CN112996913A - 寡聚核酸分子及其应用 - Google Patents
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Abstract
提供了用于治疗脊髓性肌萎缩症的小激活核酸分子及其应用。该小激活核酸分子包含正义核酸链和反义核酸链，正义核酸链和反义核酸链分别为长度为16‑35个核苷酸的寡核苷酸链，并且其中一条核苷酸链与选自靶基因SMN2启动子区的靶点存在至少75%的碱基同源性或互补性。也提供了包含小激活核酸分子和可选地药用载体的药物组合物；以及使用该小激活核酸分子或包含小激活核酸分子的药物组合物上调靶基因在细胞中表达及治疗由靶基因表达不足引起的疾病的方法。
Description
PCT国内申请，说明书已公开。
Claims (41)
- PCT国内申请，权利要求书已公开。
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN201811634268 | 2018-12-29 | ||
| CN2018116342685 | 2018-12-29 | ||
| PCT/CN2019/129025 WO2020135677A1 (zh) | 2018-12-29 | 2019-12-27 | 寡聚核酸分子及其应用 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN112996913A true CN112996913A (zh) | 2021-06-18 |
| CN112996913B CN112996913B (zh) | 2022-01-04 |
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| CN201980076095.6A Active CN112996913B (zh) | 2018-12-29 | 2019-12-27 | 寡聚核酸分子及其应用 |
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| US (1) | US20220064642A1 (zh) |
| EP (1) | EP3904517A4 (zh) |
| JP (1) | JP2022515881A (zh) |
| KR (1) | KR20210110310A (zh) |
| CN (1) | CN112996913B (zh) |
| AU (1) | AU2019414608A1 (zh) |
| BR (1) | BR112021012422A2 (zh) |
| CA (1) | CA3120534A1 (zh) |
| IL (1) | IL284333A (zh) |
| MX (1) | MX2021007932A (zh) |
| PE (1) | PE20212248A1 (zh) |
| SG (1) | SG11202106327QA (zh) |
| WO (1) | WO2020135677A1 (zh) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| AU2021318560A1 (en) * | 2020-07-31 | 2023-03-30 | Ractigen Therapeutics | Combinatory treatment of SMA with saRNA and mRNA modulators |
| CN112779252B (zh) * | 2020-12-31 | 2023-05-26 | 首都儿科研究所 | 靶向SMN2启动子区MeCP2结合的关键甲基化区域的反义寡核苷酸 |
| EP4288542A1 (en) * | 2021-02-08 | 2023-12-13 | Ractigen Therapeutics | Multi-valent oligonucleotide agent and methods of use thereof |
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| CN104583398A (zh) * | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节基因表达的组合物和方法 |
| CN104630219A (zh) * | 2013-11-15 | 2015-05-20 | 上海交通大学医学院附属瑞金医院 | 抑癌基因IRX1的saRNA分子及其组合物和其应用 |
| CN106032532A (zh) * | 2015-03-17 | 2016-10-19 | 中国医学科学院北京协和医院 | 一种小激活rna及其制备方法和应用 |
| WO2017075030A1 (en) * | 2015-10-26 | 2017-05-04 | Rana Therapeutics, Inc. | Methods and compositions for increasing smn expression |
| WO2017087486A1 (en) * | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
| WO2018017991A1 (en) * | 2016-07-21 | 2018-01-25 | The General Hospital Corporation | Extracellular mrna markers of muscular dystrophies in human urine |
| US20180305689A1 (en) * | 2015-04-22 | 2018-10-25 | Mina Therapeutics Limited | Sarna compositions and methods of use |
Family Cites Families (6)
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| US20090176724A1 (en) * | 2004-06-30 | 2009-07-09 | Daiwei Shen | Methods and Compositions for the Diagnosis, Prognosis and Treatment of Cancer |
| US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| US20080187512A1 (en) * | 2007-02-07 | 2008-08-07 | Academia Sinica | Treatment for spinal muscular atrophy |
| US10174328B2 (en) * | 2013-10-04 | 2019-01-08 | Translate Bio Ma, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| US9669109B1 (en) * | 2015-11-24 | 2017-06-06 | Universita Di Ferrara | Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy |
| EP3471779A4 (en) * | 2016-06-16 | 2020-07-08 | Ionis Pharmaceuticals, Inc. | COMBINATIONS FOR MODULATING THE SMN EXPRESSION |
-
2019
- 2019-12-27 AU AU2019414608A patent/AU2019414608A1/en not_active Abandoned
- 2019-12-27 CA CA3120534A patent/CA3120534A1/en active Pending
- 2019-12-27 BR BR112021012422-5A patent/BR112021012422A2/pt unknown
- 2019-12-27 CN CN201980076095.6A patent/CN112996913B/zh active Active
- 2019-12-27 JP JP2021538368A patent/JP2022515881A/ja active Pending
- 2019-12-27 US US17/419,569 patent/US20220064642A1/en active Pending
- 2019-12-27 SG SG11202106327QA patent/SG11202106327QA/en unknown
- 2019-12-27 WO PCT/CN2019/129025 patent/WO2020135677A1/zh active Application Filing
- 2019-12-27 PE PE2021001108A patent/PE20212248A1/es unknown
- 2019-12-27 MX MX2021007932A patent/MX2021007932A/es unknown
- 2019-12-27 KR KR1020217020377A patent/KR20210110310A/ko unknown
- 2019-12-27 EP EP19906151.6A patent/EP3904517A4/en active Pending
-
2021
- 2021-06-23 IL IL284333A patent/IL284333A/en unknown
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| CN104583398A (zh) * | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节基因表达的组合物和方法 |
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| CN106032532A (zh) * | 2015-03-17 | 2016-10-19 | 中国医学科学院北京协和医院 | 一种小激活rna及其制备方法和应用 |
| US20180305689A1 (en) * | 2015-04-22 | 2018-10-25 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| WO2017075030A1 (en) * | 2015-10-26 | 2017-05-04 | Rana Therapeutics, Inc. | Methods and compositions for increasing smn expression |
| WO2017087486A1 (en) * | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
| WO2018017991A1 (en) * | 2016-07-21 | 2018-01-25 | The General Hospital Corporation | Extracellular mrna markers of muscular dystrophies in human urine |
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| WOO CJ等: "Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy", 《PROC NATL ACAD SCI U S A》 * |
Also Published As
| Publication number | Publication date |
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| EP3904517A1 (en) | 2021-11-03 |
| MX2021007932A (es) | 2021-08-16 |
| PE20212248A1 (es) | 2021-11-24 |
| US20220064642A1 (en) | 2022-03-03 |
| IL284333A (en) | 2021-08-31 |
| JP2022515881A (ja) | 2022-02-22 |
| EP3904517A4 (en) | 2022-12-28 |
| CA3120534A1 (en) | 2020-07-02 |
| CN112996913B (zh) | 2022-01-04 |
| SG11202106327QA (en) | 2021-07-29 |
| AU2019414608A1 (en) | 2021-07-22 |
| BR112021012422A2 (pt) | 2021-10-26 |
| KR20210110310A (ko) | 2021-09-07 |
| WO2020135677A1 (zh) | 2020-07-02 |
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