JP2023130519A5 - - Google Patents
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- 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) | KR102805405B1 (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 |
| AU2018378812B2 (en) | 2017-12-06 | 2025-05-22 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle atrophy and 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 |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | 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 |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| WO2020219820A1 (en) * | 2019-04-25 | 2020-10-29 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| WO2021026075A1 (en) * | 2019-08-02 | 2021-02-11 | Research Institute At Nationwide Children's Hospital | Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies |
| CA3151789A1 (en) * | 2019-09-19 | 2021-03-25 | Arnay Sciences, Llc | Compounds and methods useful for modulating gene splicing |
| IL293997A (en) * | 2019-12-19 | 2022-08-01 | Nippon Shinyaku Co Ltd | Antistrand nucleic acids that allow exon skipping |
| KR20220125801A (ko) * | 2020-01-10 | 2022-09-14 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| WO2021188390A1 (en) | 2020-03-19 | 2021-09-23 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| SI4126066T1 (sl) | 2020-03-27 | 2026-04-30 | Avidity Biosciences, Inc. | Sestavki in postopki zdravljenja mišične distrofije |
| MX2023000984A (es) * | 2020-07-23 | 2023-05-03 | Dyne Therapeutics Inc | Complejos dirigidos al musculo y usos de los mismos para el tratamiento de distrofinopatias. |
| IL303332A (en) * | 2020-12-04 | 2023-07-01 | Dyne Therapeutics Inc | Antibody-oligonucleotide complexes and their uses |
| IL307937A (en) | 2021-04-30 | 2023-12-01 | Sarepta Therapeutics Inc | Treatment methods for muscular dystrophy |
| EP4347820A1 (en) * | 2021-05-28 | 2024-04-10 | Novo Nordisk A/S | Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (marc1) expression |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| JP2024525611A (ja) * | 2021-07-09 | 2024-07-12 | ダイン セラピューティクス，インコーポレーテッド | 筋標的化複合体およびジストロフィノパチーを処置するためのその使用 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| AU2022309238A1 (en) * | 2021-07-09 | 2024-01-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| JP2024525608A (ja) * | 2021-07-09 | 2024-07-12 | ダイン セラピューティクス，インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体および製剤 |
| 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 |
| US20240390508A1 (en) * | 2021-08-21 | 2024-11-28 | Takeda Pharmaceutical Company Limited | Human transferrin receptor binding peptide-drug conjugate |
| CN118265544A (zh) | 2021-09-16 | 2024-06-28 | 艾维迪提生物科学公司 | 治疗面肩肱型肌营养不良的组合物和方法 |
| AU2022422149A1 (en) * | 2021-12-23 | 2024-08-01 | Mirecule, Inc. | Compositions for delivery of polynucleotides |
| CA3248917A1 (en) * | 2022-01-20 | 2023-07-27 | Bolden Therapeutics, Inc. | OLIGONUCLEOTIDES TARGETING MUSK |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| CN119013401A (zh) | 2022-03-17 | 2024-11-22 | 萨勒普塔医疗公司 | 二氨基磷酸酯吗啉代寡聚物缀合物 |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| WO2024101349A1 (ja) | 2022-11-07 | 2024-05-16 | 株式会社日本触媒 | 可溶化ポリマーの製造方法および吸水性樹脂の製造方法 |
| EP4683674A1 (en) * | 2023-03-23 | 2026-01-28 | 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 |
| US20250325694A1 (en) * | 2024-04-03 | 2025-10-23 | 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 |
|---|---|---|---|---|
| ES2639852T3 (es) * | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| CA2721183C (en) * | 2008-04-11 | 2019-07-16 | 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 |
| WO2009144481A2 (en) * | 2008-05-30 | 2009-12-03 | Isis Innovation Limited | Conjugates for delivery of biologically active compounds |
| BR122020021379B1 (pt) * | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) * | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| WO2012131527A1 (en) * | 2011-04-01 | 2012-10-04 | Wyeth Llc | Antibody-drug conjugates |
| CN110218727A (zh) * | 2013-03-14 | 2019-09-10 | 萨勒普塔医疗公司 | 用于治疗肌营养不良的外显子跳跃组合物 |
| US20140329762A1 (en) * | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| MX373052B (es) * | 2014-06-17 | 2020-05-11 | Nippon Shinyaku Co Ltd | Acidos nucleicos antisentido. |
| AU2016264471B2 (en) * | 2015-05-19 | 2022-01-27 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| 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 |
| 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 MX MX2020003130A patent/MX2020003130A/es unknown
- 2018-09-21 MA MA050269A patent/MA50269A/fr unknown
- 2018-09-21 JP JP2020514269A patent/JP2020537497A/ja active Pending
- 2018-09-21 KR KR1020207011734A patent/KR102805405B1/ko active Active
- 2018-09-21 US US16/649,572 patent/US20200282074A1/en active Pending
- 2018-09-21 IL IL326219A patent/IL326219A/en unknown
- 2018-09-21 CA CA3075425A patent/CA3075425A1/en active Pending
- 2018-09-21 CN CN201880074378.2A patent/CN111770757A/zh active Pending
- 2018-09-21 EP EP18858928.7A patent/EP3684376A4/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 KR KR1020257014783A patent/KR20250065737A/ko active Pending
- 2018-09-21 WO PCT/US2018/052289 patent/WO2019060775A1/en not_active Ceased
-
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
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