JP2022528487A5 - - Google Patents

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Publication number
JP2022528487A5
JP2022528487A5 JP2021557938A JP2021557938A JP2022528487A5 JP 2022528487 A5 JP2022528487 A5 JP 2022528487A5 JP 2021557938 A JP2021557938 A JP 2021557938A JP 2021557938 A JP2021557938 A JP 2021557938A JP 2022528487 A5 JP2022528487 A5 JP 2022528487A5
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JP
Japan
Application number
JP2021557938A
Other languages
Japanese (ja)
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JPWO2020205605A5 (https=
JP2022528487A (ja
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Publication date
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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
Publication of JP2022528487A5 publication Critical patent/JP2022528487A5/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 (3)

Publication Number Publication Date
JP2022528487A JP2022528487A (ja) 2022-06-13
JPWO2020205605A5 JPWO2020205605A5 (https=) 2023-04-03
JP2022528487A5 true JP2022528487A5 (https=) 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 (https=)
EP (1) EP3946374A4 (https=)
JP (1) JP2022528487A (https=)
AU (1) AU2020252020A1 (https=)
CA (1) CA3133784A1 (https=)
WO (1) WO2020205605A2 (https=)

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Publication number Priority date Publication date Assignee Title
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
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
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ O-メチルリッチ完全安定化オリゴヌクレオチド
CN114072501A (zh) 2019-05-06 2022-02-18 马萨诸塞大学 抗c9orf72寡核苷酸及相关方法
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
JP2022545118A (ja) * 2019-08-23 2022-10-25 ユニバーシティー オブ マサチューセッツ O-メチルリッチ完全安定化オリゴヌクレオチド
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021195510A1 (en) * 2020-03-27 2021-09-30 University Of Massachusetts Dual-acting sirna based modulation of c9orf72
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
WO2022204430A1 (en) * 2021-03-24 2022-09-29 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
EP4313070A4 (en) * 2021-03-24 2025-06-11 Atalanta Therapeutics, Inc. Microglial gene silencing using double-stranded siRNA
CN117561334A (zh) * 2021-06-04 2024-02-13 阿尔尼拉姆医药品有限公司 人染色体9开放阅读框72(C9ORF72)iRNA药剂组合物和其使用方法
EP4430191A2 (en) * 2021-11-08 2024-09-18 University Of Massachusetts Oligonucleotides for sod1 modulation
WO2023133550A2 (en) * 2022-01-10 2023-07-13 University Of Massachusetts Oligonucleotides for mecp2 modulation
EP4569116A2 (en) * 2022-08-09 2025-06-18 The University of Massachusetts Identification of allele-specific target sequences for c9orf72
WO2025085593A1 (en) * 2023-10-18 2025-04-24 University Of Massachusetts Oligonucleotides with ethylene glycol modification

Family Cites Families (15)

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US20090176725A1 (en) * 2005-08-17 2009-07-09 Sirna Therapeutics Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
US9080171B2 (en) * 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
HK1212597A1 (zh) * 2012-10-15 2016-06-17 Isis Pharmaceuticals, Inc. 用於調節c90rf72表達的組合物
EP4166667A3 (en) * 2013-10-11 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3277811B1 (en) * 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
RS60230B1 (sr) * 2015-04-16 2020-06-30 Ionis Pharmaceuticals Inc Kompozicije za moduliranje ekspresije c9orf72
EP4365291A3 (en) * 2015-06-12 2024-08-14 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
BR112018012894A2 (en) * 2015-12-23 2018-12-04 Crispr Therapeutics Ag Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration
WO2017132669A1 (en) * 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
EP3576752A4 (en) * 2017-02-06 2020-12-16 Mpeg La, LLC MULTIMERIC OLIGONUCLEOTIDES WITH REDUCED RENAL CLEARANCE
US11603532B2 (en) * 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
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
WO2019094694A1 (en) * 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
EP4073251A1 (en) * 2019-12-13 2022-10-19 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof

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