JP2021522775A - 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 - Google Patents

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 Download PDF

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JP2021522775A
JP2021522775A JP2019565168A JP2019565168A JP2021522775A JP 2021522775 A JP2021522775 A JP 2021522775A JP 2019565168 A JP2019565168 A JP 2019565168A JP 2019565168 A JP2019565168 A JP 2019565168A JP 2021522775 A JP2021522775 A JP 2021522775A
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aav
serotype
derived
nucleic acid
capsid
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JP2021522775A5 (enExample
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チェンウェン リ
チェンウェン リ
リチャード ジュード サムルスキ
リチャード ジュード サムルスキ
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University of North Carolina at Chapel Hill
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2217/07—Animals genetically altered by homologous recombination
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0381—Animal model for diseases of the hematopoietic system
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material

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  • Animal Behavior & Ethology (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Manufacture Or Reproduction Of Printing Formes (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2019565168A 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 Pending JP2021522775A (ja)

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)

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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

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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

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JP2021522775A true JP2021522775A (ja) 2021-09-02
JP2021522775A5 JP2021522775A5 (enExample) 2021-10-14

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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

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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)

* Cited by examiner, † Cited by third party
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)

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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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CURR. GENE. THER., vol. 5, JPN6022027958, 2005, pages 299 - 310, ISSN: 0005208393 *

Also Published As

Publication number Publication date
NZ759802A (en) 2025-08-29
CN110691846A (zh) 2020-01-14
JP2023154428A (ja) 2023-10-19
IL269184B1 (en) 2025-04-01
WO2019216932A1 (en) 2019-11-14
IL269184B2 (en) 2025-08-01
IL269184A (en) 2019-11-28
CN117535247A (zh) 2024-02-09
EP3596203A4 (en) 2021-12-01
EP3596203A1 (en) 2020-01-22
AU2018422759A1 (en) 2020-01-02

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