JPWO2020205605A5 - - Google Patents

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Publication number
JPWO2020205605A5
JPWO2020205605A5 JP2021557938A JP2021557938A JPWO2020205605A5 JP WO2020205605 A5 JPWO2020205605 A5 JP WO2020205605A5 JP 2021557938 A JP2021557938 A JP 2021557938A JP 2021557938 A JP2021557938 A JP 2021557938A JP WO2020205605 A5 JPWO2020205605 A5 JP WO2020205605A5
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JP
Japan
Prior art keywords
nucleotides
independently
dsrna
rna
complementary
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
JP2021557938A
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English (en)
Japanese (ja)
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JP2022528487A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/025432 external-priority patent/WO2020205605A2/en
Publication of JP2022528487A publication Critical patent/JP2022528487A/ja
Publication of JPWO2020205605A5 publication Critical patent/JPWO2020205605A5/ja
Pending legal-status Critical Current

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JP2021557938A 2019-03-29 2020-03-27 Ｃ９ｏｒｆ７２のオリゴヌクレオチドベースの調節 Pending JP2022528487A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962826589P 2019-03-29 2019-03-29
US62/826,589 2019-03-29
US201962864789P 2019-06-21 2019-06-21
US62/864,789 2019-06-21
US201962910842P 2019-10-04 2019-10-04
US62/910,842 2019-10-04
PCT/US2020/025432 WO2020205605A2 (en) 2019-03-29 2020-03-27 OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72

Publications (2)

Publication Number Publication Date
JP2022528487A JP2022528487A (ja) 2022-06-13
JPWO2020205605A5 true JPWO2020205605A5 (sr) 2023-04-03

Family

ID=72667058

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021557938A Pending JP2022528487A (ja) 2019-03-29 2020-03-27 Ｃ９ｏｒｆ７２のオリゴヌクレオチドベースの調節

Country Status (6)

Country Link
US (2) US20200385737A1 (sr)
EP (1) EP3946374A4 (sr)
JP (1) JP2022528487A (sr)
AU (1) AU2020252020A1 (sr)
CA (1) CA3133784A1 (sr)
WO (1) WO2020205605A2 (sr)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021041247A1 (en) * 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
JP2024511473A (ja) * 2021-03-24 2024-03-13 アタランタ セラピューティクス，インコーポレーテッド パターン化化学修飾を有する二本鎖ｓｉｒｎａ
JP2024521907A (ja) * 2021-06-04 2024-06-04 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 ヒト９番染色体オープンリーディングフレーム７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ剤組成物及びその使用方法
US20230193281A1 (en) * 2021-11-08 2023-06-22 University Of Massachusetts Oligonucleotides for sod1 modulation
WO2023133550A2 (en) * 2022-01-10 2023-07-13 University Of Massachusetts Oligonucleotides for mecp2 modulation
WO2024036186A2 (en) * 2022-08-09 2024-02-15 The University Of Massachusetts Identification of allele-specific target sequences for c9orf72

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090176725A1 (en) * 2005-08-17 2009-07-09 Sirna Therapeutics Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
RU2730677C2 (ru) * 2012-10-15 2020-08-24 Ионис Фармасьютикалз, Инк. Соединение для модуляции экспрессии гена c9orf72 и его применение
MY192689A (en) * 2013-10-11 2022-09-01 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
SG10202001856WA (en) * 2015-04-16 2020-04-29 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
EP3394260B1 (en) * 2015-12-23 2021-02-17 CRISPR Therapeutics AG Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
WO2017132669A1 (en) * 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
IL267806B2 (en) * 2017-02-06 2024-03-01 Mpeg La Llc Multimeric oligonucleotides with reduced renal clearance
CN111050806A (zh) * 2017-06-02 2020-04-21 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
WO2019094694A1 (en) * 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
TW202140509A (zh) * 2019-12-13 2021-11-01 美商阿尼拉製藥公司 人類染色體９開讀框７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ劑組成物及其使用方法

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