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 (enExample) | 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 (enExample) |
| EP (1) | EP3697915A4 (enExample) |
| JP (1) | JP7213238B2 (enExample) |
| MA (1) | MA50836A (enExample) |
| WO (1) | WO2019078916A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3377042A4 (en) | 2015-11-16 | 2019-05-29 | The Research Institute at Nationwide Children's Hospital | MATERIALS AND METHOD FOR THE TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINOPATHIES |
| 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 |
| IL275880B2 (en) | 2018-01-31 | 2025-10-01 | Res Inst Nationwide Childrens Hospital | Gene therapy for brachioradial muscular dystrophy type 2C |
| US12491265B2 (en) | 2018-06-18 | 2025-12-09 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| SG11202012715UA (en) | 2018-06-29 | 2021-01-28 | Res Inst Nationwide Childrens Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| JP2022521776A (ja) | 2019-02-26 | 2022-04-12 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
| MY210230A (en) | 2019-08-21 | 2025-09-04 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| CN115485291A (zh) | 2020-04-29 | 2022-12-16 | 百时美施贵宝公司 | 具血影蛋白融合结构域的微型化抗肌萎缩蛋白及其用途 |
| TW202208630A (zh) * | 2020-06-15 | 2022-03-01 | 美國全美兒童醫院之研究學會 | 針對肌肉營養不良症的腺相關病毒載體遞送 |
| 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| AU688428B2 (en) | 1993-11-09 | 1998-03-12 | Johns Hopkins University, The | Generation of high titers of recombinant AAV vectors |
| DE69433922T2 (de) | 1993-11-09 | 2005-07-28 | Medical College Of Ohio, Toledo | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| 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 |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | 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 |
| AU715543B2 (en) | 1995-09-08 | 2000-02-03 | 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 |
| IL128779A0 (en) | 1996-09-06 | 2000-01-31 | Univ Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| DK1944362T3 (en) | 1997-09-05 | 2016-01-25 | Genzyme Corp | Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released 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 |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| CN100422320C (zh) | 2000-04-28 | 2008-10-01 | 肖啸 | 编码抗肌萎缩蛋白小基因的dna序列及其使用方法 |
| ATE373095T1 (de) | 2000-07-14 | 2007-09-15 | Univ Michigan | Mutierte muskelspezifische enhancer |
| AU2001296600A1 (en) | 2000-10-06 | 2002-04-15 | Regents Of The University Of Michigan | Mini-dystrophin nucleic acid and peptide sequences |
| US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital 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 |
| EP2295604B1 (en) | 2004-02-09 | 2015-04-08 | 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 |
| US20100184209A1 (en) | 2006-02-17 | 2010-07-22 | Dharmacon, Inc. | Compositions and methods for inhibiting gene silencing by rna interference |
| WO2008088895A2 (en) | 2007-01-18 | 2008-07-24 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma |
| MX2010001217A (es) | 2007-07-31 | 2010-04-07 | Regents The Unuversity Of Texa | Familia de micro-arn que modula la fibrosis y usos de la misma. |
| 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 |
| CA3106285A1 (en) | 2011-07-25 | 2013-01-31 | 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 |
| WO2013102904A1 (en) | 2012-01-05 | 2013-07-11 | Hadasit Medical Research Services & Development Ltd. | Methods and compositions for gene delivery |
| 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 |
| US11072801B2 (en) | 2014-01-21 | 2021-07-27 | Vrije Universiteit Brussel | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| WO2015157306A1 (en) | 2014-04-09 | 2015-10-15 | University Of Houston | Therapeutic mirnas for treating heart and skeletal muscle diseases |
| 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 |
| EP4599851A2 (en) | 2015-01-16 | 2025-08-13 | 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 |
| AU2016323575B2 (en) | 2015-09-17 | 2022-10-27 | Research Institute At Nationwide Children's Hospital | Methods 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 |
| US12491265B2 (en) | 2018-06-18 | 2025-12-09 | 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 EP EP18867394.1A patent/EP3697915A4/en active Pending
- 2018-03-16 WO PCT/US2018/022853 patent/WO2019078916A1/en not_active Ceased
- 2018-03-16 MA MA050836A patent/MA50836A/fr unknown
- 2018-03-16 JP JP2020522046A patent/JP7213238B2/ja active Active
- 2018-03-16 US US16/757,207 patent/US11534501B2/en active Active
-
2022
- 2022-12-27 US US18/146,840 patent/US20230302157A1/en not_active Abandoned