JP2020537497A5 - - Google Patents
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- Publication number
- JP2020537497A5 JP2020537497A5 JP2020514269A JP2020514269A JP2020537497A5 JP 2020537497 A5 JP2020537497 A5 JP 2020537497A5 JP 2020514269 A JP2020514269 A JP 2020514269A JP 2020514269 A JP2020514269 A JP 2020514269A JP 2020537497 A5 JP2020537497 A5 JP 2020537497A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleic acid
- acid conjugate
- acid molecule
- exon
- conjugate 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
- 108091033319 polynucleotide Proteins 0.000 claims 35
- 102000040430 polynucleotide Human genes 0.000 claims 35
- 108020004999 messenger RNA Proteins 0.000 claims 10
- 102000007238 Transferrin Receptors Human genes 0.000 claims 6
- 108010033576 Transferrin Receptors Proteins 0.000 claims 6
- 125000000217 alkyl group Chemical group 0.000 claims 4
- 239000012634 fragment Substances 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 101150015424 dmd gene Proteins 0.000 claims 3
- 125000004169 (C1-C6) alkyl group Chemical group 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- 108010069091 Dystrophin Proteins 0.000 claims 2
- 102100024108 Dystrophin Human genes 0.000 claims 2
- 108091027305 Heteroduplex Proteins 0.000 claims 2
- 230000002950 deficient Effects 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 1
- 102000004357 Transferases Human genes 0.000 claims 1
- 108090000992 Transferases Proteins 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000005962 receptors Human genes 0.000 claims 1
- 108020003175 receptors Proteins 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023118556A 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 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 (5)
| 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023118556A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020537497A JP2020537497A (ja) | 2020-12-24 |
| JP2020537497A5 true JP2020537497A5 (enExample) | 2021-10-21 |
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 After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20200282074A1 (enExample) |
| EP (1) | EP3684376A4 (enExample) |
| JP (3) | JP2020537497A (enExample) |
| KR (2) | KR20250065737A (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 (42)
| 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 |
| WO2018129384A1 (en) | 2017-01-06 | 2018-07-12 | Avidity Biosciences Llc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| CN118667812A (zh) | 2017-12-06 | 2024-09-20 | 艾维迪提生物科学公司 | 治疗肌萎缩和强直性肌营养不良的组合物和方法 |
| 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 |
| JP2021533200A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用 |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | 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 |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| KR20220038771A (ko) * | 2019-08-02 | 2022-03-29 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 엑손 44-표적화된 핵산 및 상기 핵산을 포함하는 디스트로핀-기반 근병증 치료용 재조합 아데노-관련 바이러스 |
| CN114929336A (zh) * | 2019-09-19 | 2022-08-19 | 阿尔奈科学有限责任公司 | 用于调节基因剪接的化合物和方法 |
| EP4079329A4 (en) * | 2019-12-19 | 2024-04-17 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid enabling exon skipping |
| US20230088865A1 (en) * | 2020-01-10 | 2023-03-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| EP4121063A4 (en) | 2020-03-19 | 2024-07-03 | Avidity Biosciences, Inc. | COMPOSITIONS AND METHODS FOR TREATING FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY |
| JP7748962B2 (ja) | 2020-03-27 | 2025-10-03 | アビディティー バイオサイエンシーズ，インク． | 筋ジストロフィーを処置するための組成物および方法 |
| JP2023535073A (ja) * | 2020-07-23 | 2023-08-15 | 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それらの使用 |
| KR20230117182A (ko) * | 2020-12-04 | 2023-08-07 | 다인 세라퓨틱스, 인크. | 항체-올리고뉴클레오티드 복합체 및 그의 용도 |
| AU2022267324A1 (en) | 2021-04-30 | 2023-12-14 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| AR125992A1 (es) * | 2021-05-28 | 2023-08-30 | Novo Nordisk As | Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1) |
| 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 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11648318B2 (en) | 2021-07-09 | 2023-05-16 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (TFR) 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 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| EP4366784A1 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| CN118355035A (zh) * | 2021-07-09 | 2024-07-16 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗肌养蛋白病的用途 |
| JPWO2023026994A1 (enExample) * | 2021-08-21 | 2023-03-02 | ||
| KR20240055874A (ko) | 2021-09-16 | 2024-04-29 | 어비디티 바이오사이언시스 인크. | 안면견갑상완 근이영양증을 치료하는 조성물 및 방법 |
| WO2023122347A2 (en) * | 2021-12-23 | 2023-06-29 | Mirecule, Inc. | Compositions for delivery of polynucleotides |
| EP4466360A1 (en) * | 2022-01-20 | 2024-11-27 | Bolden Therapeutics, Inc. | Musk-targeting oligonucleotides |
| 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 |
| KR20250004770A (ko) | 2022-04-15 | 2025-01-08 | 다인 세라퓨틱스, 인크. | 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제 |
| 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 |
| US20250325694A1 (en) * | 2024-04-03 | 2025-10-23 | Avidity Biosciences, Inc. | Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 52 skipping |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2203173E (pt) * | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| AU2009234266B2 (en) * | 2008-04-11 | 2015-08-06 | 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 |
| TR201902952T4 (tr) * | 2008-10-24 | 2019-03-21 | Sarepta Therapeutics Inc | Dmd için ekson atlama bileşimleri. |
| NZ732507A (en) * | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
| EP3159409B1 (en) * | 2014-06-17 | 2019-12-04 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy |
| NZ737757A (en) * | 2015-05-19 | 2023-07-28 | Sarepta Therapeutics Inc | Peptide oligonucleotide conjugates |
| WO2018129384A1 (en) * | 2017-01-06 | 2018-07-12 | Avidity Biosciences Llc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
| 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 KR KR1020257014783A patent/KR20250065737A/ko active Pending
- 2018-09-21 CA CA3075425A patent/CA3075425A1/en active Pending
- 2018-09-21 KR KR1020207011734A patent/KR102805405B1/ko active Active
- 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 AU AU2018335880A patent/AU2018335880B2/en active Active
- 2018-09-21 MX MX2020003130A patent/MX2020003130A/es unknown
- 2018-09-21 SG SG11202002517RA patent/SG11202002517RA/en unknown
- 2018-09-21 MA MA050269A patent/MA50269A/fr unknown
- 2018-09-21 JP JP2020514269A patent/JP2020537497A/ja active Pending
- 2018-09-21 US US16/649,572 patent/US20200282074A1/en active Pending
- 2018-09-21 WO PCT/US2018/052289 patent/WO2019060775A1/en not_active Ceased
-
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
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