JP2021522775A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2021522775A5 JP2021522775A5 JP2019565168A JP2019565168A JP2021522775A5 JP 2021522775 A5 JP2021522775 A5 JP 2021522775A5 JP 2019565168 A JP2019565168 A JP 2019565168A JP 2019565168 A JP2019565168 A JP 2019565168A JP 2021522775 A5 JP2021522775 A5 JP 2021522775A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- nucleic acid
- serotype
- derived
- virions
- 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.)
- Pending
Links
- 210000002845 virion Anatomy 0.000 claims 44
- 150000007523 nucleic acids Chemical group 0.000 claims 42
- 101710081079 Minor spike protein H Proteins 0.000 claims 28
- 108010087302 Viral Structural Proteins Proteins 0.000 claims 26
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 21
- 108020004707 nucleic acids Proteins 0.000 claims 21
- 102000039446 nucleic acids Human genes 0.000 claims 21
- 238000000034 method Methods 0.000 claims 16
- 230000035772 mutation Effects 0.000 claims 13
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 11
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 8
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 8
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 6
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 6
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 6
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 6
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 6
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 6
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 6
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 6
- 108020005067 RNA Splice Sites Proteins 0.000 claims 6
- 108091081024 Start codon Proteins 0.000 claims 6
- 108020004705 Codon Proteins 0.000 claims 4
- 108020004414 DNA Proteins 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 208000011580 syndromic disease Diseases 0.000 claims 3
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 2
- 201000002883 Scheie syndrome Diseases 0.000 claims 2
- 102000005262 Sulfatase Human genes 0.000 claims 2
- 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 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 210000002966 serum Anatomy 0.000 claims 2
- 108060007951 sulfatase Proteins 0.000 claims 2
- 102100031491 Arylsulfatase B Human genes 0.000 claims 1
- 101000588395 Bacillus subtilis (strain 168) Beta-hexosaminidase Proteins 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 102000004547 Glucosylceramidase Human genes 0.000 claims 1
- 108010017544 Glucosylceramidase Proteins 0.000 claims 1
- 229920002527 Glycogen Polymers 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 1
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 1
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 claims 1
- 108010027520 N-Acetylgalactosamine-4-Sulfatase Proteins 0.000 claims 1
- 102100023282 N-acetylglucosamine-6-sulfatase Human genes 0.000 claims 1
- 108010023320 N-acetylglucosamine-6-sulfatase Proteins 0.000 claims 1
- 101710172711 Structural protein Proteins 0.000 claims 1
- 108010067390 Viral Proteins Proteins 0.000 claims 1
- 238000009825 accumulation Methods 0.000 claims 1
- 102000005421 acetyltransferase Human genes 0.000 claims 1
- 108020002494 acetyltransferase Proteins 0.000 claims 1
- 230000002378 acidificating effect Effects 0.000 claims 1
- 230000015572 biosynthetic process Effects 0.000 claims 1
- 229940096919 glycogen Drugs 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 230000002132 lysosomal effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 1
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 claims 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 claims 1
- 210000000605 viral structure Anatomy 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023125955A JP2023154428A (ja) | 2018-05-07 | 2023-08-02 | 合理的な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製造法および使用法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862668056P | 2018-05-07 | 2018-05-07 | |
| US62/668,056 | 2018-05-07 | ||
| US201862678675P | 2018-05-31 | 2018-05-31 | |
| US62/678,675 | 2018-05-31 | ||
| PCT/US2018/044632 WO2019216932A1 (en) | 2018-05-07 | 2018-07-31 | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023125955A Division JP2023154428A (ja) | 2018-05-07 | 2023-08-02 | 合理的な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製造法および使用法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021522775A JP2021522775A (ja) | 2021-09-02 |
