JP2023130519A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2023130519A5 JP2023130519A5 JP2023118556A JP2023118556A JP2023130519A5 JP 2023130519 A5 JP2023130519 A5 JP 2023130519A5 JP 2023118556 A JP2023118556 A JP 2023118556A JP 2023118556 A JP2023118556 A JP 2023118556A JP 2023130519 A5 JP2023130519 A5 JP 2023130519A5
- Authority
- JP
- Japan
- Prior art keywords
- exon
- pharmaceutical composition
- polynucleic acid
- acid molecule
- composition 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.)
- Granted
Links
- 108091033319 polynucleotide Proteins 0.000 claims 15
- 102000040430 polynucleotide Human genes 0.000 claims 15
- 239000008194 pharmaceutical composition Substances 0.000 claims 14
- 108020004999 messenger RNA Proteins 0.000 claims 5
- 102100024108 Dystrophin Human genes 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 108010069091 Dystrophin Proteins 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 102000007238 Transferrin Receptors Human genes 0.000 claims 2
- 108010033576 Transferrin Receptors Proteins 0.000 claims 2
- 101150015424 dmd gene Proteins 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 208000035657 Abasia Diseases 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 210000000663 muscle cell Anatomy 0.000 claims 1
- 238000011144 upstream manufacturing Methods 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025021008A JP2025069453A (ja) | 2017-09-22 | 2025-02-12 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762561939P | 2017-09-22 | 2017-09-22 | |
| US62/561,939 | 2017-09-22 | ||
| US201862696766P | 2018-07-11 | 2018-07-11 | |
| US62/696,766 | 2018-07-11 | ||
| JP2020514269A JP2020537497A (ja) | 2017-09-22 | 2018-09-21 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
| PCT/US2018/052289 WO2019060775A1 (en) | 2017-09-22 | 2018-09-21 | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND EXON JUMP INDUCTION METHODS |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020514269A Division JP2020537497A (ja) | 2017-09-22 | 2018-09-21 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025021008A Division JP2025069453A (ja) | 2017-09-22 | 2025-02-12 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023130519A JP2023130519A (ja) | 2023-09-20 |
| JP2023130519A5 true JP2023130519A5 (https=) | 2023-12-25 |
| JP7635307B2 JP7635307B2 (ja) | 2025-02-25 |
Family
ID=65810507
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020514269A Pending JP2020537497A (ja) | 2017-09-22 | 2018-09-21 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
| JP2023118556A Active JP7635307B2 (ja) | 2017-09-22 | 2023-07-20 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
| JP2025021008A Pending JP2025069453A (ja) | 2017-09-22 | 2025-02-12 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020514269A Pending JP2020537497A (ja) | 2017-09-22 | 2018-09-21 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025021008A Pending JP2025069453A (ja) | 2017-09-22 | 2025-02-12 | エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US20200282074A1 (https=) |
| EP (1) | EP3684376A4 (https=) |
| JP (3) | JP2020537497A (https=) |
| KR (2) | KR20250065737A (https=) |
| CN (1) | CN111770757A (https=) |
| AU (2) | AU2018335880B2 (https=) |
| CA (1) | CA3075425A1 (https=) |
| IL (2) | IL326219A (https=) |
| MA (1) | MA50269A (https=) |
| MX (1) | MX2020003130A (https=) |
| SG (1) | SG11202002517RA (https=) |
| WO (1) | WO2019060775A1 (https=) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2019513371A (ja) | 2016-04-01 | 2019-05-30 | アビディティー バイオサイエンシーズ エルエルシー | 核酸ポリペプチド組成物とその使用 |
