JP2020513811A5 - - Google Patents
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- JP2020513811A5 JP2020513811A5 JP2019550767A JP2019550767A JP2020513811A5 JP 2020513811 A5 JP2020513811 A5 JP 2020513811A5 JP 2019550767 A JP2019550767 A JP 2019550767A JP 2019550767 A JP2019550767 A JP 2019550767A JP 2020513811 A5 JP2020513811 A5 JP 2020513811A5
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- JP
- Japan
- Prior art keywords
- composition
- muscular dystrophy
- item
- recombinant
- seq
- 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.)
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- 239000000203 mixture Substances 0.000 claims description 32
- 239000002773 nucleotide Substances 0.000 claims description 27
- 125000003729 nucleotide group Chemical group 0.000 claims description 27
- 201000006938 muscular dystrophy Diseases 0.000 claims description 25
- 210000003205 muscle Anatomy 0.000 claims description 16
- 239000013598 vector Substances 0.000 claims description 9
- 239000003814 drug Substances 0.000 claims description 8
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 6
- 238000001802 infusion Methods 0.000 claims description 5
- 238000002347 injection Methods 0.000 claims description 5
- 239000007924 injection Substances 0.000 claims description 5
- 238000002054 transplantation Methods 0.000 claims description 5
- 206010016654 Fibrosis Diseases 0.000 claims description 4
- 230000004761 fibrosis Effects 0.000 claims description 4
- 238000010255 intramuscular injection Methods 0.000 claims description 4
- 238000010253 intravenous injection Methods 0.000 claims description 4
- 238000007911 parenteral administration Methods 0.000 claims description 3
- 239000003937 drug carrier Substances 0.000 claims description 2
- 239000007927 intramuscular injection Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- 239000013607 AAV vector Substances 0.000 description 33
- 238000000034 method Methods 0.000 description 14
- 239000003623 enhancer Substances 0.000 description 8
- 108090000623 proteins and genes Proteins 0.000 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 description 5
- 239000003795 chemical substances by application Substances 0.000 description 5
- 101001044938 Dictyostelium discoideum Diacylglycerol kinase A Proteins 0.000 description 4
- 229940079593 drug Drugs 0.000 description 3
- 102000004169 proteins and genes Human genes 0.000 description 3
- 230000001105 regulatory effect Effects 0.000 description 3
- 108010085238 Actins Proteins 0.000 description 2
- 108010059343 MM Form Creatine Kinase Proteins 0.000 description 2
- 102000005604 Myosin Heavy Chains Human genes 0.000 description 2
- 108010084498 Myosin Heavy Chains Proteins 0.000 description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- 108010051583 Ventricular Myosins Proteins 0.000 description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 1
- 241000649045 Adeno-associated virus 10 Species 0.000 description 1
- 241000649046 Adeno-associated virus 11 Species 0.000 description 1
- 241000649047 Adeno-associated virus 12 Species 0.000 description 1
- 241000300529 Adeno-associated virus 13 Species 0.000 description 1
- 102000004420 Creatine Kinase Human genes 0.000 description 1
- 108010042126 Creatine kinase Proteins 0.000 description 1
- 102100024108 Dystrophin Human genes 0.000 description 1
- 206010021143 Hypoxia Diseases 0.000 description 1
- 108091000080 Phosphotransferase Proteins 0.000 description 1
- 108091027981 Response element Proteins 0.000 description 1
- 102000011856 Utrophin Human genes 0.000 description 1
- 108010075653 Utrophin Proteins 0.000 description 1
- 230000000747 cardiac effect Effects 0.000 description 1
- CVSVTCORWBXHQV-UHFFFAOYSA-N creatine Chemical compound NC(=[NH2+])N(C)CC([O-])=O CVSVTCORWBXHQV-UHFFFAOYSA-N 0.000 description 1
- 239000003862 glucocorticoid Substances 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 210000000663 muscle cell Anatomy 0.000 description 1
- 102000020233 phosphotransferase Human genes 0.000 description 1
- 238000012385 systemic delivery Methods 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023019888A JP2023053254A (ja) | 2017-03-17 | 2023-02-13 | 筋ジストロフィーを治療するための筋肉特異的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 |
