JP2021500352A5 - - Google Patents
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- Publication number
- JP2021500352A5 JP2021500352A5 JP2020522046A JP2020522046A JP2021500352A5 JP 2021500352 A5 JP2021500352 A5 JP 2021500352A5 JP 2020522046 A JP2020522046 A JP 2020522046A JP 2020522046 A JP2020522046 A JP 2020522046A JP 2021500352 A5 JP2021500352 A5 JP 2021500352A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- recombinant aav
- aav vector
- effective amount
- therapeutically effective
- 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
- 239000002773 nucleotide Substances 0.000 claims description 88
- 125000003729 nucleotide group Chemical group 0.000 claims description 88
- 239000013607 AAV vector Substances 0.000 claims description 65
- 210000003205 muscle Anatomy 0.000 claims description 44
- 239000000203 mixture Substances 0.000 claims description 29
- 201000006938 muscular dystrophy Diseases 0.000 claims description 18
- 239000003814 drug Substances 0.000 claims description 11
- 238000001802 infusion Methods 0.000 claims description 6
- 238000002347 injection Methods 0.000 claims description 6
- 239000007924 injection Substances 0.000 claims description 6
- 238000002054 transplantation Methods 0.000 claims description 6
- 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
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 5
- 206010016654 Fibrosis Diseases 0.000 claims description 5
- 229940079593 drug Drugs 0.000 claims description 5
- 230000004761 fibrosis Effects 0.000 claims description 5
- 238000007911 parenteral administration Methods 0.000 claims description 4
- 238000012385 systemic delivery Methods 0.000 claims description 4
- 238000010255 intramuscular injection Methods 0.000 claims description 3
- 238000010253 intravenous injection Methods 0.000 claims description 3
- 239000013598 vector Substances 0.000 claims description 3
- 238000007918 intramuscular administration Methods 0.000 claims description 2
- 239000007927 intramuscular injection Substances 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 229940126585 therapeutic drug Drugs 0.000 claims description 2
- 230000001225 therapeutic effect Effects 0.000 claims description 2
- 238000005728 strengthening Methods 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 description 45
- 108091047189 miR-29c stem-loop Proteins 0.000 description 36
- 108091054490 miR-29c-2 stem-loop Proteins 0.000 description 36
- 238000000034 method Methods 0.000 description 19
- 108010059343 MM Form Creatine Kinase Proteins 0.000 description 12
- 108090000623 proteins and genes Proteins 0.000 description 12
- 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 9
- 239000003623 enhancer Substances 0.000 description 9
- 108010085238 Actins Proteins 0.000 description 6
- 230000002107 myocardial effect Effects 0.000 description 6
- 102000004169 proteins and genes Human genes 0.000 description 6
- 210000002027 skeletal muscle Anatomy 0.000 description 6
- 108091028043 Nucleic acid sequence Proteins 0.000 description 5
- 102000004420 Creatine Kinase Human genes 0.000 description 3
- 108010042126 Creatine kinase Proteins 0.000 description 3
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 3
- 206010021143 Hypoxia Diseases 0.000 description 3
- 108060008487 Myosin Proteins 0.000 description 3
- 102000003505 Myosin Human genes 0.000 description 3
- 108091027981 Response element Proteins 0.000 description 3
- 108010051583 Ventricular Myosins Proteins 0.000 description 3
- 239000003862 glucocorticoid Substances 0.000 description 3
- 210000000663 muscle cell Anatomy 0.000 description 3
- 230000003387 muscular Effects 0.000 description 3
- 102000001039 Dystrophin Human genes 0.000 description 1
- 108010069091 Dystrophin Proteins 0.000 description 1
- 230000002708 enhancing effect Effects 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762573955P | 2017-10-18 | 2017-10-18 | |
| US62/573,955 | 2017-10-18 | ||
| PCT/US2018/022853 WO2019078916A1 (en) | 2017-10-18 | 2018-03-16 | ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021500352A JP2021500352A (ja) | 2021-01-07 |
| JP2021500352A5 true JP2021500352A5 (cg-RX-API-DMAC7.html) | 2021-04-30 |
| JP7213238B2 JP7213238B2 (ja) | 2023-01-26 |
Family
ID=66173432
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020522046A Active JP7213238B2 (ja) | 2017-10-18 | 2018-03-16 | 筋ジストロフィーを治療するための筋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 (5)
| Country | Link |
|---|---|
| US (2) | US11534501B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3697915A4 (cg-RX-API-DMAC7.html) |
| JP (1) | JP7213238B2 (cg-RX-API-DMAC7.html) |
| MA (1) | MA50836A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2019078916A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3005633C (en) | 2015-11-16 | 2023-11-21 | Research Institute Of Nationwide Children's Hospital | Materials and methods for treatment of titin-based myopathies and other titinopathies |
| 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 |
| KR20250099758A (ko) | 2018-01-31 | 2025-07-02 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 2c형 지대근이영양증에 대한 유전자 치료 |
