AU2020263757B2 - AAV mutant having brain-targeting property - Google Patents

AAV mutant having brain-targeting property

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Publication number
AU2020263757B2
AU2020263757B2 AU2020263757A AU2020263757A AU2020263757B2 AU 2020263757 B2 AU2020263757 B2 AU 2020263757B2 AU 2020263757 A AU2020263757 A AU 2020263757A AU 2020263757 A AU2020263757 A AU 2020263757A AU 2020263757 B2 AU2020263757 B2 AU 2020263757B2
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AU
Australia
Prior art keywords
gly
peptide
capsid protein
sequence
aav
Prior art date
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Active
Application number
AU2020263757A
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English (en)
Other versions
AU2020263757A1 (en
Inventor
Tatsuji Enoki
Junichi Mineno
Toshikazu NISHIE
Fuyuko Takashima
Yoshinori Tanaka
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Takara Bio Inc
Original Assignee
Takara Bio Inc
Priority date (The priority date 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 date listed.)
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Publication of AU2020263757A1 publication Critical patent/AU2020263757A1/en
Application granted granted Critical
Publication of AU2020263757B2 publication Critical patent/AU2020263757B2/en
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Anticipated expiration legal-status Critical

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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
    • 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
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B50/00—Methods of creating libraries, e.g. combinatorial synthesis
    • C40B50/06—Biochemical methods, e.g. using enzymes or whole viable microorganisms
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/14122—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
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2020263757A 2019-04-24 2020-04-23 AAV mutant having brain-targeting property Active AU2020263757B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019-082417 2019-04-24
JP2019082417 2019-04-24
PCT/JP2020/017495 WO2020218419A1 (ja) 2019-04-24 2020-04-23 脳に指向性を有するａａｖ変異体

Publications (2)

Publication Number Publication Date
AU2020263757A1 AU2020263757A1 (en) 2021-10-14
AU2020263757B2 true AU2020263757B2 (en) 2025-08-28

Family

ID=72942142

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020263757A Active AU2020263757B2 (en) 2019-04-24 2020-04-23 AAV mutant having brain-targeting property

Country Status (10)

Country Link
US (1) US12421526B2 (https=)
EP (1) EP3960755A4 (https=)
JP (1) JP7524166B2 (https=)
KR (1) KR102917057B1 (https=)
CN (1) CN113748122B (https=)
AU (1) AU2020263757B2 (https=)
BR (1) BR112021017603A2 (https=)
CA (1) CA3132447A1 (https=)
IL (1) IL286212A (https=)
WO (1) WO2020218419A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3575398A4 (en) * 2017-01-30 2020-11-11 Nippon Medical School Foundation ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT
CN115960177B (zh) * 2022-10-09 2023-07-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN116789739B (zh) * 2022-12-08 2024-01-26 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN116284262B (zh) * 2023-02-13 2026-03-17 和元生物技术（上海）股份有限公司 高效感染ht-22细胞的腺相关病毒突变体
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN121569040A (zh) 2023-07-27 2026-02-24 宝生物工程株式会社 脑趋向性aav突变体
CN121816425A (zh) * 2023-09-11 2026-04-07 学校法人顺天堂 能够感染内耳的aav变体

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014103957A1 (ja) * 2012-12-25 2014-07-03 タカラバイオ株式会社 Ａａｖ変異体
JP2017513486A (ja) * 2014-04-17 2017-06-01 ウニヴェルズィテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 脳および脊髄に標的化遺伝子移入するためのウイルスベクター

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
EP2176283B1 (en) 2007-07-14 2016-11-02 University of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2010114143A1 (ja) * 2009-03-30 2010-10-07 国立大学法人岡山大学 特異的プロモーターの活性を上昇させるシステム及び該システムを保持したベクター
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
WO2013014764A1 (ja) 2011-07-27 2013-01-31 三菱電機株式会社 通信システム、通信装置および通信方法
HK1222673A1 (zh) 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
CA2942289C (en) * 2014-03-10 2024-05-21 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
US11364308B2 (en) * 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
EP3890017B1 (en) 2018-12-04 2025-08-13 Ningbo Sunny Opotech Co., Ltd. Photosensitive assembly, photographic module, manufacturing method for photographic assembly, and electronic device

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014103957A1 (ja) * 2012-12-25 2014-07-03 タカラバイオ株式会社 Ａａｖ変異体
JP2017513486A (ja) * 2014-04-17 2017-06-01 ウニヴェルズィテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 脳および脊髄に標的化遺伝子移入するためのウイルスベクター

Also Published As

Publication number Publication date
JP7524166B2 (ja) 2024-07-29
JPWO2020218419A1 (https=) 2020-10-29
BR112021017603A2 (pt) 2021-11-16
IL286212A (en) 2021-10-31
CA3132447A1 (en) 2020-10-29
AU2020263757A1 (en) 2021-10-14
KR20220004072A (ko) 2022-01-11
US12421526B2 (en) 2025-09-23
KR102917057B1 (ko) 2026-01-23
EP3960755A1 (en) 2022-03-02
EP3960755A4 (en) 2023-11-22
US20220162637A1 (en) 2022-05-26
WO2020218419A1 (ja) 2020-10-29
CN113748122B (zh) 2024-11-22
CN113748122A (zh) 2021-12-03

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