| JP2024229759A JP2025061676A (ja) | 2017-03-17 | 2024-12-26 | 筋ジストロフィーを治療するための筋肉特異的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 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762473148P | 2017-03-17 | 2017-03-17 | |
| US62/473,148 | 2017-03-17 | ||
| PCT/US2018/022881 WO2018170408A1 (en) | 2017-03-17 | 2018-03-16 | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023019888A Division JP2023053254A (ja) | 2017-03-17 | 2023-02-13 | 筋ジストロフィーを治療するための筋肉特異的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 |
| JP2024229759A Division JP2025061676A (ja) | 2017-03-17 | 2024-12-26 | 筋ジストロフィーを治療するための筋肉特異的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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020513811A JP2020513811A (ja) | 2020-05-21 |
| JP2020513811A5 true JP2020513811A5 (enExample) | 2021-04-22 |
| JP7676111B2 JP7676111B2 (ja) | 2025-05-14 |
Family
ID=63523321
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019550767A Active JP7676111B2 (ja) | 2017-03-17 | 2018-03-16 | 筋ジストロフィーを治療するための筋肉特異的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 |
| JP2023019888A Pending JP2023053254A (ja) | 2017-03-17 | 2023-02-13 | 筋ジストロフィーを治療するための筋肉特異的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 |
| JP2024229759A Pending JP2025061676A (ja) | 2017-03-17 | 2024-12-26 | 筋ジストロフィーを治療するための筋肉特異的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 After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023019888A Pending JP2023053254A (ja) | 2017-03-17 | 2023-02-13 | 筋ジストロフィーを治療するための筋肉特異的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 |
| JP2024229759A Pending JP2025061676A (ja) | 2017-03-17 | 2024-12-26 | 筋ジストロフィーを治療するための筋肉特異的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 (33)
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| AU2019406214A1 (en) * | 2018-12-21 | 2021-08-05 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
| US12630840B2 (en) * | 2019-02-02 | 2026-05-19 | University of Pittsburgh—of the Commonwealth System of Higher Education | One-step gene therapy for Duchenne muscular dystrophy via gene replacement and anti-inflammation |
| 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 |
| EP3976806A1 (en) * | 2019-05-30 | 2022-04-06 | Solidus Biosciences, Inc. | Recombinant herpesvirales vector |
| 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 |
| CN119061198A (zh) * | 2019-08-29 | 2024-12-03 | 美国西门子医学诊断股份有限公司 | 用于检测aav脱落的试剂和方法 |
| AU2020392243A1 (en) * | 2019-11-28 | 2022-06-16 | Regenxbio Inc. | Microdystrophin gene therapy constructs and uses thereof |
| US20230040544A1 (en) * | 2019-12-16 | 2023-02-09 | Research Institute At Nationwide Children's Hospital | Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc) |
| US11535868B2 (en) | 2020-04-29 | 2022-12-27 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
| IL300187A (en) * | 2020-08-05 | 2023-03-01 | Spacecraft Seven Llc | Gene therapy CSRP3 (protein 3 rich in cysteine and glycine) |
| WO2022029543A1 (en) * | 2020-08-06 | 2022-02-10 | Intas Pharmaceuticals Ltd. | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
| KR20250099412A (ko) * | 2020-11-12 | 2025-07-01 | 프리시젼 바이오사이언시스 인코포레이티드 | 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제 |
| EP4086276A1 (en) | 2021-05-03 | 2022-11-09 | Université d'Aix-Marseille | Composition for treating dysferlinopathy |
| BR112023024078A2 (pt) * | 2021-05-17 | 2024-01-30 | Sarepta Therapeutics Inc | Produção de vetores de aav recombinantes para o tratamento de distrofia muscular |
| EP4108263A3 (en) * | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| WO2023015304A1 (en) * | 2021-08-05 | 2023-02-09 | Insmed Incorporated | Adeno-associated virus particles and methods of use thereof |
| JP2024531138A (ja) * | 2021-08-11 | 2024-08-29 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 筋ジストロフィーを処置するための組成物および方法 |
| 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 |
| CN114316070B (zh) * | 2021-12-29 | 2022-11-15 | 上海勉亦生物科技有限公司 | 用于治疗肌营养不良症的转基因表达盒 |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| CN118460618A (zh) * | 2022-04-19 | 2024-08-09 | 康霖生物科技（杭州）有限公司 | 一种用于遗传性凝血因子缺乏病治疗的核酸构建体 |
| WO2023248251A1 (en) * | 2022-06-24 | 2023-12-28 | Indian Institute Of Technology Kanpur | An optimized aav vector for gene therapy of muscular dystrophy |
| 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 |
| WO2026076414A1 (en) * | 2024-10-03 | 2026-04-09 | General Medicines Llc | Compositions and methods for modulating expression of therapeutic transgenes |
