JP2021523720A5 - - Google Patents

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Publication number
JP2021523720A5
JP2021523720A5 JP2020564156A JP2020564156A JP2021523720A5 JP 2021523720 A5 JP2021523720 A5 JP 2021523720A5 JP 2020564156 A JP2020564156 A JP 2020564156A JP 2020564156 A JP2020564156 A JP 2020564156A JP 2021523720 A5 JP2021523720 A5 JP 2021523720A5
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JP
Japan
Prior art keywords
thiouridine
dsrna molecule
strand
molecule according
alkyl
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2020564156A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021523720A (ja
JP7384833B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/032633 external-priority patent/WO2019222479A1/en
Publication of JP2021523720A publication Critical patent/JP2021523720A/ja
Publication of JP2021523720A5 publication Critical patent/JP2021523720A5/ja
Priority to JP2023189998A priority Critical patent/JP7684373B2/ja
Application granted granted Critical
Publication of JP7384833B2 publication Critical patent/JP7384833B2/ja
Priority to JP2025076285A priority patent/JP2025113272A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020564156A 2018-05-16 2019-05-16 オフターゲット効果が低下した修飾rna剤 Active JP7384833B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023189998A JP7684373B2 (ja) 2018-05-16 2023-11-07 オフターゲット効果が低下した修飾rna剤
JP2025076285A JP2025113272A (ja) 2018-05-16 2025-05-01 オフターゲット効果が低下した修飾rna剤

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862672405P 2018-05-16 2018-05-16
US62/672,405 2018-05-16
US201862719291P 2018-08-17 2018-08-17
US62/719,291 2018-08-17
PCT/US2019/032633 WO2019222479A1 (en) 2018-05-16 2019-05-16 Modified rna agents with reduced off-target effect

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023189998A Division JP7684373B2 (ja) 2018-05-16 2023-11-07 オフターゲット効果が低下した修飾rna剤

Publications (3)

Publication Number Publication Date
JP2021523720A JP2021523720A (ja) 2021-09-09
JP2021523720A5 true JP2021523720A5 (enExample) 2022-05-24
JP7384833B2 JP7384833B2 (ja) 2023-11-21

Family

ID=68540971

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2020564156A Active JP7384833B2 (ja) 2018-05-16 2019-05-16 オフターゲット効果が低下した修飾rna剤
JP2023189998A Active JP7684373B2 (ja) 2018-05-16 2023-11-07 オフターゲット効果が低下した修飾rna剤
JP2025076285A Pending JP2025113272A (ja) 2018-05-16 2025-05-01 オフターゲット効果が低下した修飾rna剤

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2023189998A Active JP7684373B2 (ja) 2018-05-16 2023-11-07 オフターゲット効果が低下した修飾rna剤
JP2025076285A Pending JP2025113272A (ja) 2018-05-16 2025-05-01 オフターゲット効果が低下した修飾rna剤

Country Status (6)

Country Link
US (3) US12247203B2 (enExample)
EP (1) EP3802827A4 (enExample)
JP (3) JP7384833B2 (enExample)
AU (2) AU2019270152B2 (enExample)
CA (1) CA3099930A1 (enExample)
WO (1) WO2019222479A1 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
CA3098623A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
KR20230043195A (ko) * 2020-08-04 2023-03-30 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 오프-타겟 활성이 감소된 변형된 siRNA
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
CN113073098B (zh) * 2021-02-20 2024-08-20 北京理工大学 抑制BACE1基因表达的siRNA修饰物及其应用
BR112023019981A2 (pt) 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas
WO2022206946A1 (zh) * 2021-04-02 2022-10-06 上海拓界生物医药科技有限公司 乙型肝炎病毒siRNA和siRNA缀合物
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
IL315546A (en) 2022-03-16 2024-11-01 Empirico Inc GALNAC compositions to improve siRNA bioavailability
EP4499834A2 (en) 2022-03-28 2025-02-05 Empirico Inc. Modified oligonucleotides
WO2024023256A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
JP2025532985A (ja) * 2022-09-30 2025-10-03 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 修飾二本鎖rna剤
AU2024258620A1 (en) * 2023-04-21 2025-11-06 Suzhou Siran Biotechnology Co., Ltd. Oligomeric nucleic acid, and preparation method therefore and use thereof
UY40897A (es) 2023-09-21 2025-03-31 Ionis Pharmaceuticals Inc Compuestos y métodos para inhibir lpa
US12454693B1 (en) * 2024-09-27 2025-10-28 Dexcel Pharma Technologies Ltd. Nucleic acids for inhibiting expression of transferrin receptor 2

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050032069A1 (en) 2002-11-05 2005-02-10 Muthiah Manoharan Oligomeric compounds having modified bases for binding to adenine and guanine and their use in gene modulation
US7812149B2 (en) * 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
EP2152877A2 (en) * 2007-05-02 2010-02-17 Sirna Therapeutics Inc. RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE ( PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
CN102753187A (zh) * 2009-06-03 2012-10-24 戴瑟纳制药公司 肽-dicer底物试剂及其特异性抑制基因表达的方法
WO2011133876A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
US20130190383A1 (en) * 2010-04-26 2013-07-25 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2015106128A2 (en) * 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3865576A1 (en) 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
JP6924744B2 (ja) * 2015-07-17 2021-08-25 アークトゥラス・セラピューティクス・インコーポレイテッドArcturus Therapeutics,Inc. B型肝炎ウイルスに対する組成物および薬剤ならびにその使用
WO2018098328A1 (en) * 2016-11-23 2018-05-31 Alnylam Pharmaceuticals, Inc. Modified rna agents with reduced off-target effect

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