JP2021522840A - 改善された遺伝子送達特性を示すウイルスベクター - Google Patents

改善された遺伝子送達特性を示すウイルスベクター Download PDF

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Publication number
JP2021522840A
JP2021522840A JP2020564154A JP2020564154A JP2021522840A JP 2021522840 A JP2021522840 A JP 2021522840A JP 2020564154 A JP2020564154 A JP 2020564154A JP 2020564154 A JP2020564154 A JP 2020564154A JP 2021522840 A JP2021522840 A JP 2021522840A
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seq
capsid polypeptide
viral
mutation
viral capsid
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Inventor
ジョージ エム． チャーチ，
ジョージ エム． チャーチ，
エリック ケルシック，
エリック ケルシック，
ピアース オグデン，
ピアース オグデン，
サム シナイ，
サム シナイ，
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Harvard University
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Harvard University
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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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    • C12N15/63—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020564154A 2018-05-15 2019-05-08 改善された遺伝子送達特性を示すウイルスベクター Withdrawn JP2021522840A (ja)

Priority Applications (1)

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JP2024027527A JP7741575B2 (ja) 2018-05-15 2024-02-27 改善された遺伝子送達特性を示すウイルスベクター

Applications Claiming Priority (3)

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US201862671949P 2018-05-15 2018-05-15
US62/671,949 2018-05-15
PCT/US2019/031307 WO2019221992A1 (en) 2018-05-15 2019-05-08 Viral vectors exhibiting improved gene delivery properties

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JP2024027527A Active JP7741575B2 (ja) 2018-05-15 2024-02-27 改善された遺伝子送達特性を示すウイルスベクター

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US (1) US12338267B2 (enExample)
EP (1) EP3813845A4 (enExample)
JP (2) JP2021522840A (enExample)
AU (1) AU2019270900A1 (enExample)
CA (1) CA3100066A1 (enExample)
WO (1) WO2019221992A1 (enExample)

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Publication number Priority date Publication date Assignee Title
EP3461836A1 (en) * 2017-09-28 2019-04-03 Universität zu Köln Mutated adeno-associated viral capsid proteins for chemical coupling of ligands, nanoparticles or drugs via thioether binding and production method thereof
WO2021113767A1 (en) * 2019-12-04 2021-06-10 Sangamo Therapeutics, Inc. Novel compositions and methods for producing recombinant aav
CN115989234A (zh) * 2020-01-29 2023-04-18 建新公司 用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途
US20230175013A1 (en) * 2020-05-01 2023-06-08 Trustees Of Boston College Controlled modification of adeno-associated virus (aav) for enhanced gene therapy
CA3222914A1 (en) * 2021-06-23 2022-12-29 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2023283962A1 (en) * 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
WO2023060264A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
EP4508065A2 (en) * 2022-04-15 2025-02-19 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US20250352671A1 (en) * 2022-04-15 2025-11-20 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US12480098B1 (en) * 2024-10-18 2025-11-25 Aavatar Therapeutics Co., Ltd. Adeno-associated virus variant

Citations (4)

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WO2015121501A1 (en) * 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector
US20170096683A1 (en) * 2014-05-02 2017-04-06 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2017106236A1 (en) * 2015-12-14 2017-06-22 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
WO2017147477A1 (en) * 2016-02-26 2017-08-31 University Of Florida Research Foundation, Inc. Aav heparin mutants that display significantly improved eye and brain transduction

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US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO2001068888A2 (en) 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP3910063A1 (en) 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP3492596A1 (en) * 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
WO2009108274A2 (en) * 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター

Patent Citations (4)

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WO2015121501A1 (en) * 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector
US20170096683A1 (en) * 2014-05-02 2017-04-06 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2017106236A1 (en) * 2015-12-14 2017-06-22 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
WO2017147477A1 (en) * 2016-02-26 2017-08-31 University Of Florida Research Foundation, Inc. Aav heparin mutants that display significantly improved eye and brain transduction

Non-Patent Citations (5)

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GENE THER., vol. 23, no. 11, JPN6023014055, 2016, pages 767 - 774, ISSN: 0005183966 *
J VIROL METHODS., vol. 250, JPN6023014053, December 2017 (2017-12-01), pages 47 - 54, ISSN: 0005183969 *
JOURNAL OF VIROLOGY, vol. 74, no. 18, JPN6023014051, 2000, pages 8635 - 8647, ISSN: 0005183968 *
MOLECULAR THERAPY, vol. 8, no. 1, JPN6023014054, 2003, pages 151 - 157, ISSN: 0005183970 *
PLOS ONE, vol. 8, no. 3, JPN6023014049, 2013, pages 59142 - 1, ISSN: 0005183967 *

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Publication number Publication date
EP3813845A1 (en) 2021-05-05
EP3813845A4 (en) 2022-10-26
WO2019221992A1 (en) 2019-11-21
US20210230229A1 (en) 2021-07-29
AU2019270900A1 (en) 2020-10-15
JP7741575B2 (ja) 2025-09-18
JP2024051082A (ja) 2024-04-10
US12338267B2 (en) 2025-06-24
CA3100066A1 (en) 2019-11-21

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