JP2012519008A5 - - Google Patents
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- JP2012519008A5 JP2012519008A5 JP2011552364A JP2011552364A JP2012519008A5 JP 2012519008 A5 JP2012519008 A5 JP 2012519008A5 JP 2011552364 A JP2011552364 A JP 2011552364A JP 2011552364 A JP2011552364 A JP 2011552364A JP 2012519008 A5 JP2012519008 A5 JP 2012519008A5
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- JP
- Japan
- Prior art keywords
- seq
- nucleic acid
- parvovirus
- polypeptide
- cds
- 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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- 101000805768 Banna virus (strain Indonesia/JKT-6423/1980) mRNA (guanine-N(7))-methyltransferase Proteins 0.000 claims description 114
- 101000686790 Chaetoceros protobacilladnavirus 2 Replication-associated protein Proteins 0.000 claims description 114
- 101000864475 Chlamydia phage 1 Internal scaffolding protein VP3 Proteins 0.000 claims description 114
- 101000803553 Eumenes pomiformis Venom peptide 3 Proteins 0.000 claims description 114
- 101000583961 Halorubrum pleomorphic virus 1 Matrix protein Proteins 0.000 claims description 114
- 150000007523 nucleic acids Chemical class 0.000 claims description 66
- 239000002245 particle Substances 0.000 claims description 65
- 241000125945 Protoparvovirus Species 0.000 claims description 59
- 238000000034 method Methods 0.000 claims description 53
- 108020004707 nucleic acids Proteins 0.000 claims description 51
- 102000039446 nucleic acids Human genes 0.000 claims description 51
- 230000035772 mutation Effects 0.000 claims description 42
- 239000002773 nucleotide Substances 0.000 claims description 33
- 125000003729 nucleotide group Chemical group 0.000 claims description 33
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 28
- 229920001184 polypeptide Polymers 0.000 claims description 27
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 27
- 108090000623 proteins and genes Proteins 0.000 claims description 21
- 102000004169 proteins and genes Human genes 0.000 claims description 21
- 108091081024 Start codon Proteins 0.000 claims description 19
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 14
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 14
- 108700026244 Open Reading Frames Proteins 0.000 claims description 12
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 11
- 101710081079 Minor spike protein H Proteins 0.000 claims description 11
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 claims description 11
- 101710132601 Capsid protein Proteins 0.000 claims description 10
- 101710197658 Capsid protein VP1 Proteins 0.000 claims description 10
- 101710172711 Structural protein Proteins 0.000 claims description 10
- 101710108545 Viral protein 1 Proteins 0.000 claims description 10
- 239000003795 chemical substances by application Substances 0.000 claims description 10
- 230000037431 insertion Effects 0.000 claims description 10
- 238000003780 insertion Methods 0.000 claims description 10
- 241000700605 Viruses Species 0.000 claims description 8
- 108091026890 Coding region Proteins 0.000 claims description 7
- 230000000295 complement effect Effects 0.000 claims description 7
- 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 claims description 6
- 208000026935 allergic disease Diseases 0.000 claims description 6
- 210000000234 capsid Anatomy 0.000 claims description 6
- 239000002299 complementary DNA Substances 0.000 claims description 6
- 230000014621 translational initiation Effects 0.000 claims description 6
- 229960005486 vaccine Drugs 0.000 claims description 6
- 208000023275 Autoimmune disease Diseases 0.000 claims description 5
- 206010028980 Neoplasm Diseases 0.000 claims description 5
- 208000012902 Nervous system disease Diseases 0.000 claims description 5
- 208000025966 Neurological disease Diseases 0.000 claims description 5
- 238000004519 manufacturing process Methods 0.000 claims description 5
- 208000030159 metabolic disease Diseases 0.000 claims description 5
- 238000013518 transcription Methods 0.000 claims description 5
- 230000035897 transcription Effects 0.000 claims description 5
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 4
- 208000027866 inflammatory disease Diseases 0.000 claims description 4
- 230000002265 prevention Effects 0.000 claims description 4
- 238000011144 upstream manufacturing Methods 0.000 claims description 4
- 208000035473 Communicable disease Diseases 0.000 claims description 3
- 108020004414 DNA Proteins 0.000 claims description 3
- 241000702421 Dependoparvovirus Species 0.000 claims description 3
- 108091034057 RNA (poly(A)) Proteins 0.000 claims description 3
- 230000037430 deletion Effects 0.000 claims description 3
- 238000012217 deletion Methods 0.000 claims description 3
- 239000003446 ligand Substances 0.000 claims description 3
