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 (2) | TW201702378A (enExample) |
| WO (1) | WO2016138534A2 (enExample) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102275504B1 (ko) | 2013-09-05 | 2021-07-09 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함 |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3201339A4 (en) | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| CN114681621B (zh) | 2015-05-19 | 2024-08-06 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| ES2882500T3 (es) | 2015-12-14 | 2021-12-02 | Cold Spring Harbor Laboratory | Oligómeros antisentido para el tratamiento del síndrome de Dravet |
| JP2018538302A (ja) * | 2015-12-15 | 2018-12-27 | サレプタ セラピューティクス， インコーポレイテッド | ペプチドオリゴヌクレオチド複合体 |
| MY200252A (en) | 2016-04-18 | 2023-12-16 | 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. |
| SI3673080T1 (sl) | 2017-08-25 | 2024-03-29 | Stoke Therapeutics, Inc. | Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni |
| EP3784248A4 (en) * | 2018-04-26 | 2022-08-10 | Sarepta Therapeutics, Inc. | EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY |
| WO2019213525A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| TW202020153A (zh) * | 2018-07-27 | 2020-06-01 | 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 |
| 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 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| 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 |
| CA3153902A1 (en) | 2019-10-16 | 2021-04-22 | Pardis SABETI | Engineered muscle targeting compositions |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| US20230159949A1 (en) | 2020-07-22 | 2023-05-25 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | 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 |
| JP2024537711A (ja) * | 2021-09-30 | 2024-10-16 | サレプタ セラピューティクス， インコーポレイテッド | １つ以上の脱塩基ユニットを有するアンチセンスオリゴヌクレオチド |
| KR20250004770A (ko) | 2022-04-15 | 2025-01-08 | 다인 세라퓨틱스, 인크. | 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제 |
| AU2023307223A1 (en) | 2022-07-14 | 2025-02-20 | 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 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2553955C (en) * | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| KR102182663B1 (ko) * | 2010-05-28 | 2020-11-25 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| IL273838B (en) * | 2011-05-05 | 2022-09-01 | Sarepta Therapeutics Inc | Peptide and oligonucleotide conjugates, preparations containing them and their uses |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| JP2015500204A (ja) * | 2011-11-18 | 2015-01-05 | サレプタ セラピューティクス， インコーポレイテッド | 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット |
| EA202090946A3 (ru) * | 2013-03-14 | 2021-07-30 | Сарепта Терапьютикс, Инк. | Композиции, обеспечивающие пропускание экзонов, для лечения мышечной дистрофии |
| KR102275504B1 (ko) * | 2013-09-05 | 2021-07-09 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함 |
| CA2948373A1 (en) * | 2014-05-16 | 2015-11-19 | Oregon State University | Antisense antibacterial compounds and methods |
| WO2015190922A1 (en) * | 2014-06-10 | 2015-12-17 | Erasmus University Medical Center Rotterdam | Antisense oligonucleotides useful in treatment of pompe disease |
-
2016
- 2016-02-29 CA CA2977528A patent/CA2977528A1/en active Pending
- 2016-02-29 US US15/553,911 patent/US20180216111A1/en not_active Abandoned
- 2016-02-29 MA MA041759A patent/MA41759A/fr unknown
- 2016-02-29 AU AU2016224976A patent/AU2016224976A1/en not_active Abandoned
- 2016-02-29 EP EP16756555.5A patent/EP3262056A4/en active Pending
- 2016-02-29 HK HK18108560.3A patent/HK1249106A1/zh unknown
- 2016-02-29 WO PCT/US2016/020127 patent/WO2016138534A2/en not_active Ceased
- 2016-02-29 JP JP2017545273A patent/JP2018509143A/ja not_active Ceased
- 2016-02-29 BR BR112017018383-8A patent/BR112017018383B1/pt active IP Right Grant
- 2016-02-29 MX MX2017011004A patent/MX2017011004A/es unknown
- 2016-03-01 TW TW105106275A patent/TW201702378A/zh unknown
- 2016-03-01 TW TW112123102A patent/TW202403045A/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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