JPWO2019222479A5 - - Google Patents

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JPWO2019222479A5
JPWO2019222479A5 JP2020564156A JP2020564156A JPWO2019222479A5 JP WO2019222479 A5 JPWO2019222479 A5 JP WO2019222479A5 JP 2020564156 A JP2020564156 A JP 2020564156A JP 2020564156 A JP2020564156 A JP 2020564156A JP WO2019222479 A5 JPWO2019222479 A5 JP WO2019222479A5
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Japan
Prior art keywords
strand
alkyl
dsrna
modified
dsrna molecule
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JP2020564156A
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English (en)
Japanese (ja)
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JP7384833B2 (ja
JP2021523720A5 (https=
JP2021523720A (ja
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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
Publication of JPWO2019222479A5 publication Critical patent/JPWO2019222479A5/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

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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 (4)

Publication Number Publication Date
JP2021523720A JP2021523720A (ja) 2021-09-09
JP2021523720A5 JP2021523720A5 (https=) 2022-05-24
JPWO2019222479A5 true JPWO2019222479A5 (https=) 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 (https=)
EP (1) EP3802827A4 (https=)
JP (3) JP7384833B2 (https=)
AU (2) AU2019270152B2 (https=)
CA (1) CA3099930A1 (https=)
WO (1) WO2019222479A1 (https=)

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WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
WO2019217459A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
WO2021178607A1 (en) * 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
TW202214858A (zh) * 2020-08-04 2022-04-16 大陸商上海拓界生物醫藥科技有限公司 脫靶活性降低的修飾siRNA
WO2022174000A2 (en) 2021-02-12 2022-08-18 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
WO2022212231A2 (en) 2021-03-29 2022-10-06 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
EP4493570A2 (en) 2022-03-16 2025-01-22 Empirico Inc. Galnac compositions for improving sirna bioavailability
KR20240163743A (ko) 2022-03-28 2024-11-19 엠피리코 인크. 변형된 올리고뉴클레오티드
JP2025524135A (ja) * 2022-07-27 2025-07-25 イー-セラピューティクス・ピーエルシー 核酸化合物
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
WO2024217152A2 (zh) * 2023-04-21 2024-10-24 苏州时安生物技术有限公司 一种寡聚核酸及其制备方法和应用
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

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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)
JP2012528596A (ja) 2009-06-03 2012-11-15 ダイセルナ ファーマシューティカルズ, インコーポレイテッド ペプチドダイサー基質剤およびその特異的な遺伝子発現阻害のための方法
US10913767B2 (en) 2010-04-22 2021-02-09 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
JP7105065B2 (ja) * 2014-12-15 2022-07-22 ダイセルナ ファーマシューティカルズ, インコーポレイテッド リガンド修飾二本鎖核酸
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
KR102645243B1 (ko) * 2016-11-23 2024-03-11 알닐람 파마슈티칼스 인코포레이티드 표적외 효과가 감소된 변형 rna 작용제

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