- 239000008177 pharmaceutical agent Substances 0.000 claims description 3
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 3
- 230000037432 silent mutation Effects 0.000 claims description 3
- 238000006467 substitution reaction Methods 0.000 claims description 3
- 238000013519 translation Methods 0.000 claims description 3
- 206010012335 Dependence Diseases 0.000 claims description 2
- 241000684283 Duck parvovirus Species 0.000 claims description 2
- 241001517118 Goose parvovirus Species 0.000 claims description 2
- 241000425549 Snake parvovirus Species 0.000 claims description 2
- 230000001684 chronic effect Effects 0.000 claims description 2
- 108020001507 fusion proteins Proteins 0.000 claims description 2
- 102000037865 fusion proteins Human genes 0.000 claims description 2
- 101710136297 Protein VP2 Proteins 0.000 claims 1
- 239000012634 fragment Substances 0.000 description 19
- 210000004027 cell Anatomy 0.000 description 15
- 101100524317 Adeno-associated virus 2 (isolate Srivastava/1982) Rep40 gene Proteins 0.000 description 8
- 101100524319 Adeno-associated virus 2 (isolate Srivastava/1982) Rep52 gene Proteins 0.000 description 8
- 101100524321 Adeno-associated virus 2 (isolate Srivastava/1982) Rep68 gene Proteins 0.000 description 8
- 101100524324 Adeno-associated virus 2 (isolate Srivastava/1982) Rep78 gene Proteins 0.000 description 8
- 210000003719 b-lymphocyte Anatomy 0.000 description 6
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 3
- 238000001415 gene therapy Methods 0.000 description 3
- 230000014616 translation Effects 0.000 description 3
- 208000024827 Alzheimer disease Diseases 0.000 description 2
- 201000001320 Atherosclerosis Diseases 0.000 description 2
- 241000283690 Bos taurus Species 0.000 description 2
- 108020004705 Codon Proteins 0.000 description 2
- 208000011231 Crohn disease Diseases 0.000 description 2
- 108020005202 Viral DNA Proteins 0.000 description 2
- 239000002671 adjuvant Substances 0.000 description 2
- 208000006673 asthma Diseases 0.000 description 2
- 208000037976 chronic inflammation Diseases 0.000 description 2
- 208000037893 chronic inflammatory disorder Diseases 0.000 description 2
- 201000010099 disease Diseases 0.000 description 2
- 208000016097 disease of metabolism Diseases 0.000 description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 2
- 208000015181 infectious disease Diseases 0.000 description 2
- 206010039073 rheumatoid arthritis Diseases 0.000 description 2
- 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
- 241000271566 Aves Species 0.000 description 1
- 241000282465 Canis Species 0.000 description 1
- 241000283707 Capra Species 0.000 description 1
- 102000053602 DNA Human genes 0.000 description 1
- 206010020751 Hypersensitivity Diseases 0.000 description 1
- 241000701945 Parvoviridae Species 0.000 description 1
- 201000004681 Psoriasis Diseases 0.000 description 1
- 206010039085 Rhinitis allergic Diseases 0.000 description 1
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 1
- -1 VP3 Nucleic acid Chemical class 0.000 description 1
- 108091027569 Z-DNA Proteins 0.000 description 1
- 230000004913 activation Effects 0.000 description 1
- 206010064930 age-related macular degeneration Diseases 0.000 description 1
- 201000010105 allergic rhinitis Diseases 0.000 description 1
- 230000007815 allergy Effects 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 230000001580 bacterial effect Effects 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 230000006058 immune tolerance Effects 0.000 description 1
- 230000001939 inductive effect Effects 0.000 description 1
- 208000002780 macular degeneration Diseases 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 241000894007 species Species 0.000 description 1
- 241000701447 unidentified baculovirus Species 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US15743609P | 2009-03-04 | 2009-03-04 | |
| US61/157,436 | 2009-03-04 | ||
| US30620510P | 2010-02-19 | 2010-02-19 | |
| US61/306,205 | 2010-02-19 | ||
| PCT/EP2010/001343 WO2010099960A2 (en) | 2009-03-04 | 2010-03-04 | Assembly activating protein (aap) and its use for the manufacture of parvovirus particles essential consisting of vp3 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015200962A Division JP6151328B2 (ja) | 2009-03-04 | 2015-10-09 | 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2012519008A JP2012519008A (ja) | 2012-08-23 |
| JP2012519008A5 true JP2012519008A5 (https=) | 2013-04-11 |
Family
ID=42313642
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011552364A Pending JP2012519008A (ja) | 2009-03-04 | 2010-03-04 | 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用 |
