CN115551878A - 用于增强的基因疗法的腺相关病毒(aav)的受控修饰 - Google Patents

用于增强的基因疗法的腺相关病毒(aav)的受控修饰 Download PDF

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Publication number
CN115551878A
CN115551878A CN202180032267.7A CN202180032267A CN115551878A CN 115551878 A CN115551878 A CN 115551878A CN 202180032267 A CN202180032267 A CN 202180032267A CN 115551878 A CN115551878 A CN 115551878A
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aav
amino acid
genetically modified
variant
capsid protein
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Chinese (zh)
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阿布舍克·查特吉
雷切尔·E·科莱蒙
萨拉·B·埃里克森
范群
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Boston College
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Boston College
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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
    • 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
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    • 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
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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
    • C12N2750/14122—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
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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
    • C12N2750/14141—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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C12N2750/14151—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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202180032267.7A 2020-05-01 2021-05-03 用于增强的基因疗法的腺相关病毒(aav)的受控修饰 Pending CN115551878A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063018573P 2020-05-01 2020-05-01
US63/018,573 2020-05-01
PCT/US2021/030489 WO2021222899A1 (en) 2020-05-01 2021-05-03 Controlled modification of adeno-associated virus (aav) for enhanced gene therapy

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Publication Number Publication Date
CN115551878A true CN115551878A (zh) 2022-12-30

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CN202180032267.7A Pending CN115551878A (zh) 2020-05-01 2021-05-03 用于增强的基因疗法的腺相关病毒(aav)的受控修饰

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US (1) US20230175013A1 (https=)
EP (1) EP4143206A1 (https=)
JP (2) JP2023524401A (https=)
KR (1) KR20230005941A (https=)
CN (1) CN115551878A (https=)
AU (1) AU2021264081A1 (https=)
CA (1) CA3177573A1 (https=)
IL (1) IL297780A (https=)
WO (1) WO2021222899A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025188967A1 (en) 2024-03-06 2025-09-12 Trustees Of Boston College Precise adeno-associated virus-protein conjugates and uses thereof
WO2025230000A1 (ja) * 2024-05-01 2025-11-06 Ｊｃｒファーマ株式会社 修飾されたａａｖ粒子、その製造方法、関連する医薬及びその製造
WO2026037731A1 (en) 2024-08-12 2026-02-19 Coave Therapeutics Squaramide-modified viral vectors

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1300222A (zh) * 1998-02-12 2001-06-20 免疫合成物公司 策略性修饰的乙肝核心蛋白及其衍生物
CN104592364A (zh) * 2013-10-30 2015-05-06 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
CN107532177A (zh) * 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI1633772T1 (sl) * 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
WO2010093784A2 (en) * 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
EP3813845A4 (en) * 2018-05-15 2022-10-26 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1300222A (zh) * 1998-02-12 2001-06-20 免疫合成物公司 策略性修饰的乙肝核心蛋白及其衍生物
CN104592364A (zh) * 2013-10-30 2015-05-06 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
US20160297855A1 (en) * 2013-10-30 2016-10-13 Peking University Adeno-Associated Virus with Site-Directed Mutagenesis and Site-Directed Modification and Preparation Method and Application Thereof
CN107532177A (zh) * 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
TIANZHUO YAO ET AL: "Site-Specific PEGylated Adeno-Associated Viruses with Increased Serum Stability and Reduced Immunogenicity", MOLECULES, vol. 22, no. 7, 11 July 2017 (2017-07-11), pages 2, XP093275418, DOI: 10.3390/molecules22071155 *

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Publication number Publication date
US20230175013A1 (en) 2023-06-08
JP2023524401A (ja) 2023-06-12
JP2026062759A (ja) 2026-04-10
AU2021264081A1 (en) 2022-11-03
WO2021222899A1 (en) 2021-11-04
KR20230005941A (ko) 2023-01-10
EP4143206A1 (en) 2023-03-08
CA3177573A1 (en) 2021-11-04
IL297780A (en) 2022-12-01

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