JP2019513779A5 - - Google Patents
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- JP2019513779A5 JP2019513779A5 JP2018553928A JP2018553928A JP2019513779A5 JP 2019513779 A5 JP2019513779 A5 JP 2019513779A5 JP 2018553928 A JP2018553928 A JP 2018553928A JP 2018553928 A JP2018553928 A JP 2018553928A JP 2019513779 A5 JP2019513779 A5 JP 2019513779A5
- Authority
- JP
- Japan
- Prior art keywords
- aav vector
- seq
- recombinant aav
- nucleotide sequence
- composition
- 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
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- 239000013607 AAV vector Substances 0.000 claims description 65
- 239000000203 mixture Substances 0.000 claims description 55
- 239000002773 nucleotide Substances 0.000 claims description 50
- 125000003729 nucleotide group Chemical group 0.000 claims description 50
- 210000003205 muscle Anatomy 0.000 claims description 36
- 108091047189 miR-29c stem-loop Proteins 0.000 claims description 34
- 108091054490 miR-29c-2 stem-loop Proteins 0.000 claims description 34
- 102000001039 Dystrophin Human genes 0.000 claims description 21
- 108010069091 Dystrophin Proteins 0.000 claims description 21
- 201000006938 muscular dystrophy Diseases 0.000 claims description 21
- 206010016654 Fibrosis Diseases 0.000 claims description 15
- 230000004761 fibrosis Effects 0.000 claims description 15
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 10
- 239000003814 drug Substances 0.000 claims description 8
- 238000007911 parenteral administration Methods 0.000 claims description 7
- 238000007910 systemic administration Methods 0.000 claims description 7
- 238000010253 intravenous injection Methods 0.000 claims description 6
- 239000013598 vector Substances 0.000 claims description 6
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 5
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 5
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 5
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 5
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 5
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 5
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 5
- 241000649047 Adeno-associated virus 12 Species 0.000 claims description 5
- 241000300529 Adeno-associated virus 13 Species 0.000 claims description 5
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- 230000007812 deficiency Effects 0.000 claims description 5
- 238000007918 intramuscular administration Methods 0.000 claims description 5
- 102000004169 proteins and genes Human genes 0.000 claims description 5
- 108090000623 proteins and genes Proteins 0.000 claims description 5
- 230000005856 abnormality Effects 0.000 claims description 4
- 239000003795 chemical substances by application Substances 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
- 208000035475 disorder Diseases 0.000 claims description 4
- 230000001105 regulatory effect Effects 0.000 claims description 4
- 230000003387 muscular Effects 0.000 claims description 3
- 238000010255 intramuscular injection Methods 0.000 claims description 2
- 239000007927 intramuscular injection Substances 0.000 claims description 2
- 230000001225 therapeutic effect Effects 0.000 claims description 2
- 238000000034 method Methods 0.000 description 11
- 108091007431 miR-29 Proteins 0.000 description 4
- 239000013608 rAAV vector Substances 0.000 description 4
- 108091029162 miR-29 stem-loop Proteins 0.000 description 2
- 238000002648 combination therapy Methods 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 230000004064 dysfunction Effects 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662323163P | 2016-04-15 | 2016-04-15 | |
| US62/323,163 | 2016-04-15 | ||
| US201762473253P | 2017-03-17 | 2017-03-17 | |
| US62/473,253 | 2017-03-17 | ||
| PCT/US2017/027635 WO2017181014A1 (en) | 2016-04-15 | 2017-04-14 | Adeno-associated virus vector delivery of microrna-29 and micro-dystrophin to treat muscular dystrophy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019513779A JP2019513779A (ja) | 2019-05-30 |
| JP2019513779A5 true JP2019513779A5 (enExample) | 2020-05-28 |
| JP7189771B2 JP7189771B2 (ja) | 2022-12-14 |
Family
ID=60041940
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018553951A Active JP7078546B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2018553882A Active JP7153562B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロｒｎａ－２９のアデノ随伴ウイルスベクター送達 |
| JP2018553928A Active JP7189771B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロｒｎａ－２９およびマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2021190072A Active JP7431789B2 (ja) | 2016-04-15 | 2021-11-24 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2023201579A Active JP7833440B2 (ja) | 2016-04-15 | 2023-11-29 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2025075669A Active JP7880464B2 (ja) | 2016-04-15 | 2025-04-30 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018553951A Active JP7078546B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2018553882A Active JP7153562B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロｒｎａ－２９のアデノ随伴ウイルスベクター送達 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021190072A Active JP7431789B2 (ja) | 2016-04-15 | 2021-11-24 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2023201579A Active JP7833440B2 (ja) | 2016-04-15 | 2023-11-29 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2025075669A Active JP7880464B2 (ja) | 2016-04-15 | 2025-04-30 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Country Status (26)
| Country | Link |
|---|---|
| US (7) | US11406717B2 (enExample) |
| EP (5) | EP3442561B1 (enExample) |
| JP (6) | JP7078546B2 (enExample) |
