JP2018503374A5 - - Google Patents
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- JP2018503374A5 JP2018503374A5 JP2017536343A JP2017536343A JP2018503374A5 JP 2018503374 A5 JP2018503374 A5 JP 2018503374A5 JP 2017536343 A JP2017536343 A JP 2017536343A JP 2017536343 A JP2017536343 A JP 2017536343A JP 2018503374 A5 JP2018503374 A5 JP 2018503374A5
- Authority
- JP
- Japan
- Prior art keywords
- spectrin
- repeat
- promoter
- isolated
- nucleotide sequence
- 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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- 108090000623 proteins and genes Proteins 0.000 claims description 87
- 239000008194 pharmaceutical composition Substances 0.000 claims description 41
- 239000002773 nucleotide Substances 0.000 claims description 40
- 125000003729 nucleotide group Chemical group 0.000 claims description 40
- 102000004169 proteins and genes Human genes 0.000 claims description 40
- 201000006938 muscular dystrophy Diseases 0.000 claims description 27
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 24
- 150000007523 nucleic acids Chemical group 0.000 claims description 24
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 20
- 108010069091 Dystrophin Proteins 0.000 claims description 16
- 230000000747 cardiac effect Effects 0.000 claims description 7
- 238000002955 isolation Methods 0.000 claims description 6
- 102000004987 Troponin T Human genes 0.000 claims description 5
- 108090001108 Troponin T Proteins 0.000 claims description 5
- 102000002151 Microfilament Proteins Human genes 0.000 claims description 4
- 108010040897 Microfilament Proteins Proteins 0.000 claims description 4
- 102000006538 Nitric Oxide Synthase Type I Human genes 0.000 claims description 4
- 108010008858 Nitric Oxide Synthase Type I Proteins 0.000 claims description 4
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 4
- 210000000663 muscle cell Anatomy 0.000 claims description 3
- 210000002948 striated muscle cell Anatomy 0.000 claims description 3
- 102100025682 Dystroglycan 1 Human genes 0.000 claims description 2
- 108010071885 Dystroglycans Proteins 0.000 claims description 2
- 102000005890 Spectrin Human genes 0.000 claims description 2
- 108010019965 Spectrin Proteins 0.000 claims description 2
- 238000000746 purification Methods 0.000 claims description 2
- 238000000034 method Methods 0.000 description 53
- 241000702421 Dependoparvovirus Species 0.000 description 19
- 239000013598 vector Substances 0.000 description 19
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 description 12
- 210000003205 muscle Anatomy 0.000 description 7
- 230000001575 pathological effect Effects 0.000 description 7
- 208000024891 symptom Diseases 0.000 description 7
- 239000013607 AAV vector Substances 0.000 description 6
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 description 6
- 201000006815 congenital muscular dystrophy Diseases 0.000 description 6
- 239000013608 rAAV vector Substances 0.000 description 6
- 210000002027 skeletal muscle Anatomy 0.000 description 6
- 201000006935 Becker muscular dystrophy Diseases 0.000 description 3
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 description 3
- 201000009338 distal myopathy Diseases 0.000 description 3
- 230000001965 increasing effect Effects 0.000 description 3
- 230000003274 myotonic effect Effects 0.000 description 3
- 238000011321 prophylaxis Methods 0.000 description 3
- 206010003591 Ataxia Diseases 0.000 description 2
- 208000031229 Cardiomyopathies Diseases 0.000 description 2
- 102000001039 Dystrophin Human genes 0.000 description 2
- 206010016654 Fibrosis Diseases 0.000 description 2
- 102000003886 Glycoproteins Human genes 0.000 description 2
- 108090000288 Glycoproteins Proteins 0.000 description 2
- 206010061218 Inflammation Diseases 0.000 description 2
- 241000124008 Mammalia Species 0.000 description 2
- 208000010428 Muscle Weakness Diseases 0.000 description 2
