JP7476199B2 - Ｓｃｎ１ａ脳症の治療のためのｓｃｎ２ａを標的とするアンチセンスオリゴヌクレオチド - Google Patents
Ｓｃｎ１ａ脳症の治療のためのｓｃｎ２ａを標的とするアンチセンスオリゴヌクレオチド Download PDFInfo
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Classifications
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| PCT/US2019/047313 WO2020041348A1 (en) | 2018-08-20 | 2019-08-20 | Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies |
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| JP2024066672A Division JP7851349B2 (ja) | 2018-08-20 | 2024-04-17 | Ｓｃｎ１ａ脳症の治療のためのｓｃｎ２ａを標的とするアンチセンスオリゴヌクレオチド |
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| EP3359685B1 (en) | 2015-10-09 | 2026-01-28 | University Of Southampton | Modulation of gene expression for deregulated protein expression |
| EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
| SG11202001590RA (en) | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| WO2021174036A1 (en) * | 2020-02-28 | 2021-09-02 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating scn1a expression |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| CN116368227A (zh) * | 2020-07-22 | 2023-06-30 | 弗洛里神经科学与心理健康研究所 | 用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法 |
| IL300258A (en) * | 2020-08-07 | 2023-03-01 | Ionis Pharmaceuticals Inc | Compounds and methods for modulating SCN2A |
| EP4155402A1 (en) | 2021-09-22 | 2023-03-29 | Royal College of Surgeons in Ireland | Modulation of microrna-335 for the treatment of sodium channelopathies |
| EP4562020A2 (en) * | 2022-07-29 | 2025-06-04 | Stoke Therapeutics, Inc. | Compounds for treatment of conditions and diseases |
| MX2025013084A (es) * | 2023-05-03 | 2026-02-03 | Stoke Therapeutics Inc | Compuestos y métodos para tratar sujetos humanos |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
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| JP2013535958A (ja) | 2010-06-23 | 2013-09-19 | カッパーアールエヌエー，インコーポレイテッド | ナトリウムチャネル、電位依存性、αサブユニット（ＳＣＮＡ）に対する天然アンチセンス転写物の阻害によるＳＣＮＡ関連疾患の治療 |
| JP2019501892A (ja) | 2015-12-14 | 2019-01-24 | コールド スプリング ハーバー ラボラトリー | 常染色体優性精神遅滞−５とドラベ症候群の処置のためのアンチセンスオリゴマー |
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| AU2019325255A1 (en) | 2021-04-15 |
| BR112021003224A2 (pt) | 2021-07-20 |
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