| KR (3) | KR102649707B1 (enExample) |
| CN (2) | CN118895311A (enExample) |
| AU (4) | AU2017250790B2 (enExample) |
| CA (2) | CA3020996A1 (enExample) |
| CO (1) | CO2018012084A2 (enExample) |
| DK (1) | DK3442602T3 (enExample) |
| ES (3) | ES3026989T3 (enExample) |
| FI (1) | FI3442602T3 (enExample) |
| HR (1) | HRP20250541T1 (enExample) |
| HU (1) | HUE071243T2 (enExample) |
| IL (3) | IL302200B2 (enExample) |
| LT (1) | LT3442602T (enExample) |
| MA (2) | MA44683B1 (enExample) |
| MD (1) | MD3442602T2 (enExample) |
| MX (2) | MX2018012604A (enExample) |
| MY (2) | MY209041A (enExample) |
| PL (1) | PL3442602T3 (enExample) |
| PT (1) | PT3442602T (enExample) |
| RS (1) | RS66847B1 (enExample) |
| SG (1) | SG11201808941PA (enExample) |
| SI (1) | SI3442602T1 (enExample) |
| WO (3) | WO2017181011A1 (enExample) |
| ZA (2) | ZA201806746B (enExample) |
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| CN108463211B (zh) | 2015-11-16 | 2022-09-27 | 全国儿童医院研究所 | 用于治疗肌联蛋白类肌病和其它肌联蛋白病变的材料和方法 |
| WO2017181011A1 (en) | 2016-04-15 | 2017-10-19 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy |
| US11338045B2 (en) | 2017-03-17 | 2022-05-24 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| CN110997923B (zh) * | 2017-03-17 | 2024-01-02 | 全国儿童医院研究所 | 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症 |
| WO2019078916A1 (en) * | 2017-10-18 | 2019-04-25 | Research Institute At Nationwide Children's Hospital | ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY |
| MX2020004035A (es) * | 2017-10-20 | 2020-11-09 | Res Inst Nationwide Childrens Hospital | Métodos y materiales para terapia génica con nt-3. |
| AU2019216257B2 (en) | 2018-01-31 | 2025-11-20 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2C |
| IL277270B2 (en) * | 2018-03-16 | 2025-05-01 | Res Inst Nationwide Childrens Hospital | Increasing tissue-specific gene delivery through capsid modification |
| WO2019220435A1 (en) * | 2018-05-15 | 2019-11-21 | Ramot At Tel-Aviv University Ltd. | Mir126-5p for treating motor neuron diseases |
| IL324506A (en) * | 2018-06-18 | 2026-01-01 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy |
| JP7534961B2 (ja) * | 2018-06-18 | 2024-08-15 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 |
| IL279789B2 (en) | 2018-06-29 | 2026-04-01 | Res Inst Nationwide Childrens Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A |
| EP3598978B1 (en) | 2018-07-26 | 2024-05-29 | EXOFIX S.r.l. | Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles |
| CN113613668A (zh) | 2018-10-24 | 2021-11-05 | 百时美施贵宝公司 | 小型化肌营养不良蛋白及其用途 |
| CN113646004A (zh) * | 2018-12-12 | 2021-11-12 | 坚固生物科技公司 | 用于治疗肌营养不良的组合疗法 |
| KR20210133242A (ko) | 2019-02-26 | 2021-11-05 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료 |
| EP3947427A1 (en) * | 2019-03-25 | 2022-02-09 | Genethon | Production of large-sized quasidystrophins using overlapping aav vectors |
| CA3150964A1 (en) | 2019-08-21 | 2021-02-25 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| AU2020392243A1 (en) | 2019-11-28 | 2022-06-16 | Regenxbio Inc. | Microdystrophin gene therapy constructs and uses thereof |
| WO2021142435A1 (en) * | 2020-01-10 | 2021-07-15 | Solid Biosciences Inc. | Combination therapy for treating muscular dystrophy |
| CN115279421A (zh) * | 2020-01-10 | 2022-11-01 | 坚固生物科技公司 | 用于联合疗法的病毒载体 |
| US11535868B2 (en) | 2020-04-29 | 2022-12-27 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
| EP4164668A1 (en) * | 2020-06-15 | 2023-04-19 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery for muscular dystrophies |
| CN111733185A (zh) * | 2020-06-30 | 2020-10-02 | 华南农业大学 | 通过Mdfi基因编辑来实现动物肉品质改良的方法和应用 |
| EP3936616A1 (en) * | 2020-07-10 | 2022-01-12 | Charité - Universitätsmedizin Berlin | Microrna-targeted therapy for cardiac repair |
| CA3228365A1 (en) | 2021-08-11 | 2023-02-16 | Solid Biosciences Inc. | Treatment of muscular dystrophy |
| US20250121094A1 (en) * | 2021-09-16 | 2025-04-17 | University Of Florida Research Foundation, Incorporated | Aav particles with modified inverted terminal repeats for enhanced gene expression in muscle |
| US12285497B2 (en) | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| WO2023107484A1 (en) * | 2021-12-07 | 2023-06-15 | Biogen Ma Inc. | Methods and systems for transfecting host cells |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| CN115819546B (zh) * | 2022-08-19 | 2025-12-05 | 成都金唯科生物科技有限公司 | 表达微缩抗肌营养不良蛋白基因的腺相关病毒载体及其应用 |
| WO2024044725A2 (en) | 2022-08-24 | 2024-02-29 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
| EP4634386A4 (en) * | 2022-12-16 | 2026-08-12 | Mirscience Therapeutics Servicos De Pesquisa E Desenvolvimento Ltda | Antisense oligonucleotide analogues of MIR-29 and their uses |
| WO2025188755A1 (en) * | 2024-03-04 | 2025-09-12 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy |
| CN122374875A (zh) | 2024-03-27 | 2026-07-10 | 株式会社Lg新能源 | 正极活性材料、其制备方法、包括其的正极和锂二次电池 |
| WO2026011009A1 (en) * | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
| WO2026075999A2 (en) | 2024-10-01 | 2026-04-09 | Sarepta Therapeutics, Inc. | Dystrophin and micro-dystrophin antibodies and fragments thereof |
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| CN109121395B (zh) * | 2016-04-15 | 2022-08-09 | 全国儿童医院研究所 | 腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗 |
| WO2017181011A1 (en) | 2016-04-15 | 2017-10-19 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy |
| CA2971303C (en) | 2016-06-21 | 2026-03-03 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| IL324506A (en) | 2018-06-18 | 2026-01-01 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy |
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