- 206010028289 Muscle atrophy Diseases 0.000 description 2
- 206010049565 Muscle fatigue Diseases 0.000 description 2
- 206010028372 Muscular weakness Diseases 0.000 description 2
- 208000000112 Myalgia Diseases 0.000 description 2
- 230000007812 deficiency Effects 0.000 description 2
- 229910001651 emery Inorganic materials 0.000 description 2
- 230000004761 fibrosis Effects 0.000 description 2
- 230000005021 gait Effects 0.000 description 2
- 230000004054 inflammatory process Effects 0.000 description 2
- 230000004807 localization Effects 0.000 description 2
- 230000020763 muscle atrophy Effects 0.000 description 2
- 208000013465 muscle pain Diseases 0.000 description 2
- 201000000585 muscular atrophy Diseases 0.000 description 2
- 210000004165 myocardium Anatomy 0.000 description 2
- 210000001087 myotubule Anatomy 0.000 description 2
- 238000011282 treatment Methods 0.000 description 2
- 206010006895 Cachexia Diseases 0.000 description 1
- 229920003266 Leaf® Polymers 0.000 description 1
- 208000019622 heart disease Diseases 0.000 description 1
- 239000000203 mixture Substances 0.000 description 1
- 208000001076 sarcopenia Diseases 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562104537P | 2015-01-16 | 2015-01-16 | |
| US62/104,537 | 2015-01-16 | ||
| PCT/US2016/013733 WO2016115543A2 (en) | 2015-01-16 | 2016-01-15 | Novel micro-dystrophins and related methods of use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021014384A Division JP7164896B2 (ja) | 2015-01-16 | 2021-02-01 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018503374A JP2018503374A (ja) | 2018-02-08 |
| JP2018503374A5 true JP2018503374A5 (enExample) | 2019-02-21 |
| JP6832280B2 JP6832280B2 (ja) | 2021-02-24 |
Family
ID=56406571
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017536343A Active JP6832280B2 (ja) | 2015-01-16 | 2016-01-15 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2021014384A Active JP7164896B2 (ja) | 2015-01-16 | 2021-02-01 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2022165111A Active JP7482549B2 (ja) | 2015-01-16 | 2022-10-14 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2024068905A Pending JP2024088814A (ja) | 2015-01-16 | 2024-04-22 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021014384A Active JP7164896B2 (ja) | 2015-01-16 | 2021-02-01 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2022165111A Active JP7482549B2 (ja) | 2015-01-16 | 2022-10-14 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2024068905A Pending JP2024088814A (ja) | 2015-01-16 | 2024-04-22 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US10479821B2 (enExample) |
| EP (2) | EP4599851A2 (enExample) |
| JP (4) | JP6832280B2 (enExample) |
| KR (1) | KR102684387B1 (enExample) |
| CN (1) | CN107250364A (enExample) |
| AU (2) | AU2016206518B2 (enExample) |
| CA (1) | CA2972811A1 (enExample) |
| IL (4) | IL315273A (enExample) |
| NZ (1) | NZ734019A (enExample) |
| SG (1) | SG11201705324UA (enExample) |
| WO (1) | WO2016115543A2 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11958888B2 (en) | 2015-01-16 | 2024-04-16 | University Of Washington | Micro-dystrophins and related methods of use |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| ES3040945T3 (en) | 2015-10-28 | 2025-11-06 | Vertex Pharma | Materials and methods for treatment of duchenne muscular dystrophy |
| 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 |
| US20170362635A1 (en) * | 2016-06-20 | 2017-12-21 | University Of Washington | Muscle-specific crispr/cas9 editing of genes |
| CA2971303A1 (en) | 2016-06-21 | 2017-12-21 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| JOP20190166A1 (ar) * | 2017-01-05 | 2019-07-02 | Univ Texas | استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي |
| BR112019015081A2 (pt) | 2017-01-23 | 2020-03-10 | Rutgers, The State University Of New Jersey | Composições para reduzir a expressão da sarcolipina e prevenir e tratar a distrofia muscular e a cardiomiopatia e os métodos de utilização |
