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
Links
- 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 (https=) | 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 (9)
| Country | Link |
|---|---|
| EP (3) | EP3610023B1 (https=) |
| JP (1) | JP7132934B2 (https=) |
| CN (2) | CN110770346B (https=) |
| AU (2) | AU2018234695B2 (https=) |
| CA (1) | CA3054711A1 (https=) |
| ES (1) | ES2966692T3 (https=) |
| IL (3) | IL316259A (https=) |
| NZ (1) | NZ756587A (https=) |
| WO (1) | WO2018170310A1 (https=) |
Families Citing this family (25)
| 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 |
| BR112018003665A2 (en) | 2015-09-28 | 2018-09-25 | The University Of North Carolina At Chapel Hill | methods and compositions for antibody evasion viral vectors |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | 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 |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| 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 |
| WO2019216932A1 (en) * | 2018-05-07 | 2019-11-14 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| US20200069819A1 (en) * | 2018-05-14 | 2020-03-05 | Biomarin Pharmaceutical Inc. | Stable expression of aav vectors in juvenile subjects |
| MX2021005727A (es) * | 2018-11-16 | 2021-09-10 | Spark Therapeutics Inc | Ensayo in vitro para detectar potenciadores e inhibidores de la transducción de vectores virales adenoasociados (aav) y/o para detectar o cuantificar anticuerpos de unión anti-aav. |
| CN113395975A (zh) * | 2018-11-20 | 2021-09-14 | 宾夕法尼亚州大学信托人 | 用于靶向血脑屏障的组合物和方法 |
| AU2019418750A1 (en) * | 2019-01-02 | 2021-07-22 | Intima Bioscience, Inc. | Modified adeno-associated viral vectors for use in genetic engineering |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| EP3955970A1 (en) * | 2019-04-15 | 2022-02-23 | University Of Iowa Research Foundation | Methods and compositions for transgene expression |
| PE20220014A1 (es) * | 2019-04-29 | 2022-01-11 | Univ Pennsylvania | Nuevas capsides de aav y composiciones que las contienen |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| 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 |
| AR122013A1 (es) * | 2020-05-05 | 2022-08-03 | Univ Duke | Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso |
| CN116121274B (zh) * | 2020-07-29 | 2024-11-19 | 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 |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| JP2024515626A (ja) * | 2021-04-16 | 2024-04-10 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドａａｖビリオン |
| CA3242989A1 (en) * | 2021-12-21 | 2023-06-29 | Research Institute At Nationwide Children's Hospital | MATERIALS AND METHODS FOR THE TREATMENT OF MUSCULAR BELLY DYSTROPHY |
| EP4703468A1 (en) * | 2024-08-29 | 2026-03-04 | Sartorius Albumedix Limited | Methods and uses |
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| AU7966987A (en) | 1986-09-08 | 1988-04-07 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
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| CA2373110A1 (en) * | 2000-03-14 | 2001-09-20 | Neurologix, Inc. | Production of chimeric capsid vectors |
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| WO2008027084A2 (en) * | 2006-04-28 | 2008-03-06 | 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 |
| US20090215879A1 (en) | 2008-02-26 | 2009-08-27 | University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| US20120093775A1 (en) * | 2009-03-27 | 2012-04-19 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
| ES2685611T3 (es) * | 2011-02-14 | 2018-10-10 | The Children's Hospital Of Philadelphia | Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo |
| US9441206B2 (en) | 2011-10-28 | 2016-09-13 | The University Of North Carolina At Chapel Hill | Cell line for production of adeno-associated virus |
| AU2013249202B2 (en) | 2012-04-18 | 2018-08-09 | The Children's Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using AAV capsid variants |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| KR20240090694A (ko) * | 2013-07-22 | 2024-06-21 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| WO2016210170A1 (en) * | 2015-06-23 | 2016-12-29 | The Children's Hospital Of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
-
2018
- 2018-03-15 CN CN201880031964.9A patent/CN110770346B/zh active Active
- 2018-03-15 IL IL316259A patent/IL316259A/en unknown
- 2018-03-15 EP EP18768025.1A patent/EP3610023B1/en active Active
- 2018-03-15 CA CA3054711A patent/CA3054711A1/en active Pending
- 2018-03-15 ES ES18768025T patent/ES2966692T3/es active Active
- 2018-03-15 NZ NZ756587A patent/NZ756587A/en unknown
- 2018-03-15 JP JP2019545960A patent/JP7132934B2/ja active Active
- 2018-03-15 CN CN202311805114.9A patent/CN117801075A/zh active Pending
- 2018-03-15 EP EP25211519.1A patent/EP4707292A2/en active Pending
- 2018-03-15 IL IL268891A patent/IL268891B2/en unknown
- 2018-03-15 WO PCT/US2018/022725 patent/WO2018170310A1/en not_active Ceased
- 2018-03-15 EP EP23195775.4A patent/EP4303225B1/en active Active
- 2018-03-15 AU AU2018234695A patent/AU2018234695B2/en active Active
- 2018-03-15 IL IL312266A patent/IL312266B2/en unknown
-
2024
- 2024-10-14 AU AU2024227303A patent/AU2024227303A1/en active Pending
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