| MX2019008199A (es) | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| JP7506601B2 (ja) | 2017-12-06 | 2024-06-26 | アビディティー バイオサイエンシーズ，インク． | 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US12497615B2 (en) * | 2019-04-25 | 2025-12-16 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| EP4007633A4 (en) * | 2019-08-02 | 2024-05-08 | Research Institute at Nationwide Children's Hospital | NUCLEIC ACIDS TARGETING EXON 44 AND RECOMBINANT ADENO-ASSOCIATED VIRUS COMPRISING SAID NUCLEIC ACIDS FOR THE TREATMENT OF DYSTROPHIN-BASED MYOPATHIES |
| CA3151789A1 (en) * | 2019-09-19 | 2021-03-25 | Arnay Sciences, Llc | Compounds and methods useful for modulating gene splicing |
| JP7842395B2 (ja) * | 2019-12-19 | 2026-04-08 | 日本新薬株式会社 | エクソンスキッピングを可能にするアンチセンス核酸 |
| KR20220125801A (ko) * | 2020-01-10 | 2022-09-14 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| US11555190B2 (en) | 2020-03-19 | 2023-01-17 | Avidity Biosciences, Inc. | Compositions and methods of treating Facioscapulohumeral muscular dystrophy |
| AU2021241682B2 (en) | 2020-03-27 | 2026-02-12 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle dystrophy |
| KR20230044242A (ko) * | 2020-07-23 | 2023-04-03 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 디스트로핀병증을 치료하기 위한 그의 용도 |
| US20240100177A1 (en) * | 2020-12-04 | 2024-03-28 | Dyne Therapeutics, Inc. | Antibody-oligonucleotide complexes and uses thereof |
| MX2023012559A (es) | 2021-04-30 | 2023-12-15 | Sarepta Therapeutics Inc | Métodos de tratamiento para la distrofia muscular. |
| PE20250074A1 (es) * | 2021-05-28 | 2025-01-13 | Novo Nordisk As | Composiciones y metodos para inhibir la expresion del componente 1 de reduccion de amidoxima mitocondrial (marc1) |
| IL309912A (en) * | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and their uses for the treatment of dystrophinopathy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | 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 |
| US11672872B2 (en) | 2021-07-09 | 2023-06-13 | Dyne Therapeutics, Inc. | Anti-transferrin receptor antibody and uses thereof |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| KR20240032945A (ko) * | 2021-07-09 | 2024-03-12 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 디스트로핀병증을 치료하기 위한 그의 용도 |
| IL309936A (en) * | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of dystrophinopathy |
| US20240390508A1 (en) * | 2021-08-21 | 2024-11-28 | Takeda Pharmaceutical Company Limited | Human transferrin receptor binding peptide-drug conjugate |
| MX2024003266A (es) | 2021-09-16 | 2024-04-03 | Avidity Biosciences Inc | Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral. |
| AU2022422149A1 (en) * | 2021-12-23 | 2024-08-01 | Mirecule, Inc. | Compositions for delivery of polynucleotides |
| JP2025503098A (ja) * | 2022-01-20 | 2025-01-30 | ボールデン セラピューティクス， インコーポレイテッド | ＭｕＳＫ標的化オリゴヌクレオチド |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| JP2025509438A (ja) | 2022-03-17 | 2025-04-11 | サレプタ セラピューティクス， インコーポレイテッド | ホスホロジアミデートモルホリノオリゴマーコンジュゲート |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| US20250339553A1 (en) | 2022-04-15 | 2025-11-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
| EP4617301A1 (en) | 2022-11-07 | 2025-09-17 | Nippon Shokubai Co., Ltd. | Method for producing solubilizing polymer and method for producing water absorbent resin |
| WO2024197302A1 (en) * | 2023-03-23 | 2024-09-26 | Yale University | Compositions and methods for delivering antibody oligonucleotide conjugates for exon skipping |
| CN116381125A (zh) * | 2023-06-05 | 2023-07-04 | 迦进生物医药(上海)有限公司 | 评估与蛋白偶联的核酸稳定性的方法及试剂盒 |
| WO2025072246A1 (en) * | 2023-09-26 | 2025-04-03 | Entrada Therapeutics, Inc. | Compounds and methods for skipping exon 50 in duchenne muscular dystrophy |
| WO2025212707A2 (en) * | 2024-04-03 | 2025-10-09 | Avidity Biosciences, Inc. | Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 52 skipping |
