JPWO2020205605A5 - - Google Patents

Download PDF

Info

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
Authority
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
Other languages
English (en)
Japanese (ja)
Other versions
JP2022528487A5 (https=
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
Publication of JP2022528487A5 publication Critical patent/JP2022528487A5/ja
Pending legal-status Critical Current

Links

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 true JPWO2020205605A5 (https=) 2023-04-03
JP2022528487A5 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=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
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)

* 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
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

Similar Documents

Publication Publication Date Title
US10538763B2 (en) Compounds and methods for modulation of DUX4
JPWO2020205605A5 (https=)
JP2021011501A (ja) キメラ2重鎖核酸
JP2024056820A (ja) Scn9a発現を調節するためのオリゴヌクレオチド
JP2018516091A5 (https=)
CN102803284A (zh) 用于miRNA抑制剂和模拟物的化学修饰基序
JPWO2020190768A5 (https=)
CN115397436A (zh) 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
JPWO2021188626A5 (https=)
JPWO2021216556A5 (https=)
JPWO2019022196A1 (ja) 一本鎖オリゴヌクレオチド
US20210315918A1 (en) Compounds and Methods for Modulation of Transcript Processing
CN119768186A (zh) 用于向cns组织递送治疗剂的脂质缀合物
JP2024528252A (ja) 核酸医薬の徐放性能の強化方法
JPWO2021119226A5 (https=)
CN115485381A (zh) 以apoc3为靶点的反义核酸
WO2021157730A1 (ja) 核酸医薬とその使用
JPWO2021188661A5 (https=)
JP2025542580A (ja) 遺伝子サイレンシング技術としての、デオキシリボヌクレオチドが散在している短鎖二重鎖rna、およびその使用
CN115698290A (zh) 用于治疗神经系统疾病的补体组分4抑制剂以及使用它们的相关组合物、系统和方法
EP4446414A1 (en) Antisense oligonucleotide complex
WO2024255846A1 (en) Oligonucleotide formulation
KR20240110595A (ko) 안정화된 rna 제제
JPWO2022212231A5 (https=)
KR20250164214A (ko) 아탁신-2(ATXN2)의 발현을 억제하기 위한 RNAi 제제, 이의 조성물, 및 사용 방법