JP2022528487A5 - - Google Patents

Info

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
Authority
JP
Japan
Prior art keywords
nucleotides
dsrna
rna
oligonucleotide
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
JP2021557938A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022528487A (ja
Filing date
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 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 (2)

Publication Number Publication Date
JP2022528487A JP2022528487A (ja) 2022-06-13
JP2022528487A5 true JP2022528487A5 (enExample) 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 (enExample)
EP (1) EP3946374A4 (enExample)
JP (1) JP2022528487A (enExample)
AU (1) AU2020252020A1 (enExample)
CA (1) CA3133784A1 (enExample)
WO (1) WO2020205605A2 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 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
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
US11629347B2 (en) 2019-05-06 2023-04-18 University Of Massachusetts Anti-C9ORF72 oligonucleotides and related methods
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
WO2021041247A1 (en) * 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
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
US20240200063A1 (en) * 2021-03-24 2024-06-20 Atalanta Therapeutics, Inc. Microglial gene silencing using double-stranded sirna
WO2022204430A1 (en) * 2021-03-24 2022-09-29 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
EP4347822A2 (en) * 2021-06-04 2024-04-10 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
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
US20250145992A1 (en) * 2023-10-18 2025-05-08 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
RU2615143C2 (ru) * 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
CN104968783B (zh) * 2012-10-15 2019-12-10 Ionis制药公司 用于调节c9orf72表达的组合物
US10221414B2 (en) * 2013-10-11 2019-03-05 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
US20160319278A1 (en) * 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
RS60230B1 (sr) * 2015-04-16 2020-06-30 Ionis Pharmaceuticals Inc Kompozicije za moduliranje ekspresije c9orf72
WO2016201301A1 (en) * 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
KR20180118111A (ko) * 2015-12-23 2018-10-30 크리스퍼 테라퓨틱스 아게 근위축성 측색 경화증 및/또는 전두측두엽 퇴행의 치료 물질 및 방법
US10478503B2 (en) * 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
SG11201906728TA (en) * 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
JP2020522510A (ja) * 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. オリゴヌクレオチド組成物及びその使用方法
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
US10905707B2 (en) * 2017-11-10 2021-02-02 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
KR20220115995A (ko) * 2019-12-13 2022-08-19 알닐람 파마슈티칼스 인코포레이티드 인간 염색체 9 개방 판독 프레임 72 (C9orf72) iRNA 제제 조성물 및 이의 사용 방법

Similar Documents

Publication Publication Date Title
JP2022528487A5 (enExample)
JP2022525208A5 (enExample)
US10538763B2 (en) Compounds and methods for modulation of DUX4
CN113164509B (zh) 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
JP6638923B2 (ja) キメラ2重鎖核酸
JP2023522701A5 (enExample)
JP2021533804A5 (enExample)
JP2018516091A5 (enExample)
JP2021533762A5 (enExample)
CN115397436B (zh) 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
WO2018062510A1 (ja) オーバーハングを有する二本鎖核酸複合体
CN105682687A (zh) 异染色质形成性非编码rna
JP2023518268A5 (enExample)
CA3020487A1 (en) Treatment of idiopathic pulmonary fibrosis using rna complexes that target connective tissue growth factor
KR20220069103A (ko) 최소 플루오린 함량을 갖는 작은 간섭 rna의 화학적 변형
CN119768186A (zh) 用于向cns组织递送治疗剂的脂质缀合物
JP6882735B2 (ja) 構造強化されたmiRNA阻害剤S−TuD
JP2024528252A (ja) 核酸医薬の徐放性能の強化方法
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
JP2024535869A (ja) Wfdc2の発現を調節するアンチセンス化合物
EP4446414A1 (en) Antisense oligonucleotide complex
Riasi et al. Gene Silencing Method Based on DNA.
HK40084771B (zh) 用於抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
CN121127248A (zh) 用于抑制线粒体偕胺肟还原组分1 (MARC1)表达的RNAi剂、其药用组合物及使用方法
WO2024254589A2 (en) Products and compositions