JP2024504377A5 - - Google Patents

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Publication number
JP2024504377A5
JP2024504377A5 JP2023544331A JP2023544331A JP2024504377A5 JP 2024504377 A5 JP2024504377 A5 JP 2024504377A5 JP 2023544331 A JP2023544331 A JP 2023544331A JP 2023544331 A JP2023544331 A JP 2023544331A JP 2024504377 A5 JP2024504377 A5 JP 2024504377A5
Authority
JP
Japan
Prior art keywords
nucleic acid
modified oligonucleotide
modified
nucleosides
seq
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
JP2023544331A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024504377A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/013323 external-priority patent/WO2022159712A1/en
Publication of JP2024504377A publication Critical patent/JP2024504377A/ja
Publication of JP2024504377A5 publication Critical patent/JP2024504377A5/ja
Pending legal-status Critical Current

Links

JP2023544331A 2021-01-22 2022-01-21 Dux4の発現を低減するための化合物及び方法 Pending JP2024504377A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163140674P 2021-01-22 2021-01-22
US63/140,674 2021-01-22
US202163217624P 2021-07-01 2021-07-01
US63/217,624 2021-07-01
US202163231559P 2021-08-10 2021-08-10
US63/231,559 2021-08-10
PCT/US2022/013323 WO2022159712A1 (en) 2021-01-22 2022-01-21 Compounds and methods for reducing dux4 expression

Publications (2)

Publication Number Publication Date
JP2024504377A JP2024504377A (ja) 2024-01-31
JP2024504377A5 true JP2024504377A5 (enExample) 2025-01-24

Family

ID=82549881

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023544331A Pending JP2024504377A (ja) 2021-01-22 2022-01-21 Dux4の発現を低減するための化合物及び方法

Country Status (4)

Country Link
US (1) US20240336915A1 (enExample)
EP (1) EP4281084A4 (enExample)
JP (1) JP2024504377A (enExample)
WO (1) WO2022159712A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF 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
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
CN117677698A (zh) * 2021-07-14 2024-03-08 米雷楚来股份有限公司 用于神经肌肉障碍的寡核苷酸及其组合物
WO2023018705A1 (en) * 2021-08-10 2023-02-16 University Of Massachusetts Compositions and methods for sirna treatment of muscular dystrophy
JP2025532128A (ja) * 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ, インコーポレーテッド Mecp2発現を低減する化合物及び方法
JP2025532127A (ja) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ, インコーポレーテッド Mecp2発現を低減する化合物及び方法
US20260159835A1 (en) * 2024-12-05 2026-06-11 Soufflé Therapeutics, Inc. Oligonucleotide-mediated knockdown of dux4

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3118316A1 (en) * 2010-09-02 2017-01-18 Université de Mons Agents useful in treating facioscapulohumeral muscular dystrophy
CA2842798C (en) * 2011-07-25 2021-02-23 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
EP3353301A1 (en) * 2015-09-21 2018-08-01 Association Institut de Myologie Antisense oligonucleotides and uses thereof
EP3684378A4 (en) * 2017-09-19 2021-06-16 Children's National Medical Center GAPMERS AND THEIR USES IN THE TREATMENT OF MUSCLE DYSTROPHY
AU2019218987B2 (en) * 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
EP3927827A4 (en) * 2019-02-22 2023-04-26 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
TW202104595A (zh) * 2019-03-29 2021-02-01 日商田邊三菱製藥股份有限公司 用來調節dux4的表現之化合物、方法及醫藥組成物

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