JP2023130519A5 - - Google Patents
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- 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 | ||
| PCT/US2018/052289 WO2019060775A1 (en) | 2017-09-22 | 2018-09-21 | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND EXON JUMP INDUCTION METHODS |
| 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 |
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 (enExample) | 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 (enExample) |
| EP (1) | EP3684376A4 (enExample) |
| JP (3) | JP2020537497A (enExample) |
| KR (2) | KR102805405B1 (enExample) |
| CN (1) | CN111770757A (enExample) |
| AU (2) | AU2018335880B2 (enExample) |
| CA (1) | CA3075425A1 (enExample) |
| IL (1) | IL273429A (enExample) |
| MA (1) | MA50269A (enExample) |
| MX (1) | MX2020003130A (enExample) |
| SG (1) | SG11202002517RA (enExample) |
| WO (1) | WO2019060775A1 (enExample) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| 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 |
| KR20250148679A (ko) | 2017-12-06 | 2025-10-14 | 어비디티 바이오사이언시스 인크. | 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| WO2020028832A1 (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 |
| 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 |
| 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 |
| EP3959319A4 (en) * | 2019-04-25 | 2023-06-07 | Avidity Biosciences, Inc. | NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING |
| US20220282247A1 (en) * | 2019-08-02 | 2022-09-08 | 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 |
| KR20220070227A (ko) * | 2019-09-19 | 2022-05-30 | 수디르 아그라왈 | 유전자 스플라이싱을 조절하는데 이용가능한 화합물 및 방법 |
| CN115151642A (zh) * | 2019-12-19 | 2022-10-04 | 日本新药株式会社 | 能够进行外显子跳读的反义核酸 |
| CN115349013A (zh) * | 2020-01-10 | 2022-11-15 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 |
| IL296387B2 (en) | 2020-03-19 | 2024-08-01 | Avidity Biosciences Inc | Preparations and methods for the treatment of facial, back and arm muscle atrophy |
| MX2022011880A (es) | 2020-03-27 | 2022-10-20 | Avidity Biosciences Inc | Composiciones y metodos para tratar distrofia muscular. |
| WO2022020107A1 (en) * | 2020-07-23 | 2022-01-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CN116744943A (zh) * | 2020-12-04 | 2023-09-12 | 达因疗法公司 | 抗体-寡核苷酸复合物及其用途 |
| KR20240004609A (ko) | 2021-04-30 | 2024-01-11 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근이영양증에 대한 치료 방법 |
| AU2022282579A1 (en) * | 2021-05-28 | 2023-11-02 | Novo Nordisk A/S | Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (marc1) expression |
| MX2024000494A (es) * | 2021-07-09 | 2024-04-10 | Dyne Therapeutics Inc | Complejos dirigidos al tratamiento de musculos, y usos de los mismos para tratar distrofinopatías. |
| 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 |
| JP2024525613A (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 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11672872B2 (en) | 2021-07-09 | 2023-06-13 | Dyne Therapeutics, Inc. | Anti-transferrin receptor antibody and uses thereof |
| EP4366784A1 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US20240390508A1 (en) * | 2021-08-21 | 2024-11-28 | Takeda Pharmaceutical Company Limited | Human transferrin receptor binding peptide-drug conjugate |
| KR20240055874A (ko) | 2021-09-16 | 2024-04-29 | 어비디티 바이오사이언시스 인크. | 안면견갑상완 근이영양증을 치료하는 조성물 및 방법 |
| 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 |
| EP4493693A1 (en) | 2022-03-17 | 2025-01-22 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| AU2023254846A1 (en) | 2022-04-15 | 2024-10-10 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating 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 |
| 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 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101896186A (zh) * | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| US8575123B2 (en) * | 2008-04-11 | 2013-11-05 | Tekmira Pharmaceuticals Corporation | 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 |
| SMT201900159T1 (it) * | 2008-10-24 | 2019-05-10 | Sarepta Therapeutics Inc | Composizioni di salto di esone per dmd |
| PL2694111T3 (pl) * | 2011-04-01 | 2017-01-31 | Wyeth Llc | Koniugaty przeciwciało-lek |
| HK1220154A1 (zh) * | 2013-03-15 | 2017-04-28 | Sarepta Therapeutics, Inc. | 改进的用於治疗肌营养不良的组合物 |
| CN111575282B (zh) * | 2014-06-17 | 2024-05-14 | 日本新药株式会社 | 反义核酸 |
| CA2986228A1 (en) * | 2015-05-19 | 2016-11-24 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| 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 WO PCT/US2018/052289 patent/WO2019060775A1/en not_active Ceased
- 2018-09-21 KR KR1020207011734A patent/KR102805405B1/ko active Active
- 2018-09-21 EP EP18858928.7A patent/EP3684376A4/en active Pending
- 2018-09-21 KR KR1020257014783A patent/KR20250065737A/ko active Pending
- 2018-09-21 MX MX2020003130A patent/MX2020003130A/es unknown
- 2018-09-21 CA CA3075425A patent/CA3075425A1/en active Pending
- 2018-09-21 AU AU2018335880A patent/AU2018335880B2/en active Active
- 2018-09-21 MA MA050269A patent/MA50269A/fr unknown
- 2018-09-21 JP JP2020514269A patent/JP2020537497A/ja active Pending
- 2018-09-21 CN CN201880074378.2A patent/CN111770757A/zh active Pending
-
2020
- 2020-03-19 IL IL273429A patent/IL273429A/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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