| JP2021522775A5 true JP2021522775A5 (enExample) | 2021-10-14 |
Family
ID=68468205
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019565168A Pending JP2021522775A (ja) | 2018-05-07 | 2018-07-31 | 合理的な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製造法および使用法 |
| JP2023125955A Pending JP2023154428A (ja) | 2018-05-07 | 2023-08-02 | 合理的な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製造法および使用法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023125955A Pending JP2023154428A (ja) | 2018-05-07 | 2023-08-02 | 合理的な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製造法および使用法 |
Country Status (7)
| Country | Link |
|---|---|
| EP (1) | EP3596203A4 (enExample) |
| JP (2) | JP2021522775A (enExample) |
| CN (2) | CN117535247A (enExample) |
| AU (1) | AU2018422759A1 (enExample) |
| IL (1) | IL269184B2 (enExample) |
| NZ (1) | NZ759802A (enExample) |
| WO (1) | WO2019216932A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110684800B (zh) * | 2018-11-02 | 2020-11-03 | 深圳益世康宁生物科技有限公司 | 一种携带肿瘤-睾丸抗原10基因的重组腺相关病毒载体及其应用价值 |
| US20230002786A1 (en) * | 2020-01-07 | 2023-01-05 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats and methods of their use as promoters |
| 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 |
| AU2022286647A1 (en) * | 2021-06-02 | 2023-11-30 | Uniqure Biopharma B.V. | Insect cell production of parvoviral vectors with modified capsid proteins |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ536274A (en) * | 2002-05-01 | 2008-04-30 | Univ Florida | Improved rAAV expression systems for genetic modification of specific capsid proteins |
| CA2500523A1 (en) * | 2003-05-01 | 2004-11-18 | University Of Florida Research Foundation, Inc. | Vp2-modified raav vector compositions and uses therefor |
| AU2004268375B2 (en) * | 2003-09-02 | 2010-01-28 | Glaxosmithkline Biologicals S.A. | Vaccine |
| CN101511373B (zh) * | 2006-06-19 | 2015-12-09 | 阿斯克肋匹奥生物制药公司 | 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体 |
| WO2012064960A2 (en) * | 2010-11-10 | 2012-05-18 | Fred Hutchinson Cancer Research Center | Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination |
| 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 |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| MX2016001044A (es) * | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| CN110770346B (zh) * | 2017-03-15 | 2024-01-12 | 北卡罗来纳-查佩尔山大学 | 多倍体腺相关病毒载体及其制备和使用方法 |
-
2018
- 2018-07-31 NZ NZ759802A patent/NZ759802A/en unknown
- 2018-07-31 CN CN202311045266.3A patent/CN117535247A/zh active Pending
- 2018-07-31 AU AU2018422759A patent/AU2018422759A1/en active Pending
- 2018-07-31 JP JP2019565168A patent/JP2021522775A/ja active Pending
- 2018-07-31 WO PCT/US2018/044632 patent/WO2019216932A1/en not_active Ceased
- 2018-07-31 IL IL269184A patent/IL269184B2/en unknown
- 2018-07-31 EP EP18907477.6A patent/EP3596203A4/en active Pending
- 2018-07-31 CN CN201880032343.2A patent/CN110691846A/zh active Pending
-
2023
- 2023-08-02 JP JP2023125955A patent/JP2023154428A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP6366581B2 (ja) | 改変されたアデノ随伴ウイルスベクター組成物 | |
| JP2020511127A5 (enExample) | ||
| Bantel-Schaal et al. | Human adeno-associated virus type 5 is only distantly related to other known primate helper-dependent parvoviruses | |
| Zinn et al. | Adeno-associated virus: fit to serve | |
| JP7393766B2 (ja) | 抗体回避ウイルスベクターのための方法および組成物 | |
| US11015189B2 (en) | AAV capsid proteins for nucleic acid transfer | |
| Bossis et al. | Cloning of an avian adeno-associated virus (AAAV) and generation of recombinant AAAV particles | |
| JP2022190081A5 (enExample) | ||
| EP1135468B1 (en) | Virus vectors and methods of making and administering the same | |
| JP2021003120A5 (enExample) | ||
| JP2021522775A5 (enExample) | ||
| JP2019116492A5 (enExample) | ||
| JP2018522529A5 (enExample) | ||
| JP2017509632A5 (enExample) | ||
| US11780886B2 (en) | Modified viral vectors and methods of making and using the same | |
| WO2025071848A1 (en) | Liver detargeted capsids | |
| Youjin et al. | The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapy | |
| CN120787264A (zh) | 肝脏去靶向衣壳 | |
| CN120882875A (zh) | 肌肉靶向衣壳 | |
| JPWO2021076911A5 (enExample) | ||
| JPWO2021195218A5 (enExample) | ||
| Hildinger | Content Alerts | |
| NZ738841B2 (en) | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues | |
| NZ738841A (en) | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues | |
| HK1236040B (en) | New avv capsid proteins for nucleic acid transfer |