AU2019270900A1 - Viral vectors exhibiting improved gene delivery properties - Google Patents

Viral vectors exhibiting improved gene delivery properties Download PDF

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Publication number
AU2019270900A1
AU2019270900A1 AU2019270900A AU2019270900A AU2019270900A1 AU 2019270900 A1 AU2019270900 A1 AU 2019270900A1 AU 2019270900 A AU2019270900 A AU 2019270900A AU 2019270900 A AU2019270900 A AU 2019270900A AU 2019270900 A1 AU2019270900 A1 AU 2019270900A1
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AU
Australia
Prior art keywords
mutation
seq
viral
capsid polypeptide
cell
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
AU2019270900A
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English (en)
Inventor
George M. Church
Eric KELSIC
Pierce OGDEN
Sam SINAI
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.)
Harvard University
Original Assignee
Harvard University
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.)
Filing date
Publication date
Application filed by Harvard University filed Critical Harvard University
Publication of AU2019270900A1 publication Critical patent/AU2019270900A1/en
Pending legal-status Critical Current

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • 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)
AU2019270900A 2018-05-15 2019-05-08 Viral vectors exhibiting improved gene delivery properties Pending AU2019270900A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (1)

Publication Number Publication Date
AU2019270900A1 true AU2019270900A1 (en) 2020-10-15

Family

ID=68540740

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019270900A Pending AU2019270900A1 (en) 2018-05-15 2019-05-08 Viral vectors exhibiting improved gene delivery properties

Country Status (6)

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

Families Citing this family (11)

* Cited by examiner, † Cited by third party
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

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
CN116478254A (zh) * 2015-12-14 2023-07-25 北卡罗来纳大学教堂山分校 增强细小病毒载体递送的修饰衣壳蛋白
US20190071681A1 (en) * 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction

Also Published As

Publication number Publication date
JP2021522840A (ja) 2021-09-02
EP3813845A1 (en) 2021-05-05
EP3813845A4 (en) 2022-10-26
WO2019221992A1 (en) 2019-11-21
US20210230229A1 (en) 2021-07-29
JP7741575B2 (ja) 2025-09-18
JP2024051082A (ja) 2024-04-10
US12338267B2 (en) 2025-06-24
CA3100066A1 (en) 2019-11-21

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