JP2019513393A5 - - Google Patents
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- JP2019513393A5 JP2019513393A5 JP2018553951A JP2018553951A JP2019513393A5 JP 2019513393 A5 JP2019513393 A5 JP 2019513393A5 JP 2018553951 A JP2018553951 A JP 2018553951A JP 2018553951 A JP2018553951 A JP 2018553951A JP 2019513393 A5 JP2019513393 A5 JP 2019513393A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- recombinant aav
- subject
- muscular dystrophy
- vector
- 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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- 239000000203 mixture Substances 0.000 claims description 30
- 239000013607 AAV vector Substances 0.000 claims description 24
- 210000003205 muscle Anatomy 0.000 claims description 20
- 201000006938 muscular dystrophy Diseases 0.000 claims description 13
- 239000002773 nucleotide Substances 0.000 claims description 10
- 125000003729 nucleotide group Chemical group 0.000 claims description 10
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 8
- 206010016654 Fibrosis Diseases 0.000 claims description 8
- 230000004761 fibrosis Effects 0.000 claims description 8
- 238000000034 method Methods 0.000 claims description 6
- 239000013598 vector Substances 0.000 claims description 6
- 239000003814 drug Substances 0.000 claims description 5
- 102000001039 Dystrophin Human genes 0.000 claims description 4
- 108010069091 Dystrophin Proteins 0.000 claims description 4
- 108090000623 proteins and genes Proteins 0.000 claims description 4
- 102000004169 proteins and genes Human genes 0.000 claims description 4
- 238000007911 parenteral administration Methods 0.000 claims description 3
- 238000007910 systemic administration Methods 0.000 claims description 3
- 238000007918 intramuscular administration Methods 0.000 claims description 2
- 238000010253 intravenous injection Methods 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 230000001105 regulatory effect Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 description 4
- 239000013608 rAAV vector Substances 0.000 description 4
- 108010059343 MM Form Creatine Kinase Proteins 0.000 description 3
- 108010085238 Actins Proteins 0.000 description 2
- 108090000362 Lymphotoxin-beta Proteins 0.000 description 2
- 102000005604 Myosin Heavy Chains Human genes 0.000 description 2
- 108010084498 Myosin Heavy Chains Proteins 0.000 description 2
- 210000004027 cell Anatomy 0.000 description 2
- 108091007431 miR-29 Proteins 0.000 description 2
- 108091029162 miR-29 stem-loop 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
- 241000702421 Dependoparvovirus Species 0.000 description 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 1
- 206010021143 Hypoxia Diseases 0.000 description 1
- 108091027981 Response element Proteins 0.000 description 1
- 108010065729 Troponin I Proteins 0.000 description 1
- 230000005856 abnormality Effects 0.000 description 1
- 230000000747 cardiac effect Effects 0.000 description 1
- 238000002648 combination therapy Methods 0.000 description 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
- 208000035475 disorder Diseases 0.000 description 1
- 239000003623 enhancer Substances 0.000 description 1
- 239000003862 glucocorticoid Substances 0.000 description 1
- 230000007954 hypoxia Effects 0.000 description 1
- 238000010255 intramuscular injection Methods 0.000 description 1
- 239000007927 intramuscular injection Substances 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 108091047189 miR-29c stem-loop Proteins 0.000 description 1
- 108091054490 miR-29c-2 stem-loop Proteins 0.000 description 1
- 210000000663 muscle cell Anatomy 0.000 description 1
- 108091033319 polynucleotide Proteins 0.000 description 1
- 239000002157 polynucleotide Substances 0.000 description 1
- 102000040430 polynucleotide Human genes 0.000 description 1
- 210000002027 skeletal muscle Anatomy 0.000 description 1
- 150000003431 steroids Chemical class 0.000 description 1
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021190072A 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 JP2024023439A (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 JP2025118741A (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 |
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/US2017/027636 WO2017181015A1 (en) | 2016-04-15 | 2017-04-14 | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021190072A Division 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019513393A JP2019513393A (ja) | 2019-05-30 |
| JP2019513393A5 true JP2019513393A5 (enExample) | 2020-05-28 |
| JP7078546B2 JP7078546B2 (ja) | 2022-05-31 |
Family
ID=60041940
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
| 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 |