| JP2015200962A Expired - Fee Related JP6151328B2 (ja) | 2009-03-04 | 2015-10-09 | 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015200962A Expired - Fee Related JP6151328B2 (ja) | 2009-03-04 | 2015-10-09 | 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用 |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US9464119B2 (https=) |
| EP (1) | EP2403867B1 (https=) |
| JP (2) | JP2012519008A (https=) |
| CN (1) | CN102341406B (https=) |
| BR (1) | BRPI1013364A2 (https=) |
| CA (1) | CA2754335C (https=) |
| DK (1) | DK2403867T3 (https=) |
| ES (1) | ES2742180T3 (https=) |
| MX (2) | MX2011009193A (https=) |
| PL (1) | PL2403867T3 (https=) |
| PT (1) | PT2403867T (https=) |
| WO (1) | WO2010099960A2 (https=) |
Families Citing this family (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2742180T3 (es) | 2009-03-04 | 2020-02-13 | Deutsches Krebsforsch | Proteína activadora del ensamblaje (AAP) y su uso para la fabricación de partículas de parvovirus que consisten esencialmente en VP3 |
| EP2534172A2 (en) * | 2010-02-12 | 2012-12-19 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Compositions and methods for preventing or treating a human parvovirus infection |
| AR082925A1 (es) * | 2010-09-08 | 2013-01-16 | Medigene Ag | Proteinas estructurales mutadas por parvovirus con epitopo de celulas b de proteccion cruzada, producto y metodos relacionados |
| RU2487708C1 (ru) * | 2012-03-12 | 2013-07-20 | Государственное бюджетное образовательное учреждение высшего профессионального образования "Уральская государственная медицинская академия Министерства здравоохранения и социального развития Российской Федерации" (ГБОУ ВПО УГМА Минздравсоцразвития России) | Способ лечения парвовирусной инфекции в19 у детей раннего возраста |
| TWI698240B (zh) | 2012-05-15 | 2020-07-11 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| EP3800191B1 (en) | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| US11021519B2 (en) | 2015-03-02 | 2021-06-01 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
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| CN108603235A (zh) | 2015-07-30 | 2018-09-28 | 马萨诸塞眼科耳科诊所 | 祖先病毒序列及其用途 |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
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| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| US20200231986A1 (en) * | 2017-09-29 | 2020-07-23 | Massachusetts Eye And Ear Infirmary | Production of adeno-associated viruses in insect cells |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US20190202866A1 (en) * | 2017-12-22 | 2019-07-04 | William Marsh Rice University | Viral nanoparticles and methods of use thereof |
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| AU2019227910B2 (en) * | 2018-02-28 | 2025-07-17 | The Regents Of The University Of California | A modular system for gene and protein delivery based on AAV |
| US20210147487A1 (en) * | 2018-04-04 | 2021-05-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for engineered assembly activating proteins (eaaps) |
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| EP3784697A4 (en) * | 2018-04-27 | 2022-07-06 | Spark Therapeutics, Inc. | GMO AAV CAPSIDS WITH INCREASED TROPISM AND AAV VECTORS CONTAINING THE GMO CAPSIDS, AND METHODS FOR THEIR MANUFACTURE AND USE |
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| KR20220019669A (ko) * | 2019-04-12 | 2022-02-17 | 프리라인 테라퓨틱스 리미티드 | 플라스미드 시스템 |
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| ES2742180T3 (es) | 2009-03-04 | 2020-02-13 | Deutsches Krebsforsch | Proteína activadora del ensamblaje (AAP) y su uso para la fabricación de partículas de parvovirus que consisten esencialmente en VP3 |
-
2010
- 2010-03-04 ES ES10708716T patent/ES2742180T3/es active Active
- 2010-03-04 CA CA2754335A patent/CA2754335C/en active Active
- 2010-03-04 MX MX2011009193A patent/MX2011009193A/es active IP Right Grant
- 2010-03-04 US US13/203,442 patent/US9464119B2/en active Active
- 2010-03-04 DK DK10708716.5T patent/DK2403867T3/da active
- 2010-03-04 EP EP10708716.5A patent/EP2403867B1/en active Active
- 2010-03-04 JP JP2011552364A patent/JP2012519008A/ja active Pending
- 2010-03-04 PL PL10708716T patent/PL2403867T3/pl unknown
- 2010-03-04 BR BRPI1013364A patent/BRPI1013364A2/pt not_active Application Discontinuation
- 2010-03-04 MX MX2014014365A patent/MX345729B/es unknown
- 2010-03-04 WO PCT/EP2010/001343 patent/WO2010099960A2/en not_active Ceased
- 2010-03-04 PT PT10708716T patent/PT2403867T/pt unknown
- 2010-03-04 CN CN201080010540.8A patent/CN102341406B/zh active Active
-
2015
- 2015-10-09 JP JP2015200962A patent/JP6151328B2/ja not_active Expired - Fee Related
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2016
- 2016-09-12 US US15/263,072 patent/US10344057B2/en active Active
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2019
- 2019-05-17 US US16/415,676 patent/US11267847B2/en not_active Expired - Fee Related
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