JP2024511473A5 - - Google Patents

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Publication number
JP2024511473A5
JP2024511473A5 JP2023558979A JP2023558979A JP2024511473A5 JP 2024511473 A5 JP2024511473 A5 JP 2024511473A5 JP 2023558979 A JP2023558979 A JP 2023558979A JP 2023558979 A JP2023558979 A JP 2023558979A JP 2024511473 A5 JP2024511473 A5 JP 2024511473A5
Authority
JP
Japan
Prior art keywords
item
formula
sirna molecule
ribonucleoside
independently
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
JP2023558979A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024511473A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/021790 external-priority patent/WO2022204430A1/en
Publication of JP2024511473A publication Critical patent/JP2024511473A/ja
Publication of JP2024511473A5 publication Critical patent/JP2024511473A5/ja
Pending legal-status Critical Current

Links

JP2023558979A 2021-03-24 2022-03-24 パターン化化学修飾を有する二本鎖sirna Pending JP2024511473A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163165543P 2021-03-24 2021-03-24
US63/165,543 2021-03-24
PCT/US2022/021790 WO2022204430A1 (en) 2021-03-24 2022-03-24 Double-stranded sirna having patterned chemical modifications

Publications (2)

Publication Number Publication Date
JP2024511473A JP2024511473A (ja) 2024-03-13
JP2024511473A5 true JP2024511473A5 (https=) 2025-04-01

Family

ID=83397880

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023558979A Pending JP2024511473A (ja) 2021-03-24 2022-03-24 パターン化化学修飾を有する二本鎖sirna

Country Status (7)

Country Link
US (1) US20240182892A1 (https=)
EP (1) EP4313075A4 (https=)
JP (1) JP2024511473A (https=)
CN (1) CN117597133A (https=)
AU (1) AU2022242899A1 (https=)
CA (1) CA3214660A1 (https=)
WO (1) WO2022204430A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023354001A1 (en) * 2022-09-28 2025-04-17 Atalanta Therapeutics, Inc. Compositions and methods for treatment of huntington's disease
WO2024169770A1 (zh) * 2023-02-17 2024-08-22 苏州时安生物技术有限公司 一种抑制SCN9A基因表达的siRNA、其药物组合物及用途
US20250145992A1 (en) * 2023-10-18 2025-05-08 University Of Massachusetts Oligonucleotides with ethylene glycol modification
WO2025108284A1 (en) * 2023-11-20 2025-05-30 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of sodium voltage-gated channel alpha subunit 9 (scn9a)
US12553050B2 (en) 2024-03-22 2026-02-17 Takeda Pharmaceutical Company Limited Compositions and methods for inhibiting cytochrome P450 family 7 subfamily a member 1 (CYP7A1) expression

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9181551B2 (en) * 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
IL292159B1 (en) * 2012-12-05 2026-04-01 Alnylam Pharmaceuticals Inc PCSK9 iRNA compositions and methods of using them
US20160319278A1 (en) * 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
CN115927335A (zh) * 2015-04-13 2023-04-07 阿尔尼拉姆医药品有限公司 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法
PT3329002T (pt) * 2015-07-31 2021-01-12 Alnylam Pharmaceuticals Inc Composições de iarn de transtirretina (ttr) e métodos de seu uso para tratamento ou prevenção de doenças associadas à ttr
DK3607069T3 (da) * 2017-04-05 2022-11-21 Silence Therapeutics Gmbh Produkter og sammensætninger
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
CN111527206A (zh) * 2017-11-13 2020-08-11 赛伦斯治疗有限责任公司 用于抑制靶标基因表达的包含二硫代磷酸酯键的核酸
CN113811311A (zh) * 2019-03-15 2021-12-17 马萨诸塞大学 用于组织特异性apoe调节的寡核苷酸
JP2022528487A (ja) * 2019-03-29 2022-06-13 ユニバーシティ・オブ・マサチューセッツ C9orf72のオリゴヌクレオチドベースの調節
WO2021041247A1 (en) * 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides

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