JP2020511127A5 - - Google Patents
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- Publication number
- JP2020511127A5 JP2020511127A5 JP2019545960A JP2019545960A JP2020511127A5 JP 2020511127 A5 JP2020511127 A5 JP 2020511127A5 JP 2019545960 A JP2019545960 A JP 2019545960A JP 2019545960 A JP2019545960 A JP 2019545960A JP 2020511127 A5 JP2020511127 A5 JP 2020511127A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- capsid
- protein
- aav8
- aav2
- 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
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- 210000000234 capsid Anatomy 0.000 claims description 66
- 108090000623 proteins and genes Proteins 0.000 claims description 38
- 101000686790 Chaetoceros protobacilladnavirus 2 Replication-associated protein Proteins 0.000 claims description 30
- 101710108545 Viral protein 1 Proteins 0.000 claims description 29
- 102000004169 proteins and genes Human genes 0.000 claims description 26
- 108090000565 Capsid Proteins Proteins 0.000 claims description 24
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 24
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 22
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 22
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 19
- 239000013612 plasmid Substances 0.000 claims description 17
- 241000702421 Dependoparvovirus Species 0.000 claims description 14
- 238000000034 method Methods 0.000 claims description 14
- 239000002245 particle Substances 0.000 claims description 14
- 201000010099 disease Diseases 0.000 claims description 13
- 150000007523 nucleic acids Chemical group 0.000 claims description 10
- 208000035475 disorder Diseases 0.000 claims description 9
- 108020004707 nucleic acids Proteins 0.000 claims description 6
- 102000039446 nucleic acids Human genes 0.000 claims description 6
- 238000004519 manufacturing process Methods 0.000 claims description 5
- 239000013598 vector Substances 0.000 claims description 5
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 3
- 241000701447 unidentified baculovirus Species 0.000 claims description 3
- 241001529453 unidentified herpesvirus Species 0.000 claims description 3
- 238000004806 packaging method and process Methods 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 101000714457 Chaetoceros protobacilladnavirus 2 Capsid protein Proteins 0.000 claims 11
- 210000004027 cell Anatomy 0.000 claims 6
- 239000013607 AAV vector Substances 0.000 claims 5
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 4
- 210000002845 virion Anatomy 0.000 claims 4
- 241000700605 Viruses Species 0.000 claims 3
- 210000004436 artificial bacterial chromosome Anatomy 0.000 claims 2
- 210000001106 artificial yeast chromosome Anatomy 0.000 claims 2
- 241000710929 Alphavirus Species 0.000 claims 1
- 206010046865 Vaccinia virus infection Diseases 0.000 claims 1
- 108010087302 Viral Structural Proteins Proteins 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 208000007089 vaccinia Diseases 0.000 claims 1
- 101710197658 Capsid protein VP1 Proteins 0.000 description 26
- 101000805768 Banna virus (strain Indonesia/JKT-6423/1980) mRNA (guanine-N(7))-methyltransferase Proteins 0.000 description 18
- 101000864475 Chlamydia phage 1 Internal scaffolding protein VP3 Proteins 0.000 description 18
- 101000803553 Eumenes pomiformis Venom peptide 3 Proteins 0.000 description 18
- 101000583961 Halorubrum pleomorphic virus 1 Matrix protein Proteins 0.000 description 18
- 101710132601 Capsid protein Proteins 0.000 description 16
- 101710081079 Minor spike protein H Proteins 0.000 description 16
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 description 16
- 101710197665 Capsid protein VP2 Proteins 0.000 description 10
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 description 8
- 201000002883 Scheie syndrome Diseases 0.000 description 8
- ZSLZBFCDCINBPY-ZSJPKINUSA-N acetyl-CoA Chemical compound O[C@@H]1[C@H](OP(O)(O)=O)[C@@H](COP(O)(=O)OP(O)(=O)OCC(C)(C)[C@@H](O)C(=O)NCCC(=O)NCCSC(=O)C)O[C@H]1N1C2=NC=NC(N)=C2N=C1 ZSLZBFCDCINBPY-ZSJPKINUSA-N 0.000 description 8
- 210000002966 serum Anatomy 0.000 description 8
- 208000011580 syndromic disease Diseases 0.000 description 8
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 5
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 5
- 101800001319 Capsid protein VP3 Proteins 0.000 description 5
- 208000024720 Fabry Disease Diseases 0.000 description 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 4
- 241000649045 Adeno-associated virus 10 Species 0.000 description 4
- 102100031491 Arylsulfatase B Human genes 0.000 description 4
- 101000588395 Bacillus subtilis (strain 168) Beta-hexosaminidase Proteins 0.000 description 4
- 108091026890 Coding region Proteins 0.000 description 4
