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
Links
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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| CA3064590A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
| 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 |
| WO2020227395A2 (en) | 2019-05-06 | 2020-11-12 | University Of Massachusetts | Anti-c9orf72 oligonucleotides and related methods |
| CA3149835A1 (en) | 2019-08-09 | 2021-02-18 | University Of Massachusetts | Chemically modified oligonucleotides targeting snps |
| 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 |
| EP4157289A4 (en) | 2020-05-26 | 2024-06-26 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| JP2024511473A (ja) * | 2021-03-24 | 2024-03-13 | アタランタ セラピューティクス，インコーポレーテッド | パターン化化学修飾を有する二本鎖ｓｉｒｎａ |
| EP4313070A4 (en) * | 2021-03-24 | 2025-06-11 | Atalanta Therapeutics, Inc. | Microglial gene silencing using double-stranded siRNA |
| BR112023025224A2 (pt) * | 2021-06-04 | 2024-02-27 | Alnylam Pharmaceuticals Inc | Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos |
| US20230193281A1 (en) * | 2021-11-08 | 2023-06-22 | 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 |
| CN122029280A (zh) * | 2023-10-18 | 2026-05-12 | 马萨诸塞大学 | 具有乙二醇修饰的寡核苷酸 |
Family Cites Families (15)
| 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 |
| EP2550000A4 (en) * | 2010-03-24 | 2014-03-26 | Advirna Inc | RNAI COMPOUNDS OF REDUCED SIZE ADMINISTERING |
| RU2730677C2 (ru) * | 2012-10-15 | 2020-08-24 | Ионис Фармасьютикалз, Инк. | Соединение для модуляции экспрессии гена c9orf72 и его применение |
| SG10201808903UA (en) * | 2013-10-11 | 2018-11-29 | 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 |
| WO2016161388A1 (en) * | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| SI3283080T1 (sl) * | 2015-04-16 | 2020-06-30 | Ionis Pharmaceuticals, Inc. | Sestavki za moduliranje izražanja C9ORF72 |
| WO2016201301A1 (en) * | 2015-06-12 | 2016-12-15 | Alnylam Pharmaceuticals, Inc. | Complement component c5 irna compositions and methods of use thereof |
| MX2018007840A (es) * | 2015-12-23 | 2019-05-02 | Crispr Therapeutics Ag | Materiales y metodos para el tratamiento de la esclerosis lateral amiotrofica y/o la degeneracion lobar frontotemporal. |
| US10478503B2 (en) * | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| EP3576752A4 (en) * | 2017-02-06 | 2020-12-16 | Mpeg La, LLC | MULTIMERIC OLIGONUCLEOTIDES WITH REDUCED RENAL CLEARANCE |
| CN111050806A (zh) * | 2017-06-02 | 2020-04-21 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| CA3064590A1 (en) * | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
| WO2019094694A1 (en) * | 2017-11-10 | 2019-05-16 | University Of Massachusetts | Compositions and methods for the treatment of expanded repeat-associated disorders |
| TWI897902B (zh) * | 2019-12-13 | 2025-09-21 | 美商阿尼拉製藥公司 | 人類染色體9開讀框72 (C9ORF72) iRNA劑組成物及其使用方法 |
-
2020
- 2020-03-27 EP EP20782396.4A patent/EP3946374A4/en active Pending
- 2020-03-27 AU AU2020252020A patent/AU2020252020A1/en active Pending
- 2020-03-27 JP JP2021557938A patent/JP2022528487A/ja active Pending
- 2020-03-27 WO PCT/US2020/025432 patent/WO2020205605A2/en not_active Ceased
- 2020-03-27 CA CA3133784A patent/CA3133784A1/en active Pending
- 2020-03-27 US US16/833,107 patent/US20200385737A1/en active Pending
-
2023
- 2023-06-09 US US18/332,415 patent/US20240132892A1/en active Pending
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