JPWO2020190768A5 - - Google Patents

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Publication number
JPWO2020190768A5
JPWO2020190768A5 JP2021555491A JP2021555491A JPWO2020190768A5 JP WO2020190768 A5 JPWO2020190768 A5 JP WO2020190768A5 JP 2021555491 A JP2021555491 A JP 2021555491A JP 2021555491 A JP2021555491 A JP 2021555491A JP WO2020190768 A5 JPWO2020190768 A5 JP WO2020190768A5
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JP
Japan
Prior art keywords
dsrna
compound
oligonucleotide
independently
rna
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JP2021555491A
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English (en)
Japanese (ja)
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JP2022525208A5 (https=
JP2022525208A (ja
JP7687685B2 (ja
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Priority claimed from PCT/US2020/022748 external-priority patent/WO2020190768A1/en
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Publication of JPWO2020190768A5 publication Critical patent/JPWO2020190768A5/ja
Publication of JP2022525208A5 publication Critical patent/JP2022525208A5/ja
Priority to JP2025081846A priority Critical patent/JP2025156319A/ja
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JP2021555491A 2019-03-15 2020-03-13 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド Active JP7687685B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025081846A JP2025156319A (ja) 2019-03-15 2025-05-15 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962819189P 2019-03-15 2019-03-15
US62/819,189 2019-03-15
US201962864797P 2019-06-21 2019-06-21
US62/864,797 2019-06-21
US201962951441P 2019-12-20 2019-12-20
US62/951,441 2019-12-20
PCT/US2020/022748 WO2020190768A1 (en) 2019-03-15 2020-03-13 Oligonucleotides for tissue specific apoe modulation

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025081846A Division JP2025156319A (ja) 2019-03-15 2025-05-15 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド

Publications (4)

Publication Number Publication Date
JP2022525208A JP2022525208A (ja) 2022-05-11
JPWO2020190768A5 true JPWO2020190768A5 (https=) 2023-03-17
JP2022525208A5 JP2022525208A5 (https=) 2023-03-17
JP7687685B2 JP7687685B2 (ja) 2025-06-03

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ID=72520528

Family Applications (2)

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JP2021555491A Active JP7687685B2 (ja) 2019-03-15 2020-03-13 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド
JP2025081846A Pending JP2025156319A (ja) 2019-03-15 2025-05-15 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド

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JP2025081846A Pending JP2025156319A (ja) 2019-03-15 2025-05-15 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド

Country Status (7)

Country Link
US (1) US20200362341A1 (https=)
EP (1) EP3937951A4 (https=)
JP (2) JP7687685B2 (https=)
CN (1) CN113811311A (https=)
AU (1) AU2020239987A1 (https=)
CA (1) CA3133241A1 (https=)
WO (1) WO2020190768A1 (https=)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
JP2022545118A (ja) * 2019-08-23 2022-10-25 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
KR20230018377A (ko) * 2020-04-27 2023-02-07 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 e (apoe) irna 제제 조성물 및 이의 사용 방법
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
CN114716518A (zh) * 2021-01-06 2022-07-08 圣诺制药公司 一种能够抑制pcsk9表达的分子构造及药物组合物
WO2022187617A1 (en) * 2021-03-05 2022-09-09 Alnylam Pharmaceuticals, Inc. Dosing of sirna compounds to the cisterna magna
WO2022204430A1 (en) * 2021-03-24 2022-09-29 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
JP2024523466A (ja) * 2021-06-22 2024-06-28 ユニバーシティー オブ マサチューセッツ ＩＦＮ－γシグナル伝達経路の調節のためのオリゴヌクレオチド
WO2023086777A1 (en) * 2021-11-09 2023-05-19 Children's National Medical Center Targeting apoe enhances t-cell killing in cancer immunotherapy
US20240173420A1 (en) * 2022-09-30 2024-05-30 University Of Massachusetts Ocular delivery of oligonucleotides
WO2024233864A2 (en) * 2023-05-10 2024-11-14 Dicerna Pharmaceuticals, Inc. Galnac-conjugated rnai oligonucleotides
WO2025213111A2 (en) * 2024-04-05 2025-10-09 Cornell University Aav expression of mirna to suppress human apoe mrna
US20250353873A1 (en) * 2024-05-17 2025-11-20 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apoe expression
CN118460654B (zh) * 2024-07-10 2025-02-14 凯莱英医药集团（天津）股份有限公司 一种Givosiran的制备方法
WO2026057066A1 (zh) * 2024-09-14 2026-03-19 瑞典瑞博科尔制药公司 寡核苷酸、寡核苷酸缀合物及组合物和用途
WO2026067590A1 (zh) * 2024-09-27 2026-04-02 南京金斯瑞生物科技有限公司 修饰的双链rna分子

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20060257851A1 (en) * 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
EP1989307B1 (en) * 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
CA2643048A1 (en) 2006-02-21 2007-08-30 Oklahoma Medical Research Foundation Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor
US8178503B2 (en) * 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
WO2008021353A2 (en) 2006-08-14 2008-02-21 Guangxiang Luo Composition and method for controlling hepatitis c virus infection
JP5524189B2 (ja) * 2008-06-06 2014-06-18 クォーク ファーマシューティカルズ インコーポレーティッド 耳障害治療のための組成物および方法
CN102459596B (zh) * 2009-05-06 2016-09-07 库尔纳公司 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病
DK2470656T3 (da) 2009-08-27 2015-06-22 Idera Pharmaceuticals Inc Sammensætning til hæmning af genekspression og anvendelser heraf
KR101029699B1 (ko) 2010-09-29 2011-04-18 동국대학교 산학협력단 생약추출물을 유효성분으로 함유하는 알쯔하이머 치매 치료 및 예방용 조성물
WO2013173652A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
WO2013181618A2 (en) 2012-05-31 2013-12-05 The Trustees Of Columbia University In The City Of New York Methods to treat alzheimer's disease using apoe inhibitors
EP3842514A1 (en) 2014-09-12 2021-06-30 Whitehead Institute for Biomedical Research Cells expressing apolipoprotein e and uses thereof
WO2016064895A1 (en) * 2014-10-20 2016-04-28 The Brigham And Women's Hospital, Inc. Targeting apolipoprotein e (apoe) in neurologic disease
KR20160121450A (ko) 2015-04-10 2016-10-19 경북대학교병원 콜레스테롤 축적에 의한 질환 진단용 마커 조성물 및 이의 이용
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
KR102825946B1 (ko) * 2016-02-02 2025-06-27 올릭스 주식회사 IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
EP4081302A4 (en) * 2019-12-23 2025-01-29 University Of Massachusetts OLIGONUCLEOTIDES FOR TISSUE-SPECIFIC GENE EXPRESSION MODULATION

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