- 102000004547 Glucosylceramidase Human genes 0.000 description 4
- 108010017544 Glucosylceramidase Proteins 0.000 description 4
- 229920002527 Glycogen Polymers 0.000 description 4
- 229920002683 Glycosaminoglycan Polymers 0.000 description 4
- 208000015439 Lysosomal storage disease Diseases 0.000 description 4
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 description 4
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 description 4
- 108010027520 N-Acetylgalactosamine-4-Sulfatase Proteins 0.000 description 4
- 108010023320 N-acetylglucosamine-6-sulfatase Proteins 0.000 description 4
- 102000056067 N-acetylglucosamine-6-sulfatases Human genes 0.000 description 4
- 201000001828 Sly syndrome Diseases 0.000 description 4
- 102000005262 Sulfatase Human genes 0.000 description 4
- 102000005421 acetyltransferase Human genes 0.000 description 4
- 108020002494 acetyltransferase Proteins 0.000 description 4
- 239000002253 acid Substances 0.000 description 4
- 230000002378 acidificating effect Effects 0.000 description 4
- 229930182830 galactose Natural products 0.000 description 4
- 229940096919 glycogen Drugs 0.000 description 4
- 230000002132 lysosomal effect Effects 0.000 description 4
- 201000002273 mucopolysaccharidosis II Diseases 0.000 description 4
- 208000005340 mucopolysaccharidosis III Diseases 0.000 description 4
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 description 4
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 description 4
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 description 4
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 description 4
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 description 4
- 108060007951 sulfatase Proteins 0.000 description 4
- 206010053185 Glycogen storage disease type II Diseases 0.000 description 3
- 201000004502 glycogen storage disease II Diseases 0.000 description 3
- 241001180141 Adeno-associated virus-Go.1 Species 0.000 description 1
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 description 1
- 208000012770 hereditary angioedema type 1 Diseases 0.000 description 1
- 208000012737 hereditary angioedema type 2 Diseases 0.000 description 1
- 238000003780 insertion Methods 0.000 description 1
- 230000037431 insertion Effects 0.000 description 1
- 230000003252 repetitive effect Effects 0.000 description 1
- 238000001890 transfection Methods 0.000 description 1
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762471762P | 2017-03-15 | 2017-03-15 | |
| US62/471,762 | 2017-03-15 | ||
| US201762520901P | 2017-06-16 | 2017-06-16 | |
| US62/520,901 | 2017-06-16 | ||
| US201862630558P | 2018-02-14 | 2018-02-14 | |
| US62/630,558 | 2018-02-14 | ||
| PCT/US2018/022725 WO2018170310A1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020511127A JP2020511127A (ja) | 2020-04-16 |
| JP2020511127A5 true JP2020511127A5 (enExample) | 2021-04-30 |
| JP7132934B2 JP7132934B2 (ja) | 2022-09-07 |
Family
ID=63522576
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019545960A Active JP7132934B2 (ja) | 2017-03-15 | 2018-03-15 | ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法 |
Country Status (8)
| Country | Link |
|---|---|
| EP (2) | EP4303225B1 (enExample) |
| JP (1) | JP7132934B2 (enExample) |
| CN (2) | CN110770346B (enExample) |
| AU (2) | AU2018234695B2 (enExample) |
| CA (1) | CA3054711A1 (enExample) |
| ES (1) | ES2966692T3 (enExample) |
| IL (3) | IL316259A (enExample) |
| WO (1) | WO2018170310A1 (enExample) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| WO2017205739A1 (en) | 2016-05-26 | 2017-11-30 | University Of Iowa Research Foundation | cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1 |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| WO2019006418A2 (en) | 2017-06-30 | 2019-01-03 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| CN110691846A (zh) * | 2018-05-07 | 2020-01-14 | 北卡罗来纳-查佩尔山大学 | 合理的多倍体腺相关病毒载体及其制造和使用方法 |
| EP3794112A1 (en) * | 2018-05-14 | 2021-03-24 | BioMarin Pharmaceutical Inc. | Stable expression of aav vectors in juvenile subjects |
| WO2020102753A1 (en) * | 2018-11-16 | 2020-05-22 | Spark Therapeutics, Inc. | In vitro assay for detecting enhancers and inhibitors of adeno associated virus (aav) vector transduction and/or detecting or quantitating anti-aav binding antibodies |
| US20210393713A1 (en) * | 2018-11-20 | 2021-12-23 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for targeting the blood-brain barrier |
| AU2019418750A1 (en) * | 2019-01-02 | 2021-07-22 | Intima Bioscience, Inc. | Modified adeno-associated viral vectors for use in genetic engineering |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| SG11202111353QA (en) * | 2019-04-15 | 2021-11-29 | Univ Iowa Res Found | Methods and compositions for transgene expression |
