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
Priority to JP2026010434A priority Critical patent/JP2026090298A/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)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2026010434A JP2026090298A (ja) 2019-03-29 2026-01-26 C9orf72のオリゴヌクレオチドベースの調節

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2026010434A Division JP2026090298A (ja) 2019-03-29 2026-01-26 C9orf72のオリゴヌクレオチドベースの調節

Publications (2)

Publication Number Publication Date
JP2022528487A JP2022528487A (ja) 2022-06-13
JP2022528487A5 true JP2022528487A5 (enExample) 2023-04-03

Family

ID=72667058

Family Applications (2)

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
JP2026010434A Pending JP2026090298A (ja) 2019-03-29 2026-01-26 C9orf72のオリゴヌクレオチドベースの調節

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2026010434A Pending JP2026090298A (ja) 2019-03-29 2026-01-26 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 (2) JP2022528487A (enExample)
AU (1) AU2020252020A1 (enExample)
CA (1) CA3133784A1 (enExample)
WO (1) WO2020205605A2 (enExample)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
EP3966328A4 (en) 2019-05-06 2023-10-18 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
US20210115442A1 (en) * 2019-08-23 2021-04-22 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
IL293824A (en) 2019-12-13 2022-08-01 Alnylam Pharmaceuticals Inc Compositions of RNA material (c9orf72) human chromosome 9 open reading frame 72 and methods of using them
US12344839B2 (en) 2020-03-27 2025-07-01 University Of Massachusetts Dual-acting siRNA based modulation of C9orf72
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova Synthetic oligonucleotides having regions of block and cluster modifications
EP4313075A4 (en) * 2021-03-24 2025-06-18 Atalanta Therapeutics, Inc. DOUBLE-STRANDED SIRNA WITH PATTERNED CHEMICAL MODIFICATIONS
JP2024512612A (ja) * 2021-03-24 2024-03-19 アタランタ セラピューティクス,インコーポレーテッド 二本鎖siRNAを使用したミクログリア遺伝子サイレンシング
MX2023014168A (es) * 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Marco de lectura abierto 72 del cromosoma humano 9 (c9orf72) composiciones de agente de arni y metodos de uso de este.
WO2023081908A2 (en) * 2021-11-08 2023-05-11 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
WO2024211480A2 (en) * 2023-04-05 2024-10-10 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
KR20260112707A (ko) * 2023-10-18 2026-07-20 유니버시티 오브 매사추세츠 에틸렌 글라이콜이 개질된 올리고뉴클레오타이드

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
WO2011119852A1 (en) * 2010-03-24 2011-09-29 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
US10443052B2 (en) * 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
MY192689A (en) * 2013-10-11 2022-09-01 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
EP3929293A3 (en) * 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
WO2016168592A2 (en) * 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
EP3307316A1 (en) * 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
CA3009308A1 (en) * 2015-12-23 2017-06-29 Chad Albert COWAN 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
CN110248665A (zh) * 2017-02-06 2019-09-17 Mpeg La有限责任公司 具有降低的肾清除的多聚体寡核苷酸
US11603532B2 (en) * 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2018237245A1 (en) * 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
WO2019094694A1 (en) * 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
IL293824A (en) * 2019-12-13 2022-08-01 Alnylam Pharmaceuticals Inc Compositions of RNA material (c9orf72) human chromosome 9 open reading frame 72 and methods of using them

Similar Documents

Publication Publication Date Title
JP2022525208A5 (enExample)
US10538763B2 (en) Compounds and methods for modulation of DUX4
JP6638923B2 (ja) キメラ2重鎖核酸
JP2023522701A5 (enExample)
JP2025066730A (ja) 分岐オリゴヌクレオチド
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
JP2019503394A5 (enExample)
WO2018062510A1 (ja) オーバーハングを有する二本鎖核酸複合体
KR20220069103A (ko) 최소 플루오린 함량을 갖는 작은 간섭 rna의 화학적 변형
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
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
JP2026082999A (ja) Wfdc2の発現を調節するアンチセンス化合物
JP2024528252A (ja) 核酸医薬の徐放性能の強化方法
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
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
EP4446414A1 (en) Antisense oligonucleotide complex
Riasi et al. Gene Silencing Method Based on DNA.
HK40084771B (en) RNAi AGENTS FOR INHIBITING EXPRESSION OF PNPLA3, PHARMACEUTICAL COMPOSITIONS THEREOF, AND METHODS OF USE
KR20250164214A (ko) 아탁신-2(ATXN2)의 발현을 억제하기 위한 RNAi 제제, 이의 조성물, 및 사용 방법