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.)
- Granted
Links
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 (https=) | 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 (https=) |
| EP (2) | EP4599851A3 (https=) |
| JP (4) | JP6832280B2 (https=) |
| KR (1) | KR102684387B1 (https=) |
| CN (1) | CN107250364A (https=) |
| AU (2) | AU2016206518B2 (https=) |
| CA (1) | CA2972811A1 (https=) |
| IL (4) | IL286316B2 (https=) |
| NZ (1) | NZ734019A (https=) |
| SG (1) | SG11201705324UA (https=) |
| WO (1) | WO2016115543A2 (https=) |
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 |
| AU2016344609B2 (en) | 2015-10-28 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | 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 |
| ES2977730T3 (es) | 2016-04-15 | 2024-08-29 | Res Inst Nationwide Childrens Hospital | Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular |
| US20170362635A1 (en) * | 2016-06-20 | 2017-12-21 | University Of Washington | Muscle-specific crispr/cas9 editing of genes |
| CA2971303C (en) | 2016-06-21 | 2026-03-03 | 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 |
| EP3570896B1 (en) * | 2017-01-23 | 2023-07-26 | Rutgers, The State University of New Jersey | Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use |
| 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 |
| EP3596112A2 (en) | 2017-03-17 | 2020-01-22 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| MA50836A (fr) | 2017-10-18 | 2020-08-26 | Res Inst Nationwide Childrens Hospital | Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire |
| WO2019118806A1 (en) | 2017-12-14 | 2019-06-20 | Solid Biosciences Inc. | Non-viral production and delivery of genes |
| AR114350A1 (es) | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
| US12460226B2 (en) | 2018-04-16 | 2025-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating 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 |
| JP7450945B2 (ja) | 2018-08-30 | 2024-03-18 | テナヤ セラピューティクス， インコーポレイテッド | ミオカルディンおよびａｓｃｌ１を用いた心細胞リプログラミング |
| MX2021004688A (es) * | 2018-10-24 | 2021-06-04 | Bristol Myers Squibb Co | Distrofinas miniaturizadas y usos de las mismas. |
| WO2020123645A1 (en) * | 2018-12-12 | 2020-06-18 | Solid Biosciences Inc. | Combination therapy for treating muscular dystrophy |
| AU2020229340A1 (en) | 2019-02-26 | 2021-09-16 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy |
| US12466864B2 (en) * | 2019-03-25 | 2025-11-11 | Genethon | Production of large-sized quasidystrophins using overlapping AAV vectors |
| AU2020284255A1 (en) * | 2019-05-30 | 2022-01-06 | Solid Biosciences Inc. | Recombinant herpesvirales vector |
| 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 |
| RS65421B1 (sr) | 2019-08-21 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| TWI908746B (zh) * | 2019-11-28 | 2025-12-21 | 美商銳進科斯生物股份有限公司 | 微小肌縮蛋白基因療法之構築體及其用途 |
| EP4103214B1 (en) * | 2020-02-13 | 2026-04-29 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| WO2021163357A2 (en) * | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| IL297753A (en) | 2020-04-29 | 2022-12-01 | Bristol Myers Squibb Co | Minimized dystrophins with spectrin fusion domains and uses thereof |
| IL299094A (en) | 2020-06-15 | 2023-02-01 | Res Inst Nationwide Childrens Hospital | Administration of an adeno-associated virus vector for muscular dystrophies |
| TWI893157B (zh) * | 2020-06-30 | 2025-08-11 | 日商東麗股份有限公司 | 具有嗎啡喃骨架之化合物或其藥理學上可容許之酸加成鹽的用途 |
| 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 |
| EP4320139A1 (en) | 2021-04-05 | 2024-02-14 | Solid Biosciences Inc. | Recombinant herpesvirales vector |
| EP4707401A2 (en) * | 2021-04-09 | 2026-03-11 | University of Washington | Artificial regulatory cassettes for muscle-specific gene expression |
| JP2024517143A (ja) * | 2021-04-26 | 2024-04-19 | リジェネックスバイオ インコーポレイテッド | ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与 |
| EP4337267A1 (en) * | 2021-05-11 | 2024-03-20 | 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 |
| WO2023018854A2 (en) | 2021-08-11 | 2023-02-16 | Solid Biosciences Inc. | Treatment of muscular dystrophy |
| 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 |
| JP2024540181A (ja) | 2021-11-04 | 2024-10-31 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 骨格筋に再標的化されたウイルス粒子 |
| 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 |
| EP4665764A1 (en) | 2023-02-13 | 2025-12-24 | 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 |
|---|---|---|---|---|
| EP1366160B1 (en) | 2000-10-06 | 2008-07-09 | The Regents Of The University Of Michigan | Mini-dystrophin nucleic acid and peptide sequences |
| US20070202587A1 (en) | 2002-09-23 | 2007-08-30 | Applied Genetic Technologies Corporation | Recombinant AAV production in mammalian cells |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | 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 |
| CN101711164B (zh) * | 2007-01-18 | 2014-06-04 | 密苏里-哥伦比亚大学 | 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 |
| ES2751999T3 (es) | 2008-01-29 | 2020-04-02 | Applied Genetic Tech Corporation | Producción recombinante de virus adeno-asociados usando células BHK en suspensión |
| EP2365443B1 (en) * | 2008-11-11 | 2018-07-04 | International Business Machines Corporation | Data providing device, system, server device, program, and method |
| EP2714709B1 (en) * | 2011-05-26 | 2018-04-18 | University of Washington | Compositions for use in cell and gene based methods to improve cardiac function |
| 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 |
| JP6832280B2 (ja) | 2015-01-16 | 2021-02-24 | ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ | 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 |
-
2016
- 2016-01-15 JP JP2017536343A patent/JP6832280B2/ja active Active
- 2016-01-15 EP EP25169094.7A patent/EP4599851A3/en active Pending
- 2016-01-15 CA CA2972811A patent/CA2972811A1/en active Pending
- 2016-01-15 KR KR1020177022718A patent/KR102684387B1/ko active Active
- 2016-01-15 SG SG11201705324UA patent/SG11201705324UA/en unknown
- 2016-01-15 IL IL286316A patent/IL286316B2/en unknown
- 2016-01-15 IL IL315273A patent/IL315273A/en unknown
- 2016-01-15 AU AU2016206518A patent/AU2016206518B2/en active Active
- 2016-01-15 WO PCT/US2016/013733 patent/WO2016115543A2/en not_active Ceased
- 2016-01-15 IL IL297239A patent/IL297239B2/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 US US15/541,870 patent/US10479821B2/en active Active
- 2016-01-15 EP EP16738022.9A patent/EP3245291B1/en active Active
-
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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