KR102848518B1 - 캡시드 탈아미드화가 감소된 신규 아데노-연관 바이러스(aav) 벡터 및 이의 용도 - Google Patents

캡시드 탈아미드화가 감소된 신규 아데노-연관 바이러스(aav) 벡터 및 이의 용도

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KR102848518B1
KR102848518B1 KR1020207027462A KR20207027462A KR102848518B1 KR 102848518 B1 KR102848518 B1 KR 102848518B1 KR 1020207027462 A KR1020207027462 A KR 1020207027462A KR 20207027462 A KR20207027462 A KR 20207027462A KR 102848518 B1 KR102848518 B1 KR 102848518B1
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hsa
mir
seq
capsid
aav
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KR20210006327A (ko
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제임스 엠. 윌슨
에이프릴 테페
케빈 터너
조슈아 조이너 심스
치앙 왕
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더 트러스티스 오브 더 유니버시티 오브 펜실베니아
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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)
KR1020207027462A 2018-02-27 2019-02-27 캡시드 탈아미드화가 감소된 신규 아데노-연관 바이러스(aav) 벡터 및 이의 용도 Active KR102848518B1 (ko)

Applications Claiming Priority (29)

Application Number Priority Date Filing Date Title
US201862635964P 2018-02-27 2018-02-27
US201862635968P 2018-02-27 2018-02-27
US62/635,968 2018-02-27
US62/635,964 2018-02-27
US201862663788P 2018-04-27 2018-04-27
US201862663797P 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
US201862667888P 2018-05-07 2018-05-07
US201862667881P 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
US62/677,474 2018-05-29
US62/667,585 2018-05-29
US62/677,471 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US201862703673P 2018-07-26 2018-07-26
US62/703,673 2018-07-26
US62/703,670 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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KR20210006327A KR20210006327A (ko) 2021-01-18
KR102848518B1 true KR102848518B1 (ko) 2025-08-22

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US (2) US12416016B2 (https=)
EP (1) EP3768695A4 (https=)
JP (2) JP7498665B2 (https=)
KR (1) KR102848518B1 (https=)
CN (2) CN112236443B (https=)
AU (1) AU2019228504B2 (https=)
BR (1) BR112020017278A2 (https=)
CA (1) CA3091795A1 (https=)
CL (1) CL2020002201A1 (https=)
IL (1) IL276860A (https=)
MA (1) MA52087A (https=)
MX (1) MX2020008932A (https=)
SG (1) SG11202007942YA (https=)
WO (1) WO2019169004A1 (https=)

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WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
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CN112236443B (zh) 2025-02-07
CN112236443A (zh) 2021-01-15
JP7498665B2 (ja) 2024-06-12
BR112020017278A2 (pt) 2020-12-22
MA52087A (fr) 2021-06-02
EP3768695A1 (en) 2021-01-27
WO2019169004A1 (en) 2019-09-06
JP2021515548A (ja) 2021-06-24
EP3768695A4 (en) 2022-04-06
AU2019228504A1 (en) 2020-09-10
KR20210006327A (ko) 2021-01-18
WO2019169004A8 (en) 2019-12-05
JP2024115558A (ja) 2024-08-26
CA3091795A1 (en) 2019-09-06
AU2019228504B2 (en) 2025-06-26
US20210123073A1 (en) 2021-04-29
IL276860A (en) 2020-10-29
SG11202007942YA (en) 2020-09-29
US12416016B2 (en) 2025-09-16
MX2020008932A (es) 2020-10-01
CN120519403A (zh) 2025-08-22
CL2020002201A1 (es) 2020-12-18
US20260049334A1 (en) 2026-02-19
WO2019169004A9 (en) 2019-10-31

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