| US10687520B2 (en) | 2017-03-07 | 2020-06-23 | The Board Of Regents Of The University Of Texas System | Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44 |
| 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 |
| 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 |
| TW201936201A (zh) | 2017-12-14 | 2019-09-16 | 美商堅固生物科技公司 | 基因之非病毒生產及遞送 |
| SG11202006722RA (en) | 2018-01-31 | 2020-08-28 | Res Inst Nationwide Childrens Hospital | Gene therapy for limb-girdle muscular dystrophy type 2c |
| EP3780952A4 (en) | 2018-04-16 | 2022-03-09 | The Trustees of the University of Pennsylvania | COMPOSITIONS AND METHODS OF TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY |
| 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 |
| 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 |
| AU2019333315B2 (en) | 2018-08-30 | 2022-09-29 | Tenaya Therapeutics, Inc. | Cardiac cell reprogramming with myocardin and ASCL1 |
| BR112021007480A2 (pt) * | 2018-10-24 | 2021-08-10 | Bristol-Myers Squibb Company | distrofinas miniaturizadas e seus usos |
| 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 |
| CN114207120A (zh) | 2019-05-30 | 2022-03-18 | 坚固生物科技公司 | 重组疱疹病毒载体 |
| US20200405824A1 (en) * | 2019-06-26 | 2020-12-31 | University Of Washington | Use of ribonucleotide reductase alone or in combination with micro-dystrophin to treat duchenne muscular dystrophy striated muscle disease |
| 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 |
| CA3153902A1 (en) | 2019-10-16 | 2021-04-22 | Pardis SABETI | Engineered muscle targeting compositions |
| KR20220107222A (ko) * | 2019-11-28 | 2022-08-02 | 리젠엑스바이오 인크. | 마이크로디스트로핀 유전자 치료 작제물 및 이의 용도 |
| WO2021163357A2 (en) * | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| MY209860A (en) * | 2020-02-13 | 2025-08-07 | Tenaya Therapeutics Inc | Gene therapy vectors for treating heart disease |
| 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 |
| WO2022004788A1 (ja) * | 2020-06-30 | 2022-01-06 | 東レ株式会社 | 代謝異常を伴う疾患又は症候群における筋力低下症状の改善剤又は予防剤 |
| WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| US20240003898A1 (en) | 2020-10-30 | 2024-01-04 | Pfizer Inc. | Methods for measuring dystrophin in tissue samples |
| RU2767335C1 (ru) * | 2021-03-02 | 2022-03-17 | Общество с ограниченной ответственностью «Марлин Биотех» | Химерные белки на основе утрофина и дистрофина человека и их применение для лечения миодистрофии Дюшенна |
| WO2022216749A1 (en) | 2021-04-05 | 2022-10-13 | Solid Biosciences Inc. | Recombinant herpesvirales vector |
| US20240200093A1 (en) * | 2021-04-09 | 2024-06-20 | University Of Washington | Artificial regulatory cassettes for muscle-specific gene expression |
| CA3216744A1 (en) * | 2021-04-26 | 2022-11-03 | Regenxbio Inc. | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
| US20240358857A1 (en) * | 2021-05-11 | 2024-10-31 | Regenxbio Inc. | Treatment of duchenne muscular dystrophy and combinations thereof |
| 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 |
| US20240269329A1 (en) * | 2021-06-07 | 2024-08-15 | University Of Florida Research Foundation, Incorporated | Gene therapy for duchenne muscular dystrophy |
| US20250019721A1 (en) | 2021-08-11 | 2025-01-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| KR20240095165A (ko) | 2021-08-11 | 2024-06-25 | 솔리드 바이오사이언시즈 인크. | 근이영양증의 치료 |
| WO2023060269A1 (en) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for targeted delivery |
| 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 |
| WO2023081850A1 (en) | 2021-11-04 | 2023-05-11 | Regeneron Pharmaceuticals, Inc. | Viral particles retargeted to skeletal muscle |
| 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 |
| US20250288641A1 (en) * | 2022-04-27 | 2025-09-18 | The Curators Of The University Of Missouri | Micro-dystrophin for heart protection |
