JP2018509143A5 - - Google Patents
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- Publication number
- JP2018509143A5 JP2018509143A5 JP2017545273A JP2017545273A JP2018509143A5 JP 2018509143 A5 JP2018509143 A5 JP 2018509143A5 JP 2017545273 A JP2017545273 A JP 2017545273A JP 2017545273 A JP2017545273 A JP 2017545273A JP 2018509143 A5 JP2018509143 A5 JP 2018509143A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- formula
- alkyl
- compound
- pharmaceutically acceptable
- 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.)
- Ceased
Links
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021114792A JP2021166543A (ja) | 2015-02-27 | 2021-07-12 | 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
| JP2023115841A JP2023129494A (ja) | 2015-02-27 | 2023-07-14 | 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
| JP2024060083A JP2024074908A (ja) | 2015-02-27 | 2024-04-03 | 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562126346P | 2015-02-27 | 2015-02-27 | |
| US62/126,346 | 2015-02-27 | ||
| US201562234263P | 2015-09-29 | 2015-09-29 | |
| US62/234,263 | 2015-09-29 | ||
| US201662300635P | 2016-02-26 | 2016-02-26 | |
| US62/300,635 | 2016-02-26 | ||
| PCT/US2016/020127 WO2016138534A2 (en) | 2015-02-27 | 2016-02-29 | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021114792A Division JP2021166543A (ja) | 2015-02-27 | 2021-07-12 | 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018509143A JP2018509143A (ja) | 2018-04-05 |
| JP2018509143A5 true JP2018509143A5 (enExample) | 2019-04-11 |
Family
ID=56789314
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017545273A Ceased JP2018509143A (ja) | 2015-02-27 | 2016-02-29 | 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
| JP2021114792A Pending JP2021166543A (ja) | 2015-02-27 | 2021-07-12 | 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
| JP2023115841A Pending JP2023129494A (ja) | 2015-02-27 | 2023-07-14 | 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
| JP2024060083A Pending JP2024074908A (ja) | 2015-02-27 | 2024-04-03 | 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021114792A Pending JP2021166543A (ja) | 2015-02-27 | 2021-07-12 | 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
| JP2023115841A Pending JP2023129494A (ja) | 2015-02-27 | 2023-07-14 | 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
| JP2024060083A Pending JP2024074908A (ja) | 2015-02-27 | 2024-04-03 | 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20180216111A1 (enExample) |
| EP (1) | EP3262056A4 (enExample) |
| JP (4) | JP2018509143A (enExample) |
| AU (2) | AU2016224976A1 (enExample) |
| BR (1) | BR112017018383B1 (enExample) |
| CA (1) | CA2977528A1 (enExample) |
| HK (1) | HK1249106A1 (enExample) |
| IL (2) | IL254112B (enExample) |
| MA (1) | MA41759A (enExample) |
| MX (2) | MX2017011004A (enExample) |
| TW (3) | TW202403045A (enExample) |
| WO (1) | WO2016138534A2 (enExample) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2014317961B2 (en) | 2013-09-05 | 2020-07-30 | Murdoch University | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| KR102644053B1 (ko) | 2015-05-19 | 2024-03-06 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 펩티드 올리고뉴클레오티드 콘주게이트 |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| CN116059236A (zh) | 2015-12-14 | 2023-05-05 | 冷泉港实验室 | 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| EA201891419A1 (ru) | 2015-12-15 | 2019-01-31 | Сарепта Терапьютикс, Инк. | Пептид-олигонуклеотидные конъюгаты |
| US11060089B2 (en) | 2016-04-18 | 2021-07-13 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| NL2017295B1 (en) * | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Antisense oligomeric compound for Pompe disease |
| NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| KR20260009385A (ko) | 2017-08-25 | 2026-01-19 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| WO2019209764A2 (en) * | 2018-04-26 | 2019-10-31 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス，インク． | コレステリルエステル蓄積症の処置のための方法及び組成物 |
