034959A KR102426487B1 KR 102426487 B1 KR102426487 B1 KR 102426487B1 KR 1020187034959 A KR1020187034959 A KR 1020187034959A KR 20187034959 A KR20187034959 A KR 20187034959A KR 102426487 B1 KR102426487 B1 KR 102426487B1
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alkynyl
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optionally substituted
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KR20190015277A (ko
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첸 리
타오 페이
마이클 라울러
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애로우헤드 파마슈티컬스 인코포레이티드
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KR1020187034959A 2016-06-06 2017-06-06 5'-시클로-포스포네이트 변형된 뉴클레오티드 Active KR102426487B1 (ko)

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KR1020227025704A KR102639586B1 (ko) 2016-06-06 2017-06-06 5'-시클로-포스포네이트 변형된 뉴클레오티드

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US201662346304P 2016-06-06 2016-06-06
US62/346,304 2016-06-06
PCT/US2017/036108 WO2017214112A1 (en) 2016-06-06 2017-06-06 5'-cyclo-phosphonate modified nucleotides

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CN (2) CN109526222B (enExample)
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EA (1) EA201892285A1 (enExample)
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Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202423483A (zh) * 2016-06-06 2024-06-16 美商愛羅海德製藥公司 5’-環-磷酸修飾之核苷酸
CN110520531A (zh) 2017-04-05 2019-11-29 赛伦斯治疗有限责任公司 产品和组合物
MX2020003596A (es) 2017-10-04 2020-07-22 Avidity Biosciences Inc Composiciones de acido nucleico-polipeptido y usos de las mismos.
CA3142283A1 (en) * 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
WO2022056266A2 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
IL303800A (en) 2020-12-18 2023-08-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating factor xii
KR20230167082A (ko) 2021-04-08 2023-12-07 애로우헤드 파마슈티컬스 인코포레이티드 진행성 당화 최종 산물에 대한 수용체의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
AU2022280863A1 (en) 2021-05-28 2023-11-30 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of mucin 5ac (muc5ac), compositions thereof, and methods of use
WO2023014938A1 (en) 2021-08-05 2023-02-09 Sanegene Bio Usa Inc. 1'-alkyl modified ribose derivatives and methods of use
WO2023049258A1 (en) 2021-09-22 2023-03-30 Sanegene Bio Usa Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
CN118076621A (zh) 2021-10-05 2024-05-24 美国圣因生物股份有限公司 多羟基化的环戊烷衍生物及使用方法
AU2023224208A1 (en) 2022-02-22 2024-08-22 Sanegene Bio Usa Inc. 5'-modified carbocyclic ribonucleotide derivatives and methods of use
WO2023220561A1 (en) 2022-05-09 2023-11-16 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
AU2023295010A1 (en) 2022-06-14 2025-01-09 Rona Bioscience, Limited Cyclic phosphonate-modified nucleotide
JP2025522433A (ja) 2022-06-15 2025-07-15 アローヘッド ファーマシューティカルズ インコーポレイテッド スーパーオキシドジスムターゼ1(SOD1)の発現を阻害するためのRNAi剤、その組成物、及び使用の方法
AU2023307152A1 (en) 2022-07-11 2025-01-09 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
WO2024031101A1 (en) 2022-08-05 2024-02-08 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
CN119894915A (zh) * 2022-09-21 2025-04-25 詹森生物科技公司 新型稳定的核苷磷酸及其类似物
EP4602164A2 (en) 2022-10-14 2025-08-20 Sanegene Bio USA Inc. Small interfering rna targeting c3 and uses thereof
JP2025535539A (ja) * 2022-11-02 2025-10-24 アリゴス セラピューティクス,インコーポレーテッド 修飾された短い干渉核酸(siNA)分子及びその使用
WO2024137590A2 (en) 2022-12-19 2024-06-27 Sanegene Bio Usa Inc. Small interfering rna targeting cfb and uses thereof
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
AU2024240837A1 (en) 2023-03-21 2025-09-11 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement factor b (cfb) pharmaceutical compositions thereof, and methods of use
TW202444908A (zh) 2023-04-06 2024-11-16 大陸商上海舶望製藥有限公司 5'-膦酸酯修飾的核苷類似物及其製備的寡核苷酸
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
US20250064939A1 (en) 2023-07-24 2025-02-27 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
US12503696B2 (en) 2023-08-30 2025-12-23 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of inhibin subunit beta E (INHBE), pharmaceutical compositions thereof, and methods of use
WO2025137384A1 (en) 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Methods and reagents for improved oligonucleotide synthesis
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110196141A1 (en) 2009-09-07 2011-08-11 Council Of Scientific & Industrial Research Locked and unlocked 2'-o phosphoramidite nucleosides, process of preparation thereof and oligomers comprising the nucleosides
US20130116420A1 (en) 2010-04-28 2013-05-09 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9009980D0 (en) * 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
DE69400208T2 (de) 1993-01-25 1996-11-28 Hybridon Inc Olionukleotidalkylphosphonate und -phosphonothioate
EP0691979A1 (en) 1993-03-31 1996-01-17 Sanofi Novel 5'-substituted nucleosides and oligomers produced therefrom
WO1999014226A2 (en) 1997-09-12 1999-03-25 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
ATE408699T1 (de) 1999-03-10 2008-10-15 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
US7637807B2 (en) * 2004-04-29 2009-12-29 Cfph, L.L.C. System and method for mapping results from sporting events to game inputs
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
MX2009012568A (es) 2007-05-22 2009-12-08 Mdrna Inc Oligonucleotidos de acido ribonucleico sustituidos con hidroximetilo y complejos de acido ribonucleico.
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
NZ601737A (en) 2010-02-24 2013-06-28 Arrowhead Res Corp Compositions for targeted delivery of sirna
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
JP2014531476A (ja) 2011-08-26 2014-11-27 アローヘッド リサーチ コーポレイション インビボ核酸送達のためのポリ(ビニルエステル)ポリマー
CA2842041A1 (en) 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
JP6613143B2 (ja) 2013-02-22 2019-11-27 サーナ・セラピューティクス・インコーポレイテッド 2’ヌクレオシド間結合を含有する低分子干渉核酸(siNA)分子
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
NZ744357A (en) 2016-03-07 2025-09-26 Arrowhead Pharmaceuticals Inc Targeting ligands for therapeutic compounds
TW202423483A (zh) * 2016-06-06 2024-06-16 美商愛羅海德製藥公司 5’-環-磷酸修飾之核苷酸
KR102856522B1 (ko) 2016-09-02 2025-09-08 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
TWI826365B (zh) * 2017-01-10 2023-12-21 美商愛羅海德製藥公司 α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110196141A1 (en) 2009-09-07 2011-08-11 Council Of Scientific & Industrial Research Locked and unlocked 2'-o phosphoramidite nucleosides, process of preparation thereof and oligomers comprising the nucleosides
US20130116420A1 (en) 2010-04-28 2013-05-09 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom

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