| EP3807413B1 (en) | 2018-06-18 | 2025-05-21 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| CN112543810A (zh) | 2018-06-29 | 2021-03-23 | 全国儿童医院研究所 | 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法 |
| CN113766935A (zh) | 2019-02-26 | 2021-12-07 | 全国儿童医院研究所 | β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗 |
| EP4488287A3 (en) | 2019-08-21 | 2025-03-05 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| IL297753A (en) | 2020-04-29 | 2022-12-01 | Bristol Myers Squibb Co | Minimized dystrophins with spectrin fusion domains and uses thereof |
| MX2022016332A (es) * | 2020-06-15 | 2023-04-03 | Res Inst Nationwide Childrens Hospital | Administracion de vectores de virus adenoasociados para distrofias musculares. |
| 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 |
| 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 |
| 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 |
| CN115838725B (zh) * | 2022-12-30 | 2023-09-08 | 广州派真生物技术有限公司 | 在哺乳动物心脏中特异性启动基因的启动子序列及其应用 |
Family Cites Families (46)
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| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| CA2176215C (en) | 1993-11-09 | 2007-06-26 | James P. Trempe | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| WO1996017947A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| CA2230655C (en) | 1995-08-30 | 2008-06-17 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| WO1997009441A2 (en) | 1995-09-08 | 1997-03-13 | Genzyme Corporation | Improved aav vectors for gene therapy |
| US5795872A (en) | 1995-09-19 | 1998-08-18 | Pharmadigm, Inc. | DNA construct for immunization |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| ES2344660T3 (es) | 1996-09-06 | 2010-09-02 | The Trustees Of The University Of Pennsylvania | Uso de virus adeno-asociados recombinantes en la preparacion de un medicamento para la terapia genica mediante celulas musculares. |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO1999011764A2 (en) | 1997-09-05 | 1999-03-11 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of recombinant aav vectors |
| JPH11318467A (ja) | 1998-05-08 | 1999-11-24 | Japan Science & Technology Corp | 短縮型ジストロフィン |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2001083695A2 (en) | 2000-04-28 | 2001-11-08 | Xiao Xiao | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| AU2294102A (en) | 2000-07-14 | 2002-01-30 | Univ Michigan | Mutant muscle-specific enhancers |
| WO2002029056A2 (en) | 2000-10-06 | 2002-04-11 | Regents Of The University Of Michigan | Mini-dystrophin nucleic acid and peptide sequences |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| JP4810062B2 (ja) | 2001-12-17 | 2011-11-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）血清型８の配列 |
| WO2005078139A2 (en) | 2004-02-09 | 2005-08-25 | Thomas Jefferson University | DIAGNOSIS AND TREATMENT OF CANCERS WITH MicroRNA LOCATED IN OR NEAR CANCER-ASSOCIATED CHROMOSOMAL FEATURES |
| US20080044393A1 (en) | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
| WO2007095387A2 (en) | 2006-02-17 | 2007-08-23 | Dharmacon, Inc. | Compositions and methods for inhibiting gene silencing by rna interference |
| 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 |
| CN101883576B (zh) | 2007-07-31 | 2015-08-19 | 得克萨斯系统大学董事会 | 调控纤维化的微小rna家族及其用途 |
| US8236557B2 (en) | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| WO2010071454A1 (en) | 2008-12-17 | 2010-06-24 | Auckland Uniservices Limited | Adeno-associated viral vectors and uses thereof |
| EP2736539B1 (en) | 2011-07-25 | 2017-08-23 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
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| 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 |
| GB201400598D0 (en) | 2014-01-14 | 2014-03-05 | Univ Glasgow | Materials and methods for modulation of tendon healing |
| PL3097197T3 (pl) | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
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| US10119140B2 (en) | 2014-04-18 | 2018-11-06 | Board Of Regents Of The Nevada System Of Higher Education On Behalf Of The University Of Nevada, Reno | Methods for enhancing or decreasing the levels of MIR124 and MIR29 in subjects with muscular dystrophy |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| IL286316B2 (en) * | 2015-01-16 | 2023-03-01 | Univ Washington | New micro-dystrophins and related method 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 |
| EP3350331A4 (en) | 2015-09-17 | 2019-01-23 | Research Institute at Nationwide Children's Hospital | METHOD AND MATERIALS FOR GALGT2 GENE THERAPY |
| 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 |
| CA2971303A1 (en) | 2016-06-21 | 2017-12-21 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| EP3807413B1 (en) | 2018-06-18 | 2025-05-21 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
-
2018
- 2018-03-16 JP JP2020522046A patent/JP7213238B2/ja active Active
- 2018-03-16 WO PCT/US2018/022853 patent/WO2019078916A1/en not_active Ceased
- 2018-03-16 US US16/757,207 patent/US11534501B2/en active Active
- 2018-03-16 EP EP18867394.1A patent/EP3697915A4/en active Pending
- 2018-03-16 MA MA050836A patent/MA50836A/fr unknown
-
2022
- 2022-12-27 US US18/146,840 patent/US20230302157A1/en not_active Abandoned