| WO2026034445A1 (ja) * | 2024-08-05 | 2026-02-12 | 日本新薬株式会社 | ホスホロジアミデートモルフォリノオリゴ核酸（ｐｍｏ）とポリアニオンとを含む、ｐｍｏ複合体 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2008317566B2 (en) * | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| WO2009126933A2 (en) * | 2008-04-11 | 2009-10-15 | Alnylam Pharmaceuticals, Inc. | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components |
| EP2119783A1 (en) * | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| US20110130346A1 (en) * | 2008-05-30 | 2011-06-02 | Isis Innovation Limited | Peptide conjugates for delvery of biologically active compounds |
| CN112574988A (zh) * | 2008-10-24 | 2021-03-30 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| LT2499249T (lt) * | 2009-11-12 | 2018-12-27 | The University Of Western Australia | Priešprasmės molekulės ir patologijų gydymo būdai |
| DK2694111T3 (en) * | 2011-04-01 | 2016-10-10 | Wyeth Llc | Antibody pharmaceutical conjugates |
| NZ731587A (en) * | 2013-03-14 | 2021-07-30 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| CA2906812A1 (en) * | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| RU2695430C2 (ru) * | 2014-06-17 | 2019-07-23 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
| EP3297649B1 (en) * | 2015-05-19 | 2023-10-11 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| WO2017192679A1 (en) * | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd. | Methods and compositions of biologically active agents |
| MX2019008199A (es) * | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| US12071621B2 (en) * | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
-
2018
- 2018-09-21 US US16/649,572 patent/US20200282074A1/en active Pending
- 2018-09-21 SG SG11202002517RA patent/SG11202002517RA/en unknown
- 2018-09-21 AU AU2018335880A patent/AU2018335880B2/en active Active
- 2018-09-21 JP JP2020514269A patent/JP2020537497A/ja active Pending
- 2018-09-21 EP EP18858928.7A patent/EP3684376A4/en active Pending
- 2018-09-21 CA CA3075425A patent/CA3075425A1/en active Pending
- 2018-09-21 MX MX2020003130A patent/MX2020003130A/es unknown
- 2018-09-21 KR KR1020257014783A patent/KR20250065737A/ko active Pending
- 2018-09-21 CN CN201880074378.2A patent/CN111770757A/zh active Pending
- 2018-09-21 IL IL326219A patent/IL326219A/en unknown
- 2018-09-21 KR KR1020207011734A patent/KR102805405B1/ko active Active
- 2018-09-21 WO PCT/US2018/052289 patent/WO2019060775A1/en not_active Ceased
- 2018-09-21 MA MA050269A patent/MA50269A/fr unknown
-
2020
- 2020-03-19 IL IL273429A patent/IL273429B1/en unknown
-
2023
- 2023-07-20 JP JP2023118556A patent/JP7635307B2/ja active Active
-
2025
- 2025-01-17 US US19/031,269 patent/US20250170260A1/en active Pending
- 2025-02-12 JP JP2025021008A patent/JP2025069453A/ja active Pending
- 2025-07-07 AU AU2025205176A patent/AU2025205176A1/en active Pending
- 2025-08-27 US US19/311,956 patent/US20250381286A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2023130519A5 (https=) | ||
| JP2020537497A5 (https=) | ||
| JP2021507686A5 (https=) | ||
| JP2025067909A5 (https=) | ||
| CN105838714B (zh) | 反义分子和治疗疾病的方法 | |
| JP2021533200A5 (https=) | ||
| JPWO2020028864A5 (https=) | ||
| JP2021533197A5 (https=) | ||
| JPWO2022270585A5 (https=) | ||
| JPWO2022020107A5 (https=) | ||
| JP7286731B2 (ja) | アンチセンスオリゴヌクレオチド | |
| JP2026500220A (ja) | 高度ｒｎａ標的化（ａｒｎａｔａｒ） | |
| EP4669754A1 (en) | OLIGONUCLEOTI TARGETING SOD1 | |
| US20250066779A1 (en) | Advanced rna targeting (arnatar) for angiotensinogen | |
| Svahn et al. | Adding functional entities to plasmids | |
| JPWO2021158858A5 (https=) | ||
| CN113924364A (zh) | 治疗亨廷顿病的组合物和方法 | |
| JP2021513851A5 (https=) | ||
| WO2021232098A1 (en) | Method for treating cyclophilin a associated diseases | |
| TW202338088A (zh) | 活化抗肌萎縮蛋白相關蛋白表達的寡核苷酸調節劑 | |
| Kiely | Recent advances in antisense technology | |
| CN115551519A (zh) | 用于治疗神经系统疾病的补体组分c1s抑制剂以及相关的组合物、系统和使用它们的方法 | |
| JPWO2020146521A5 (https=) | ||
| CN115698290A (zh) | 用于治疗神经系统疾病的补体组分4抑制剂以及使用它们的相关组合物、系统和方法 | |
| JP2021527649A5 (https=) |