JP2021523720A5 - - Google Patents

Info

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
Application number
JP2020564156A
Other languages
English (en)
Japanese (ja)
Other versions
JP7384833B2 (ja
JP2021523720A (ja
JPWO2019222479A5 (https=
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
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

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 (4)

Publication Number Publication Date
JP2021523720A JP2021523720A (ja) 2021-09-09
JP2021523720A5 true JP2021523720A5 (https=) 2022-05-24
JPWO2019222479A5 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=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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)
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

Similar Documents

Publication Publication Date Title
JP2021523720A5 (https=)
KR102530513B1 (ko) 가역적으로 변형된 올리고뉴클레오티드를 포함하는 조성물 및 그의 용도
KR102493872B1 (ko) 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
JPWO2019222479A5 (https=)
IL316808A (en) Modified double-stranded RNA materials and their uses
JP7798836B2 (ja) Ｂ型肝炎感染の治療用のＰＡＰＤ５及びＰＡＰＤ７ ｍＲＮＡを低減させるための核酸分子
CN113286803A (zh) S-抗原转运抑制寡核苷酸聚合物和方法
CN110475784B (zh) 用于制备立体限定硫代磷酸酯寡核苷酸的正交保护基
JP7476101B2 (ja) ホスホロジチオアートヌクレオシド間結合を含むギャップマーオリゴヌクレオチド
CN116655715A (zh) 一种GalNAc衍生物、缀合物、组合物以及它们的用途
WO2025157271A1 (zh) 一种抑制f11基因表达的核酸分子
CN121194983A (zh) 5'-膦酸酯修饰的核苷类似物及其制备的寡核苷酸
CN116854753B (zh) 一种化合物、缀合物、组合物以及它们的用途
CN110446718A (zh) Unylinker快速裂解
CN121816417A (zh) 双链寡核苷酸药剂和其用途
JP7476102B2 (ja) ホスホロジチオアートヌクレオシド間結合を含むオリゴヌクレオチド
EP2854813B1 (en) Pyrazolotriazolyl nucleoside analogues and oligonucleotides comprising them
CN121532510A (zh) Cfb抑制剂组合物及其应用
CN120917033A (zh) 化合物、缀合物、组合物以及它们的用途
JPWO2020146521A5 (https=)
JPWO2021072395A5 (https=)
TWI900022B (zh) GalNAc化合物、綴合物、組合物以及它們的用途
CN119019475B (zh) GalNAc化合物、缀合物、组合物以及它们的用途
JPWO2023098908A5 (https=)
JP2023127483A (ja) ２’位にアミノスルホニルアルコキシ基を有する新規人工核酸