JP2023539341A5 - - Google Patents

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Publication number
JP2023539341A5
JP2023539341A5 JP2023514067A JP2023514067A JP2023539341A5 JP 2023539341 A5 JP2023539341 A5 JP 2023539341A5 JP 2023514067 A JP2023514067 A JP 2023514067A JP 2023514067 A JP2023514067 A JP 2023514067A JP 2023539341 A5 JP2023539341 A5 JP 2023539341A5
Authority
JP
Japan
Prior art keywords
double
stranded sirna
section
stranded
pharmaceutical composition
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.)
Pending
Application number
JP2023514067A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023539341A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/048611 external-priority patent/WO2022051332A1/en
Publication of JP2023539341A publication Critical patent/JP2023539341A/ja
Publication of JP2023539341A5 publication Critical patent/JP2023539341A5/ja
Pending legal-status Critical Current

Links

JP2023514067A 2020-09-01 2021-09-01 Dux4阻害剤およびその使用方法 Pending JP2023539341A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063073304P 2020-09-01 2020-09-01
US63/073,304 2020-09-01
PCT/US2021/048611 WO2022051332A1 (en) 2020-09-01 2021-09-01 Dux4 inhibitors and methods of use thereof

Publications (2)

Publication Number Publication Date
JP2023539341A JP2023539341A (ja) 2023-09-13
JP2023539341A5 true JP2023539341A5 (enExample) 2024-08-23

Family

ID=80492176

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023514067A Pending JP2023539341A (ja) 2020-09-01 2021-09-01 Dux4阻害剤およびその使用方法

Country Status (8)

Country Link
US (1) US20230348906A1 (enExample)
EP (1) EP4208548A4 (enExample)
JP (1) JP2023539341A (enExample)
AR (1) AR123420A1 (enExample)
AU (1) AU2021337595A1 (enExample)
CA (1) CA3189861A1 (enExample)
TW (1) TW202227626A (enExample)
WO (1) WO2022051332A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
KR20230023612A (ko) 2020-04-02 2023-02-17 마이레큘, 인크. 조작된 올리고뉴클레오티드를 사용한 표적화된 억제
CA3189065A1 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
WO2023185946A1 (zh) * 2022-03-30 2023-10-05 苏州瑞博生物技术股份有限公司 一种寡核苷酸缀合物、含有该寡核苷酸缀合物的组合物及制备方法和用途

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
EP3192880B1 (en) * 2010-08-18 2019-10-09 Fred Hutchinson Cancer Research Center Nucleic acid agents for use in treating facioscapulohumeral dystrophy (fshd)
EP2736539B1 (en) * 2011-07-25 2017-08-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
US10538763B2 (en) * 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
AU2018345772B2 (en) * 2017-10-02 2025-04-17 Research Institute At Nationwide Children's Hospital MiRNA detargeting system for tissue specific interference
CN120247995A (zh) * 2018-05-30 2025-07-04 诺华股份有限公司 脂质修饰的核酸化合物和方法
WO2020028134A1 (en) * 2018-07-30 2020-02-06 Fred Hutchinson Cancer Research Center Methods and compositions for treating cancer
SG11202100928QA (en) * 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

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