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
Authority
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
JP7384833B2 (ja
JP2021523720A (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 (15)

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US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
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
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
TW202328449A (zh) 2021-09-24 2023-07-16 美商艾拉倫製藥公司 微管相關蛋白TAU(MAPT)iRNA試劑組合物及其使用方法
US11879125B2 (en) 2022-03-16 2024-01-23 Empirico Inc. GalNAc compositions for improving siRNA bioavailability
AU2023245603A1 (en) 2022-03-28 2024-11-07 Empirico Inc. Modified oligonucleotides
AU2023313030A1 (en) * 2022-07-27 2025-01-09 E-Therapeutics Plc Nucleic acid compounds
WO2024073732A1 (en) * 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
EP4700125A2 (en) * 2023-04-21 2026-02-25 Suzhou Siran Biotechnology Co., Ltd. Oligomeric nucleic acid, and preparation method therefore and use thereof
WO2025064821A2 (en) 2023-09-21 2025-03-27 Ionis Pharmaceuticals, Inc. Compounds and methods for inhibiting 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
US7812149B2 (en) * 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
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
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
US10913767B2 (en) 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
EP2563922A1 (en) 2010-04-26 2013-03-06 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
US10119136B2 (en) 2014-01-09 2018-11-06 Alnylam Pharmaceuticals, Inc. RNAi agents modified at the 4′-C position
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
CA2996722A1 (en) 2015-07-17 2017-01-26 Arcturus Therapeutics, Inc. Compositions and agents against hepatitis b virus and uses thereof
US11504391B1 (en) 2016-11-23 2022-11-22 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect

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