AU2019228504B2 - Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor - Google Patents

Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor Download PDF

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AU2019228504B2
AU2019228504B2 AU2019228504A AU2019228504A AU2019228504B2 AU 2019228504 B2 AU2019228504 B2 AU 2019228504B2 AU 2019228504 A AU2019228504 A AU 2019228504A AU 2019228504 A AU2019228504 A AU 2019228504A AU 2019228504 B2 AU2019228504 B2 AU 2019228504B2
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hsa
mir
capsid
aav
proteins
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AU2019228504A1 (en
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Joshua Joyner SIMS
April TEPE
Kevin Turner
Qiang Wang
James M. Wilson
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University of Pennsylvania
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University of Pennsylvania
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    • 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
    • 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
    • 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
    • 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
    • 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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2019228504A 2018-02-27 2019-02-27 Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor Active AU2019228504B2 (en)

Applications Claiming Priority (29)

Application Number Priority Date Filing Date Title
US201862635968P 2018-02-27 2018-02-27
US201862635964P 2018-02-27 2018-02-27
US62/635,964 2018-02-27
US62/635,968 2018-02-27
US201862663797P 2018-04-27 2018-04-27
US201862663788P 2018-04-27 2018-04-27
US62/663,788 2018-04-27
US62/663,797 2018-04-27
US201862667587P 2018-05-06 2018-05-06
US62/667,587 2018-05-06
US201862667881P 2018-05-07 2018-05-07
US201862667888P 2018-05-07 2018-05-07
US62/667,881 2018-05-07
US62/667,888 2018-05-07
US201862677474P 2018-05-29 2018-05-29
US201862667585P 2018-05-29 2018-05-29
US201862677471P 2018-05-29 2018-05-29
US62/677,471 2018-05-29
US62/667,585 2018-05-29
US62/677,474 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US201862703673P 2018-07-26 2018-07-26
US62/703,670 2018-07-26
US62/703,673 2018-07-26
US201862722382P 2018-08-24 2018-08-24
US201862722388P 2018-08-24 2018-08-24
US62/722,388 2018-08-24
US62/722,382 2018-08-24
PCT/US2019/019861 WO2019169004A1 (en) 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Publications (2)

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AU2019228504A1 AU2019228504A1 (en) 2020-09-10
AU2019228504B2 true AU2019228504B2 (en) 2025-06-26

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US (2) US12416016B2 (enExample)
EP (1) EP3768695A4 (enExample)
JP (2) JP7498665B2 (enExample)
KR (1) KR102848518B1 (enExample)
CN (2) CN120519403A (enExample)
AU (1) AU2019228504B2 (enExample)
BR (1) BR112020017278A2 (enExample)
CA (1) CA3091795A1 (enExample)
CL (1) CL2020002201A1 (enExample)
IL (1) IL276860A (enExample)
MA (1) MA52087A (enExample)
MX (1) MX2020008932A (enExample)
SG (1) SG11202007942YA (enExample)
WO (1) WO2019169004A1 (enExample)

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BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
EP3884057A4 (en) * 2018-12-21 2022-09-07 The University of North Carolina at Chapel Hill OPTIMIZED GALC GENES AND EXPRESSION CASSETTE AND THEIR USE
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
WO2020223232A1 (en) 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
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AU2020369508A1 (en) * 2019-10-21 2022-05-26 The Trustees Of The University Of Pennsylvania AAV3B variants with improved production yield and liver tropism
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US20230175014A1 (en) * 2020-04-27 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity
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WO2022015715A1 (en) 2020-07-13 2022-01-20 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
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WO2022082109A1 (en) * 2020-10-18 2022-04-21 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
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WO2023081807A1 (en) * 2021-11-04 2023-05-11 The Trustees of the University of Pennsylvania Penn Center for Innovation Compositions and methods for reducing pcsk9 levels in a subject
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
US20250177495A1 (en) 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
CN121487959A (zh) * 2023-03-25 2026-02-06 杜克大学 包含肾脏趋向性aav的组合物及其使用方法
WO2025250683A1 (en) * 2024-05-29 2025-12-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid

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WO2018035059A1 (en) * 2016-08-15 2018-02-22 Genzyme Corporation Methods for detecting aav

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Also Published As

Publication number Publication date
WO2019169004A9 (en) 2019-10-31
US20210123073A1 (en) 2021-04-29
KR20210006327A (ko) 2021-01-18
MX2020008932A (es) 2020-10-01
CN112236443B (zh) 2025-02-07
US20260049334A1 (en) 2026-02-19
CN120519403A (zh) 2025-08-22
JP7498665B2 (ja) 2024-06-12
CA3091795A1 (en) 2019-09-06
US12416016B2 (en) 2025-09-16
CN112236443A (zh) 2021-01-15
BR112020017278A2 (pt) 2020-12-22
KR102848518B1 (ko) 2025-08-22
MA52087A (fr) 2021-06-02
WO2019169004A1 (en) 2019-09-06
AU2019228504A1 (en) 2020-09-10
JP7860169B2 (ja) 2026-05-15
SG11202007942YA (en) 2020-09-29
JP2021515548A (ja) 2021-06-24
JP2024115558A (ja) 2024-08-26
CL2020002201A1 (es) 2020-12-18
IL276860A (en) 2020-10-29
EP3768695A1 (en) 2021-01-27
EP3768695A4 (en) 2022-04-06
WO2019169004A8 (en) 2019-12-05

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