| WO2024138158A2 (en) * | 2022-12-23 | 2024-06-27 | Ultragenyx Pharmaceutical Inc. | Method of producing soluble microdystrophin protein and uses of the same |
| AU2024220647A1 (en) | 2023-02-13 | 2025-08-21 | Regeneron Pharmaceuticals, Inc. | Treatment of muscle related disorders with anti-human cacng1 antibodies |
| WO2024216244A2 (en) | 2023-04-13 | 2024-10-17 | Regenxbio Inc. | Targeting aav capsids, methods of manufacturing and using same |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE60134786D1 (de) * | 2000-10-06 | 2008-08-21 | Univ Michigan | Mini-dystrophin nukleinsäure- und peptidsequenzen |
| US20070202587A1 (en) | 2002-09-23 | 2007-08-30 | Applied Genetic Technologies Corporation | Recombinant AAV production in mammalian cells |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US20080044393A1 (en) * | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
| US7436622B2 (en) * | 2006-07-31 | 2008-10-14 | Imation Corp. | Concurrent servo and data track writing |
| JP5575486B2 (ja) * | 2007-01-18 | 2014-08-20 | ユニヴァーシティ オブ ミズーリー−コロンビア | 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子 |
| WO2009097129A1 (en) | 2008-01-29 | 2009-08-06 | Applied Genetic Technologies Corporation | Recombinant virus production using mammalian cells in suspension |
| KR101555083B1 (ko) * | 2008-11-11 | 2015-09-22 | 인터내셔널 비지네스 머신즈 코포레이션 | 제공 장치, 시스템, 서버 장치, 프로그램 및 방법 |
| KR20140035967A (ko) * | 2011-05-26 | 2014-03-24 | 유니버시티 오브 워싱톤 | 세포 및 유전자에 기반한 심장 기능을 개선하는 방법 |
| US20130136729A1 (en) * | 2011-11-11 | 2013-05-30 | University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group | Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors |
| US9624282B2 (en) * | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| US20150329609A1 (en) * | 2014-03-03 | 2015-11-19 | Sujata Acharjee | Chimeric dystrophin proteins to treat dystrophinopathies |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| AU2016206518B2 (en) * | 2015-01-16 | 2020-03-05 | University Of Washington | Novel micro-dystrophins and related methods of use |
-
2016
- 2016-01-15 AU AU2016206518A patent/AU2016206518B2/en active Active
- 2016-01-15 JP JP2017536343A patent/JP6832280B2/ja active Active
- 2016-01-15 EP EP25169094.7A patent/EP4599851A2/en active Pending
- 2016-01-15 IL IL315273A patent/IL315273A/en unknown
- 2016-01-15 CN CN201680005836.8A patent/CN107250364A/zh active Pending
- 2016-01-15 NZ NZ734019A patent/NZ734019A/en unknown
- 2016-01-15 WO PCT/US2016/013733 patent/WO2016115543A2/en not_active Ceased
- 2016-01-15 SG SG11201705324UA patent/SG11201705324UA/en unknown
- 2016-01-15 IL IL286316A patent/IL286316B2/en unknown
- 2016-01-15 EP EP16738022.9A patent/EP3245291A4/en active Pending
- 2016-01-15 US US15/541,870 patent/US10479821B2/en active Active
- 2016-01-15 CA CA2972811A patent/CA2972811A1/en active Pending
- 2016-01-15 KR KR1020177022718A patent/KR102684387B1/ko active Active
- 2016-01-15 IL IL297239A patent/IL297239B2/en unknown
-
2017
- 2017-06-29 IL IL253246A patent/IL253246B/en unknown
-
2019
- 2019-10-04 US US16/593,833 patent/US11958888B2/en active Active
- 2019-10-04 US US16/593,840 patent/US12421285B2/en active Active
-
2020
- 2020-05-22 AU AU2020203358A patent/AU2020203358B2/en active Active
-
2021
- 2021-02-01 JP JP2021014384A patent/JP7164896B2/ja active Active
-
2022
- 2022-10-14 JP JP2022165111A patent/JP7482549B2/ja active Active
-
2024
- 2024-04-22 JP JP2024068905A patent/JP2024088814A/ja active Pending
-
2025
- 2025-08-22 US US19/307,571 patent/US20250376496A1/en active Pending
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11958888B2 (en) | 2015-01-16 | 2024-04-16 | University Of Washington | Micro-dystrophins and related methods of use |
| US12421285B2 (en) | 2015-01-16 | 2025-09-23 | University Of Washington | Micro-dystrophins and related methods of use |
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