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
- 150000001875 compounds Chemical class 0.000 claims 19
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 18
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 18
- 150000003839 salts Chemical class 0.000 claims 10
- 229940113082 thymine Drugs 0.000 claims 9
- 229940035893 uracil Drugs 0.000 claims 9
- 230000008685 targeting Effects 0.000 claims 6
- -1 4-methoxytrityl Chemical group 0.000 claims 5
- 125000002252 acyl group Chemical group 0.000 claims 5
- 125000002221 trityl group Chemical group [H]C1=C([H])C([H])=C([H])C([H])=C1C([*])(C1=C(C(=C(C(=C1[H])[H])[H])[H])[H])C1=C([H])C([H])=C([H])C([H])=C1[H] 0.000 claims 5
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims 4
- 125000003696 stearoyl group Chemical group O=C([*])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims 4
- 125000002777 acetyl group Chemical group [H]C([H])([H])C(*)=O 0.000 claims 2
- 125000000217 alkyl group Chemical group 0.000 claims 2
- 208000007345 glycogen storage disease Diseases 0.000 claims 2
- 125000005647 linker group Chemical group 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims 1
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims 1
- 125000003710 aryl alkyl group Chemical group 0.000 claims 1
- 210000004899 c-terminal region Anatomy 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3262056A4 (OSRAM) |
| JP (4) | JP2018509143A (OSRAM) |
| AU (2) | AU2016224976A1 (OSRAM) |
| BR (1) | BR112017018383B1 (OSRAM) |
| CA (1) | CA2977528A1 (OSRAM) |
| HK (1) | HK1249106A1 (OSRAM) |
| IL (2) | IL254112B (OSRAM) |
| MA (1) | MA41759A (OSRAM) |
| MX (2) | MX2017011004A (OSRAM) |
| TW (2) | TW202403045A (OSRAM) |
| WO (1) | WO2016138534A2 (OSRAM) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL300444A (en) | 2013-09-05 | 2023-04-01 | Sarepta Therapeutics Inc | Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CA2963288A1 (en) | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| EP3297649B1 (en) | 2015-05-19 | 2023-10-11 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| ES2901772T3 (es) * | 2015-12-15 | 2022-03-23 | Sarepta Therapeutics Inc | Conjugados de péptido oligonucleótido |
| 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. |
| LT3673080T (lt) | 2017-08-25 | 2023-12-27 | Stoke Therapeutics, Inc. | Priešprasminiai oligomerai, skirti būklėms ir ligoms gydyti |
| WO2019209764A2 (en) * | 2018-04-26 | 2019-10-31 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| BR112020022512A2 (pt) | 2018-05-04 | 2021-05-04 | Stoke Therapeutics, Inc. | métodos e composições para tratamento de doença de armazenamento de éster de colesteril |
| 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 |
| KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| EP4045637A4 (en) | 2019-10-16 | 2023-11-22 | The Broad Institute, Inc. | TECHNICAL COMPOSITIONS FOR MUSCLE BUILDING |
| EP4150092A4 (en) | 2020-05-11 | 2024-11-06 | Stoke Therapeutics, Inc. | OPA1 ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASES AND CONDITIONS |
| WO2023283629A1 (en) * | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations 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 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| IL311568A (en) * | 2021-09-30 | 2024-05-01 | 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 |
| TW202428311A (zh) | 2022-07-14 | 2024-07-16 | 美商博得學院股份有限公司 | 藉由與運鐵蛋白受體的相互作用實現ｃｎｓ範圍之基因傳遞的ａａｖ衣殼 |
| 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 |
| 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 |
| WO2025217163A2 (en) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Novel aav capsids binding to human cd59 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN1922313B (zh) * | 2004-02-10 | 2011-10-26 | 生物马林医药公司 | 酸性α-糖苷酶及其片段 |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| CN107353317A (zh) * | 2010-05-28 | 2017-11-17 | 萨勒普塔医疗公司 | 具有修饰的亚基间键和/或端基的寡核苷酸类似物 |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| WO2012150960A1 (en) * | 2011-05-05 | 2012-11-08 | Avi Biopharma, 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 |
| KR20230116945A (ko) * | 2013-03-14 | 2023-08-04 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| IL300444A (en) * | 2013-09-05 | 2023-04-01 | Sarepta Therapeutics Inc | Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase |
| CA2948373A1 (en) * | 2014-05-16 | 2015-11-19 | Oregon State University | Antisense antibacterial compounds and methods |
| KR102524543B1 (ko) * | 2014-06-10 | 2023-04-20 | 에라스무스 유니버시티 메디컬 센터 로테르담 | 폼페병의 치료에 유용한 안티센스 올리고뉴클레오티드 |
-
2016
- 2016-02-29 MX MX2017011004A patent/MX2017011004A/es unknown
- 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 AU AU2016224976A patent/AU2016224976A1/en not_active Abandoned
- 2016-02-29 WO PCT/US2016/020127 patent/WO2016138534A2/en not_active Ceased
- 2016-02-29 CA CA2977528A patent/CA2977528A1/en active Pending
- 2016-02-29 HK HK18108560.3A patent/HK1249106A1/zh unknown
- 2016-02-29 JP JP2017545273A patent/JP2018509143A/ja not_active Ceased
- 2016-02-29 MA MA041759A patent/MA41759A/fr unknown
- 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
-
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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