JP2023503644A5 - - Google Patents
Info
- Publication number
- JP2023503644A5 JP2023503644A5 JP2022531426A JP2022531426A JP2023503644A5 JP 2023503644 A5 JP2023503644 A5 JP 2023503644A5 JP 2022531426 A JP2022531426 A JP 2022531426A JP 2022531426 A JP2022531426 A JP 2022531426A JP 2023503644 A5 JP2023503644 A5 JP 2023503644A5
- Authority
- JP
- Japan
- Prior art keywords
- unsubstituted
- pharmaceutically acceptable
- acceptable salt
- substituted
- compound according
- 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.)
- Pending
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962941549P | 2019-11-27 | 2019-11-27 | |
| US62/941,549 | 2019-11-27 | ||
| PCT/US2020/062333 WO2021108640A1 (en) | 2019-11-27 | 2020-11-25 | Compounds and methods for the treatment of duchenne muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023503644A JP2023503644A (ja) | 2023-01-31 |
| JP2023503644A5 true JP2023503644A5 (https=) | 2023-12-04 |
Family
ID=74046138
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022531426A Pending JP2023503644A (ja) | 2019-11-27 | 2020-11-25 | デュシェンヌ型筋ジストロフィーの治療のための化合物および方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230108783A1 (https=) |
| EP (1) | EP4065225A1 (https=) |
| JP (1) | JP2023503644A (https=) |
| KR (1) | KR20220147575A (https=) |
| CN (1) | CN115348883B (https=) |
| AU (1) | AU2020392220A1 (https=) |
| CA (1) | CA3166370A1 (https=) |
| WO (1) | WO2021108640A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12370264B1 (en) | 2018-08-02 | 2025-07-29 | Dyne Therapeutics, Inc. | Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject |
| 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020219820A1 (en) | 2019-04-25 | 2020-10-29 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| AU2022388810A1 (en) * | 2021-11-15 | 2024-04-11 | Novartis Ag | Branched triple lipid-modified nucleic acid compounds |
| JP2024543372A (ja) * | 2021-11-15 | 2024-11-21 | ノバルティス アーゲー | ねじれ形３種脂質修飾核酸化合物 |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| JP2025533454A (ja) * | 2022-09-21 | 2025-10-07 | サレプタ セラピューティクス， インコーポレイテッド | Ｄｍｄアンチセンスオリゴヌクレオチド介在性エクソンスキッピング効率 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4783443A (en) | 1986-03-03 | 1988-11-08 | The University Of Chicago | Amino acyl cephalosporin derivatives |
| KR20200133284A (ko) * | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| JP6928025B2 (ja) * | 2012-01-27 | 2021-09-01 | バイオマリン テクノロジーズ ベー．フェー． | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| TW202503057A (zh) * | 2014-10-10 | 2025-01-16 | 美商艾爾妮蘭製藥公司 | 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法 |
| EP3858993A1 (en) * | 2015-10-09 | 2021-08-04 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| CN109462995A (zh) * | 2016-05-04 | 2019-03-12 | 波涛生命科学有限公司 | 生物活性剂的方法和组合物 |
| US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
| WO2018193428A1 (en) * | 2017-04-20 | 2018-10-25 | Synthena Ag | Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof |
| WO2019067979A1 (en) * | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
-
2020
- 2020-11-25 CA CA3166370A patent/CA3166370A1/en active Pending
- 2020-11-25 WO PCT/US2020/062333 patent/WO2021108640A1/en not_active Ceased
- 2020-11-25 EP EP20829413.2A patent/EP4065225A1/en not_active Withdrawn
- 2020-11-25 KR KR1020227021941A patent/KR20220147575A/ko not_active Ceased
- 2020-11-25 JP JP2022531426A patent/JP2023503644A/ja active Pending
- 2020-11-25 CN CN202080093533.2A patent/CN115348883B/zh active Active
- 2020-11-25 US US17/778,380 patent/US20230108783A1/en not_active Abandoned
- 2020-11-25 AU AU2020392220A patent/AU2020392220A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2023503644A5 (https=) | ||
| CN102643267B (zh) | 4-氨基-2-(2,6-二氧哌啶-3-基)异吲哚啉-1,3-二酮化合物的制备方法 | |
| JP6853193B2 (ja) | 核酸複合体 | |
| JP2023139036A5 (https=) | ||
| AU2017338986A1 (en) | Oligonucleotide compositions and methods thereof | |
| EP3341479B1 (en) | Lna-g process | |
| CN115850358B (zh) | GalNAc簇亚磷酰胺和靶向治疗性核苷 | |
| JP7348064B2 (ja) | 分子を経膜送達するための化合物及び方法 | |
| WO2016060135A1 (ja) | モルフォリノオリゴヌクレオチドの製造方法 | |
| EP3601309A1 (en) | Orthogonal protecting groups for the preparation of stereodefined phosphorothioate oligonucleotides | |
| US20220010348A1 (en) | Method for producing oligonucleotide having phosphorothioated site | |
| US12264174B2 (en) | Process for preparing imetelstat | |
| JP2023503722A5 (https=) | ||
| JP2016130232A (ja) | オリゴヌクレオチド | |
| TWI848981B (zh) | 新穎亞磷醯胺、及髮夾型單股ｒｎａ分子之製造方法 | |
| TW202440920A (zh) | 雙環脫鹼基核酸類似物以及由它們製備的寡聚化合物 | |
| JP5284084B2 (ja) | ねじれたインターカレーティング核酸（ｔｉｎａ）を用いた、フーグスティーン型の三重鎖および二重鎖の安定でそして選択的な形成、ならびにｔｉｎａの調製法 | |
| JP2016202097A (ja) | 核酸固相合成用リンカー及び担体 | |
| US6465522B1 (en) | Anthraquinone anticancer drugs | |
| EP4136083A1 (en) | Thyclotides | |
| JP7602734B2 (ja) | オリゴヌクレオチド誘導体またはその塩 | |
| US20130284944A1 (en) | Insulator and use thereof | |
| US7786296B2 (en) | Silyl linker for solid-phase synthesis of nucleic acid | |
| CN120917033A (zh) | 化合物、缀合物、组合物以及它们的用途 | |
| AU2020202816A1 (en) | Lipid containing formulations |