AU2017302013B2 - Adeno-associated virus virions with variant capsid and methods of use thereof - Google Patents

Adeno-associated virus virions with variant capsid and methods of use thereof Download PDF

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Publication number
AU2017302013B2
AU2017302013B2 AU2017302013A AU2017302013A AU2017302013B2 AU 2017302013 B2 AU2017302013 B2 AU 2017302013B2 AU 2017302013 A AU2017302013 A AU 2017302013A AU 2017302013 A AU2017302013 A AU 2017302013A AU 2017302013 B2 AU2017302013 B2 AU 2017302013B2
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Prior art keywords
seq
amino acids
aav
capsid protein
amino acid
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AU2017302013A1 (en
Inventor
William A. Beltran
Leah C. Byrne
John G. Flannery
David V. Schaffer
Meike VISEL
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University of Pennsylvania
University of California, San Diego
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University of Pennsylvania
University of California Berkeley
University of California, San Diego
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Priority to AU2022202556A priority Critical patent/AU2022202556B2/en
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Priority to AU2024219380A priority patent/AU2024219380C1/en
Priority to AU2026202557A priority patent/AU2026202557A1/en
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    • 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
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P27/06—Antiglaucoma agents or miotics
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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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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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
    • 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
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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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Manufacturing & Machinery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
AU2017302013A 2016-07-29 2017-07-27 Adeno-associated virus virions with variant capsid and methods of use thereof Active AU2017302013B2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU2022202556A AU2022202556B2 (en) 2016-07-29 2022-04-19 Adeno-associated virus virions with variant capsid and methods of use thereof
AU2024219380A AU2024219380C1 (en) 2016-07-29 2024-09-04 Adeno-associated virus virions with variant capsid and methods of use thereof
AU2026202557A AU2026202557A1 (en) 2016-07-29 2026-04-07 Adeno-associated virus virions with variant capsid and methods of use thereof

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662368929P 2016-07-29 2016-07-29
US62/368,929 2016-07-29
PCT/US2017/044206 WO2018022905A2 (en) 2016-07-29 2017-07-27 Adeno-associated virus virions with variant capsid and methods of use thereof

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AU2022202556A Division AU2022202556B2 (en) 2016-07-29 2022-04-19 Adeno-associated virus virions with variant capsid and methods of use thereof

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AU2017302013A1 AU2017302013A1 (en) 2019-02-07
AU2017302013B2 true AU2017302013B2 (en) 2022-05-26

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AU2017302013A Active AU2017302013B2 (en) 2016-07-29 2017-07-27 Adeno-associated virus virions with variant capsid and methods of use thereof
AU2022202556A Active AU2022202556B2 (en) 2016-07-29 2022-04-19 Adeno-associated virus virions with variant capsid and methods of use thereof
AU2024219380A Active AU2024219380C1 (en) 2016-07-29 2024-09-04 Adeno-associated virus virions with variant capsid and methods of use thereof
AU2026202557A Pending AU2026202557A1 (en) 2016-07-29 2026-04-07 Adeno-associated virus virions with variant capsid and methods of use thereof

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AU2022202556A Active AU2022202556B2 (en) 2016-07-29 2022-04-19 Adeno-associated virus virions with variant capsid and methods of use thereof
AU2024219380A Active AU2024219380C1 (en) 2016-07-29 2024-09-04 Adeno-associated virus virions with variant capsid and methods of use thereof
AU2026202557A Pending AU2026202557A1 (en) 2016-07-29 2026-04-07 Adeno-associated virus virions with variant capsid and methods of use thereof

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US (5) US11554180B2 (enExample)
EP (3) EP3827812B1 (enExample)
JP (3) JP7071332B2 (enExample)
KR (3) KR102508820B1 (enExample)
CN (3) CN109640949B (enExample)
AU (4) AU2017302013B2 (enExample)
CA (1) CA3029833A1 (enExample)
MX (3) MX2019001276A (enExample)
WO (1) WO2018022905A2 (enExample)

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