JP7028865B2 - 5’-シクロ-ホスホン酸修飾ヌクレオチド - Google Patents

5’-シクロ-ホスホン酸修飾ヌクレオチド Download PDF

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JP7028865B2
JP7028865B2 JP2019516096A JP2019516096A JP7028865B2 JP 7028865 B2 JP7028865 B2 JP 7028865B2 JP 2019516096 A JP2019516096 A JP 2019516096A JP 2019516096 A JP2019516096 A JP 2019516096A JP 7028865 B2 JP7028865 B2 JP 7028865B2
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リー チェン
ペイ タオ
ローラー マイケル
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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
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JP2019516096A 2016-06-06 2017-06-06 5’-シクロ-ホスホン酸修飾ヌクレオチド Active JP7028865B2 (ja)

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JP2022022652A JP7530927B2 (ja) 2016-06-06 2022-02-17 5’-シクロ-ホスホン酸修飾ヌクレオチド
JP2024074835A JP2024097860A (ja) 2016-06-06 2024-05-02 5’-シクロ-ホスホン酸修飾ヌクレオチド

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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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JP2019517588A5 JP2019517588A5 (cg-RX-API-DMAC7.html) 2020-07-16
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JP2024074835A Pending JP2024097860A (ja) 2016-06-06 2024-05-02 5’-シクロ-ホスホン酸修飾ヌクレオチド

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EP (1) EP3464313A4 (cg-RX-API-DMAC7.html)
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CN (2) CN109526222B (cg-RX-API-DMAC7.html)
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CA (1) CA3023764A1 (cg-RX-API-DMAC7.html)
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Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109526222B (zh) * 2016-06-06 2022-04-29 箭头药业股份有限公司 5’-环膦酸酯修饰核苷酸
US11015198B2 (en) 2017-04-05 2021-05-25 Silence Therapeutics Gmbh Products and compositions
CN118267396A (zh) 2017-10-04 2024-07-02 艾维迪提生物科学公司 核酸-多肽组合物及其用途
AU2020289464B2 (en) * 2019-06-06 2025-12-04 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
KR20230064620A (ko) 2020-09-11 2023-05-10 애로우헤드 파마슈티컬스 인코포레이티드 DUX4의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
TW202242111A (zh) 2020-12-18 2022-11-01 美商Ionis製藥公司 用於調節因子xii之化合物及方法
IL307421A (en) 2021-04-08 2023-12-01 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of receptor for advanced glycation end-products, compositions thereof, and methods of use
IL308883A (en) 2021-05-28 2024-01-01 Arrowhead Pharmaceuticals Inc RNAi AGENTS FOR INHIBITING EXPRESSION OF MUCIN 5AC (MUC5AC), COMPOSITIONS THEREOF, AND METHODS OF USE
MX2024001581A (es) 2021-08-05 2024-04-18 Sanegene Bio Usa Inc Derivados de ribosa modificados con 1'-alquilo y metodos de uso.
CA3233113A1 (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
US20230227480A1 (en) 2021-10-05 2023-07-20 Sanegene Bio Usa Inc. Polyhydroxylated cyclopentane derivatives and methods of use
US20230346819A1 (en) 2022-02-22 2023-11-02 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
US20240218363A1 (en) 2022-07-11 2024-07-04 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
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
JP2025534701A (ja) 2022-10-14 2025-10-17 セーンジーン バイオ ユーエスエー インコーポレイティド C3を標的とする低分子干渉rnaおよびその使用
EP4612294A2 (en) * 2022-11-02 2025-09-10 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
JP2025542234A (ja) 2022-12-19 2025-12-25 セーンジーン バイオ ユーエスエー インコーポレイティド 低分子干渉rna標的化cfb及びその使用
WO2024175111A2 (en) 2023-02-24 2024-08-29 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
KR20250174651A (ko) * 2023-04-06 2025-12-12 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. 5'-포스포네이트 변형 뉴클레오시드 유사체 및 이로부터 제조된 올리고뉴클레오티드
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
US12442002B2 (en) 2023-12-20 2025-10-14 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of activin receptor-like kinase 7 (ALK7), compositions thereof, and methods of use
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130116420A1 (en) 2010-04-28 2013-05-09 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom

Family Cites Families (25)

* 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
AU6165494A (en) 1993-01-25 1994-08-15 Hybridon, Inc. Oligonucleotide alkylphosphonates and alkylphosphonothioates
CA2159632A1 (en) 1993-03-31 1994-10-13 Ashis Kumar Saha Novel 5'-substituted nucleosides and oligomers produced therefrom
EP2253639A1 (en) 1997-09-12 2010-11-24 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
CA2365625A1 (en) 1999-03-10 2000-09-14 Phogen Limited Delivery of substances to cells
US7637807B2 (en) * 2004-04-29 2009-12-29 Cfph, L.L.C. System and method for mapping results from sporting events to game inputs
JP5441688B2 (ja) 2006-05-11 2014-03-12 アイシス ファーマシューティカルズ, インコーポレーテッド 5’修飾二環式核酸類似体
KR101133799B1 (ko) 2006-08-18 2012-04-24 에프. 호프만-라 로슈 아게 폴리뉴클레오타이드의 생체내 전달을 위한 다가접합체
EP2125852B1 (en) 2007-02-15 2016-04-06 Ionis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
NZ580712A (en) * 2007-05-22 2011-12-22 Marina Biotech Inc Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
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
WO2011104169A1 (en) 2010-02-24 2011-09-01 F. Hoffmann-La Roche Ag Compositions for targeted delivery of sirna
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
US8993738B2 (en) 2010-04-28 2015-03-31 Isis Pharmaceuticals, Inc. Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
HK1199736A1 (en) 2011-08-26 2015-07-17 箭头研究公司 Poly(vinyl ester) polymers for in vivo nucleic acid delivery
US8933047B2 (en) 2012-04-18 2015-01-13 Arrowhead Madison Inc. Poly(acrylate) polymers for in vivo nucleic acid delivery
DK2958998T3 (en) 2013-02-22 2018-04-16 Sirna Therapeutics Inc SHORT INTERFERATING NUCLEIC ACID (SINA) MOLECULES CONTAINING A 2'-INTERNUCLEOSIDE BOND
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
US10246709B2 (en) 2016-03-07 2019-04-02 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
CN109526222B (zh) * 2016-06-06 2022-04-29 箭头药业股份有限公司 5’-环膦酸酯修饰核苷酸
CN116942841A (zh) 2016-09-02 2023-10-27 箭头药业股份有限公司 靶向配体
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130116420A1 (en) 2010-04-28 2013-05-09 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom

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