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 (ru) 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 (ru)
EP (1) EP3946374A4 (ru)
JP (1) JP2022528487A (ru)
AU (1) AU2020252020A1 (ru)
CA (1) CA3133784A1 (ru)
WO (1) WO2020205605A2 (ru)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4017504A4 (en) * 2019-08-23 2024-01-24 Univ Massachusetts O-METHYL-RICH FULLY STABILIZED OLIGONUCLEOTIDES
WO2022204430A1 (en) * 2021-03-24 2022-09-29 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
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
US20230348907A1 (en) * 2022-01-10 2023-11-02 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
JP6570447B2 (ja) * 2012-10-15 2019-09-04 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｃ９ｏｒｆ７２発現を調節するための組成物
CN105637090B (zh) * 2013-10-11 2020-10-16 Ionis制药公司 用于调节c9orf72表达的组合物
CA2978103A1 (en) * 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
WO2017109757A1 (en) * 2015-12-23 2017-06-29 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
CA3011894A1 (en) * 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
IL307726A (en) * 2017-02-06 2023-12-01 Mpeg La Llc Multimeric oligonucleotides with reduced renal clearance
EP3630199A4 (en) * 2017-06-02 2021-11-10 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR THEIR USE
WO2019094694A1 (en) * 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
CN115151641A (zh) * 2019-12-13 2022-10-04 阿尔尼拉姆医药品有限公司 人类染色体9开放阅读框72(C9ORF72)iRNA剂组合物及其使用方法

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