| WO2026080823A1 (en) | 2024-10-10 | 2026-04-16 | Research Institute At Nationwide Children's Hospital | Methods for diagnosing and treating muscular dystrophy diseases |
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| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
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| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| JPH11511326A (ja) | 1995-08-30 | 1999-10-05 | ジエンザイム コーポレイション | アデノウィルスおよびａａｖの精製 |
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| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
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| AU2015208247B2 (en) * | 2014-01-21 | 2021-05-27 | Universiteit Gent | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
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| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| 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 |
| 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 |
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2018
- 2018-03-16 CN CN201880018893.9A patent/CN110997923B/zh active Active
- 2018-03-16 DK DK18768395.8T patent/DK3596222T5/da active
- 2018-03-16 ES ES18768395T patent/ES2948233T3/es active Active
- 2018-03-16 AU AU2018233732A patent/AU2018233732B2/en active Active
- 2018-03-16 MY MYPI2019005365A patent/MY203547A/en unknown
- 2018-03-16 CN CN202311798281.5A patent/CN118497275A/zh active Pending
- 2018-03-16 WO PCT/US2018/022881 patent/WO2018170408A1/en not_active Ceased
- 2018-03-16 FI FIEP18768395.8T patent/FI3596222T3/fi active
- 2018-03-16 EP EP23167357.5A patent/EP4245852A3/en active Pending
- 2018-03-16 KR KR1020197029367A patent/KR102683682B1/ko active Active
- 2018-03-16 MD MDE20200102T patent/MD3596222T2/ro unknown
- 2018-03-16 RS RS20230434A patent/RS64299B1/sr unknown
- 2018-03-16 PL PL18768395.8T patent/PL3596222T3/pl unknown
- 2018-03-16 LT LTEPPCT/US2018/022881T patent/LT3596222T/lt unknown
- 2018-03-16 HU HUE18768395A patent/HUE062476T2/hu unknown
- 2018-03-16 MA MA52112A patent/MA52112B1/fr unknown
- 2018-03-16 MX MX2019011046A patent/MX2019011046A/es unknown
- 2018-03-16 HR HRP20230522TT patent/HRP20230522T1/hr unknown
- 2018-03-16 BR BR112019019248-4A patent/BR112019019248A2/pt active IP Right Grant
- 2018-03-16 PT PT187683958T patent/PT3596222T/pt unknown
- 2018-03-16 KR KR1020247022488A patent/KR102887103B1/ko active Active
- 2018-03-16 IL IL320791A patent/IL320791A/en unknown
- 2018-03-16 CA CA3056638A patent/CA3056638A1/en active Pending
- 2018-03-16 IL IL311713A patent/IL311713B2/en unknown
- 2018-03-16 EP EP18768395.8A patent/EP3596222B1/en active Active
- 2018-03-16 SG SG11201908575S patent/SG11201908575SA/en unknown
- 2018-03-16 SM SM20230201T patent/SMT202300201T1/it unknown
- 2018-03-16 IL IL269391A patent/IL269391B2/en unknown
- 2018-03-16 US US16/494,614 patent/US20200199621A1/en not_active Abandoned
- 2018-03-16 JP JP2019550767A patent/JP7676111B2/ja active Active
- 2018-03-16 EA EA201992201A patent/EA201992201A1/ru unknown
- 2018-03-16 SI SI201830926T patent/SI3596222T1/sl unknown
- 2018-03-16 KR KR1020257037800A patent/KR20250163423A/ko active Pending
- 2018-03-19 AR ARP180100629A patent/AR111292A1/es unknown
- 2018-03-19 TW TW114108403A patent/TW202525835A/zh unknown
- 2018-03-19 TW TW107109334A patent/TWI878204B/zh active
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2019
- 2019-09-17 SA SA519410146A patent/SA519410146B1/ar unknown
- 2019-09-17 MX MX2025007402A patent/MX2025007402A/es unknown
- 2019-09-17 MX MX2025007401A patent/MX2025007401A/es unknown
- 2019-09-20 ZA ZA2019/06251A patent/ZA201906251B/en unknown
- 2019-10-10 CO CONC2019/0011250A patent/CO2019011250A2/es unknown
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2022
- 2022-06-03 US US17/832,325 patent/US20220364117A1/en active Pending
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2023
- 2023-02-13 JP JP2023019888A patent/JP2023053254A/ja active Pending
- 2023-06-21 CY CY20231100291T patent/CY1126080T1/el unknown
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2024
- 2024-11-19 AU AU2024266707A patent/AU2024266707B2/en active Active
- 2024-12-26 JP JP2024229759A patent/JP2025061676A/ja active Pending
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2025
- 2025-01-22 AU AU2025200437A patent/AU2025200437A1/en active Pending