JP2025516599A - 組換えパルボウイルス生成のための組成物及び方法 - Google Patents

組換えパルボウイルス生成のための組成物及び方法 Download PDF

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Publication number
JP2025516599A
JP2025516599A JP2024566373A JP2024566373A JP2025516599A JP 2025516599 A JP2025516599 A JP 2025516599A JP 2024566373 A JP2024566373 A JP 2024566373A JP 2024566373 A JP2024566373 A JP 2024566373A JP 2025516599 A JP2025516599 A JP 2025516599A
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Prior art keywords
aav
coding region
expression construct
intron
seq
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Pending
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JP2024566373A
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Japanese (ja)
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JPWO2023220502A5 (https=
JP2025516599A5 (https=
Inventor
ワン，チージャオ
ユ，ダオジャン
リウ，ユ
チェン，クイピン
ヤン，ロンツェ
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Aavnergene Inc
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Aavnergene Inc
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Publication of JP2025516599A publication Critical patent/JP2025516599A/ja
Publication of JPWO2023220502A5 publication Critical patent/JPWO2023220502A5/ja
Publication of JP2025516599A5 publication Critical patent/JP2025516599A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
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    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10344—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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    • C12N2750/00011—Details
    • C12N2750/00041—Use of virus, viral particle or viral elements as a vector
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2024566373A 2022-05-12 2023-03-10 組換えパルボウイルス生成のための組成物及び方法 Pending JP2025516599A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263341201P 2022-05-12 2022-05-12
US63/341,201 2022-05-12
PCT/US2023/064152 WO2023220502A1 (en) 2022-05-12 2023-03-10 Compositions and methods for recombinant parvovirus production

Publications (3)

Publication Number Publication Date
JP2025516599A true JP2025516599A (ja) 2025-05-30
JPWO2023220502A5 JPWO2023220502A5 (https=) 2026-02-03
JP2025516599A5 JP2025516599A5 (https=) 2026-02-03

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JP2024566373A Pending JP2025516599A (ja) 2022-05-12 2023-03-10 組換えパルボウイルス生成のための組成物及び方法

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Country Link
US (1) US20230365991A1 (https=)
EP (1) EP4522729A1 (https=)
JP (1) JP2025516599A (https=)
KR (1) KR20250009975A (https=)
CN (1) CN119487183A (https=)
AU (1) AU2023269763A1 (https=)
WO (1) WO2023220502A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024211780A1 (en) * 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025090962A1 (en) * 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
TW202548023A (zh) * 2024-03-14 2025-12-16 美商雅菲納治療公司 用於生產重組腺相關病毒的質體系統

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5945335A (en) * 1995-11-09 1999-08-31 Avigen, Inc. Adenovirus helper-free system for producing recombinant AAV virions lacking oncogenic sequences
US6114148C1 (en) 1996-09-20 2012-05-01 Gen Hospital Corp High level expression of proteins
DE60336736D1 (de) 2002-07-16 2011-05-26 VGX Pharmaceuticals LLC Codon-optimierte synthetische plasmide
US20130095558A1 (en) * 2011-07-19 2013-04-18 Wenlin Huang Process for producing recombinant human endostatin adenovirus
EP3870148A4 (en) * 2018-10-25 2022-11-09 Takeda Pharmaceutical Company Limited AAV TRIPLE PLASMID SYSTEM
EP3722434B1 (en) * 2019-04-12 2022-07-27 Freeline Therapeutics Limited Plasmid system
EP4085143B1 (en) * 2019-12-31 2025-08-13 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)

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US20230365991A1 (en) 2023-11-16
WO2023220502A1 (en) 2023-11-16
CN119487183A (zh) 2025-02-18
EP4522729A1 (en) 2025-03-19
AU2023269763A1 (en) 2024-10-31
KR20250009975A (ko) 2025-01-20

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