| 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 Pending JP2024023439A (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 Pending JP2025118741A (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 (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 Pending JP2024023439A (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 Pending JP2025118741A (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 (28)
| Country | Link |
|---|---|
| US (6) | US11298429B2 (enExample) |
| EP (5) | EP3442602B1 (enExample) |
| JP (6) | JP7153562B2 (enExample) |
| KR (3) | KR102649707B1 (enExample) |
| CN (2) | CN118895311A (enExample) |
| AU (4) | AU2017250790B2 (enExample) |
| BR (1) | BR112018071182A2 (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) | MY195438A (enExample) |
| NZ (2) | NZ787316A (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) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102877920B1 (ko) | 2015-11-16 | 2025-10-30 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법 |
| MA45477A (fr) | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| JP7162021B2 (ja) | 2017-03-17 | 2022-10-27 | ニューカッスル ユニバーシティ | 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断片のアデノ随伴ウイルスベクター送達 |
| WO2018170408A1 (en) * | 2017-03-17 | 2018-09-20 | Research Institute At Nationwide Children's Hospital, Inc. | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| US11534501B2 (en) | 2017-10-18 | 2022-12-27 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| EP3697916A4 (en) * | 2017-10-20 | 2021-08-11 | Research Institute at Nationwide Children's Hospital | METHODS AND MATERIALS FOR NT-3 GENE THERAPY |
| SG11202006722RA (en) | 2018-01-31 | 2020-08-28 | Res Inst Nationwide Childrens Hospital | Gene therapy for limb-girdle muscular dystrophy type 2c |
| EP3765624A4 (en) * | 2018-03-16 | 2022-05-25 | Research Institute at Nationwide Children's Hospital | Increasing tissue specific gene delivery by capsid modification |
| WO2019220435A1 (en) * | 2018-05-15 | 2019-11-21 | Ramot At Tel-Aviv University Ltd. | Mir126-5p for treating motor neuron diseases |
| MY208145A (en) * | 2018-06-18 | 2025-04-18 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| EP3807309A1 (en) * | 2018-06-18 | 2021-04-21 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies |
| KR20210028162A (ko) | 2018-06-29 | 2021-03-11 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 |
| EP3598978B1 (en) | 2018-07-26 | 2024-05-29 | EXOFIX S.r.l. | Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles |
| US20220031865A1 (en) * | 2018-12-12 | 2022-02-03 | Solid Biosciences Inc. | Combination therapy for treating muscular dystrophy |
| CA3131390A1 (en) | 2019-02-26 | 2020-09-03 | Research Institute Of Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| CA3130196A1 (en) * | 2019-03-25 | 2020-10-01 | Isabelle Richard | Production of large-sized quasidystrophins using overlapping aav vectors |
| HRP20240505T1 (hr) | 2019-08-21 | 2024-07-05 | Research Institute At Nationwide Children's Hospital | Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije |
| WO2021142435A1 (en) * | 2020-01-10 | 2021-07-15 | Solid Biosciences Inc. | Combination therapy for treating muscular dystrophy |
| EP4087617A4 (en) * | 2020-01-10 | 2024-05-01 | Solid Biosciences Inc. | VIRAL VECTOR FOR COMBINATION THERAPY |
| MX2022013477A (es) | 2020-04-29 | 2022-11-30 | Bristol Myers Squibb Co | Distrofinas miniaturizadas con dominios de fusion de espectrina y usos de las mismas. |
| WO2021257595A1 (en) * | 2020-06-15 | 2021-12-23 | 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 |
| KR20240095165A (ko) | 2021-08-11 | 2024-06-25 | 솔리드 바이오사이언시즈 인크. | 근이영양증의 치료 |
| 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 |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| 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 |
| EP4577663A2 (en) | 2022-08-24 | 2025-07-02 | RegenxBio Inc. | Recombinant adeno-associated viruses and uses thereof |
| WO2025188755A1 (en) * | 2024-03-04 | 2025-09-12 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy |
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| AU2016206518B2 (en) | 2015-01-16 | 2020-03-05 | University Of Washington | Novel micro-dystrophins and related methods of use |
| GB201507842D0 (en) | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
| WO2017165859A1 (en) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| MA45477A (fr) | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| HUE066442T2 (hu) | 2016-04-15 | 2024-08-28 | Res Institute At Nationwide | ß-szarkoglikán és mikroRNS-29 adenoasszociáltvírus-vektorral történõ bejuttatása és izomdisztrófia kezelése |
| CA2971303A1 (en) | 2016-06-21 | 2017-12-21 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| MY208145A (en) | 2018-06-18 | 2025-04-18 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
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