| TW202449155A (zh) * | 2018-07-27 | 2024-12-16 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| EP3830259A4 (en) | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| JP2022551986A (ja) | 2019-10-16 | 2022-12-14 | ザ・ブロード・インスティテュート・インコーポレイテッド | 修飾筋肉標的化組成物 |
| US12338437B2 (en) | 2020-05-11 | 2025-06-24 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
| US12668814B2 (en) | 2020-07-22 | 2026-06-30 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| WO2023283629A1 (en) * | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations thereof for treating facioscapulohumeral muscular dystrophy |
| US20240425864A1 (en) * | 2021-09-30 | 2024-12-26 | Sarepta Therapeutics, Inc. | Antisense oligonucleotides having one or more abasic units |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| EP4554961A2 (en) | 2022-07-14 | 2025-05-21 | The Broad Institute, Inc. | Aav capsids that enable cns-wide gene delivery through interactions with the transferrin receptor |
| WO2025155923A1 (en) | 2024-01-17 | 2025-07-24 | The Broad Institute, Inc. | Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor |
| WO2025217163A2 (en) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Novel aav capsids binding to human cd59 |
| WO2025217174A1 (en) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv |
| WO2026107393A2 (en) | 2024-11-14 | 2026-05-21 | The Broad Institute, Inc. | Aav capsid modifications that reduce immunogenicity |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE602005020745D1 (de) * | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| KR101981705B1 (ko) * | 2010-05-28 | 2019-05-24 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| AU2011367230B2 (en) * | 2011-05-05 | 2017-08-10 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| KR20140097398A (ko) * | 2011-11-18 | 2014-08-06 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트 |
| US9217148B2 (en) * | 2013-03-14 | 2015-12-22 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| AU2014317961B2 (en) * | 2013-09-05 | 2020-07-30 | Murdoch University | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| US9790495B2 (en) * | 2014-05-16 | 2017-10-17 | Oregon State University | Antisense antibacterial compounds and methods |
| EP4039807A1 (en) * | 2014-06-10 | 2022-08-10 | Erasmus University Rotterdam Medical Center | Antisense oligonucleotides useful in treatment of pompe disease |
-
2016
- 2016-02-29 MX MX2017011004A patent/MX2017011004A/es unknown
- 2016-02-29 AU AU2016224976A patent/AU2016224976A1/en not_active Abandoned
- 2016-02-29 WO PCT/US2016/020127 patent/WO2016138534A2/en not_active Ceased
- 2016-02-29 US US15/553,911 patent/US20180216111A1/en not_active Abandoned
- 2016-02-29 BR BR112017018383-8A patent/BR112017018383B1/pt active IP Right Grant
- 2016-02-29 CA CA2977528A patent/CA2977528A1/en active Pending
- 2016-02-29 MA MA041759A patent/MA41759A/fr unknown
- 2016-02-29 HK HK18108560.3A patent/HK1249106A1/zh unknown
- 2016-02-29 JP JP2017545273A patent/JP2018509143A/ja not_active Ceased
- 2016-02-29 EP EP16756555.5A patent/EP3262056A4/en active Pending
- 2016-03-01 TW TW112123102A patent/TW202403045A/zh unknown
- 2016-03-01 TW TW105106275A patent/TW201702378A/zh unknown
- 2016-03-01 TW TW114133800A patent/TW202617995A/zh unknown
-
2017
- 2017-08-23 IL IL254112A patent/IL254112B/en active IP Right Grant
- 2017-08-25 MX MX2024010190A patent/MX2024010190A/es unknown
-
2020
- 2020-06-10 AU AU2020203825A patent/AU2020203825B2/en active Active
-
2021
- 2021-03-02 IL IL281199A patent/IL281199B/en unknown
- 2021-07-12 JP JP2021114792A patent/JP2021166543A/ja active Pending
-
2023
- 2023-07-14 JP JP2023115841A patent/JP2023129494A/ja active Pending
-
2024
- 2024-04-03 JP JP2024060083A patent/JP2024074908A/ja active Pending
- 2024-05-10 US US18/660,789 patent/US20250171783A1/en active Pending
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