| PH12021552608A1 (en) * | 2019-04-29 | 2022-06-20 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
| MX2022013963A (es) * | 2020-05-05 | 2023-02-22 | Univ Duke | Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas. |
| CN116121275B (zh) * | 2020-07-29 | 2024-11-29 | 北京三诺佳邑生物技术有限责任公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| WO2022221529A1 (en) * | 2021-04-16 | 2022-10-20 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
| CA3242989A1 (en) * | 2021-12-21 | 2023-06-29 | Res Inst Nationwide Childrens Hospital | Materials and methods for the treatment of limb girdle muscular dystrophy |
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| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| JPH01503675A (ja) | 1986-09-08 | 1989-12-14 | アプライド・バイオテクノロジー・インコーポレーテツド | 中空ウイルスキヤプシドのワクチン |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| EP0442926A1 (en) | 1988-11-10 | 1991-08-28 | Imperial Cancer Research Technology Limited | Polypeptides |
| US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
| US6204059B1 (en) * | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| JP4002605B2 (ja) | 1995-06-07 | 2007-11-07 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 筋組織のａａｖ形質導入 |
| EP0883344B2 (en) | 1995-12-15 | 2010-06-09 | VIRxSYS Corporation | Therapeutic molecules generated by trans-splicing |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| ES2313784T3 (es) | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| NZ511171A (en) | 1998-09-22 | 2004-02-27 | Univ Florida | Methods for large-scale production of recombinant AAV vectors |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| EP1228234A2 (en) * | 2000-03-14 | 2002-08-07 | Neurologix, Inc. | Production of chimeric capsid vectors |
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| DK1496944T3 (da) * | 2002-05-01 | 2008-12-01 | Univ Florida | Forbedrede rAAV-ekspressionssystemer til genetisk modificering af specifikke sapsid-proteiner |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
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| WO2006029319A2 (en) | 2004-09-09 | 2006-03-16 | The General Hospital Corporation | Modulating phosphatase activity in cardiac cells |
| CA2591544A1 (en) | 2004-12-15 | 2006-06-22 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| CN101213203A (zh) | 2005-04-29 | 2008-07-02 | 教堂山北卡罗莱纳州大学 | 在转录后水平调节核酸表达的方法和组合物 |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| EP1994043A4 (en) | 2006-02-10 | 2009-05-20 | Univ Cincinnati | PHOSPHATASE INHIBITOR PROTEIN 1 AS A REGULATOR OF HEART FUNCTION |
| US20090317417A1 (en) | 2006-04-28 | 2009-12-24 | The Trustees Of The University Of Pennsylvania | Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof |
| 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 |
| WO2009108274A2 (en) | 2008-02-26 | 2009-09-03 | The University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| MX2011010097A (es) | 2009-03-27 | 2011-10-19 | Proyecto Biomedicina Cima Sl | Metodos y composiciones para el tratamiento de cirrosis y fibrosis hepatica. |
| CA2827375C (en) * | 2011-02-14 | 2022-07-19 | The Children's Hospital Of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| JP6165752B2 (ja) | 2011-10-28 | 2017-07-19 | ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | アデノ随伴ウイルスの産生のための細胞株 |
| SI2839014T1 (sl) | 2012-04-18 | 2021-05-31 | The Children's Hospital Of Philadelphia | Sestavek in postopki za zelo učinkovit prenos genov z uporabo variant kapside AAV-JA |
| US9163259B2 (en) | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| HUE047996T2 (hu) * | 2013-07-22 | 2020-05-28 | Childrens Hospital Philadelphia | AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
| PT3313991T (pt) * | 2015-06-23 | 2024-09-27 | Childrens Hospital Philadelphia | Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos |
-
2018
- 2018-03-15 EP EP23195775.4A patent/EP4303225B1/en active Active
- 2018-03-15 IL IL316259A patent/IL316259A/en unknown
- 2018-03-15 CN CN201880031964.9A patent/CN110770346B/zh active Active
- 2018-03-15 CA CA3054711A patent/CA3054711A1/en active Pending
- 2018-03-15 JP JP2019545960A patent/JP7132934B2/ja active Active
- 2018-03-15 WO PCT/US2018/022725 patent/WO2018170310A1/en not_active Ceased
- 2018-03-15 AU AU2018234695A patent/AU2018234695B2/en active Active
- 2018-03-15 ES ES18768025T patent/ES2966692T3/es active Active
- 2018-03-15 IL IL312266A patent/IL312266B2/en unknown
- 2018-03-15 CN CN202311805114.9A patent/CN117801075A/zh active Pending
- 2018-03-15 EP EP18768025.1A patent/EP3610023B1/en active Active
- 2018-03-15 IL IL268891A patent/IL268891B2/en unknown
-
2024
- 2024-10-14 AU AU2024227303A patent/AU2024227303A1/en active Pending
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