BR112022004183A2 - Métodos e composições para modular a interação entre vírus adeno-associado (aav) e receptor de aav (aavr) para biodistribuição alterada de aav - Google Patents

Métodos e composições para modular a interação entre vírus adeno-associado (aav) e receptor de aav (aavr) para biodistribuição alterada de aav

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Publication number
BR112022004183A2
BR112022004183A2 BR112022004183A BR112022004183A BR112022004183A2 BR 112022004183 A2 BR112022004183 A2 BR 112022004183A2 BR 112022004183 A BR112022004183 A BR 112022004183A BR 112022004183 A BR112022004183 A BR 112022004183A BR 112022004183 A2 BR112022004183 A2 BR 112022004183A2
Authority
BR
Brazil
Prior art keywords
aav
adeno
compositions
methods
aavr
Prior art date
Application number
BR112022004183A
Other languages
English (en)
Portuguese (pt)
Inventor
Christopher Tipper
Eric Zinn
H Vandenberghe Luk
Original Assignee
Massachusetts Eye & Ear Infirmary
Schepens Eye Res Inst
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 Massachusetts Eye & Ear Infirmary, Schepens Eye Res Inst filed Critical Massachusetts Eye & Ear Infirmary
Publication of BR112022004183A2 publication Critical patent/BR112022004183A2/pt

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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Peptides Or Proteins (AREA)
BR112022004183A 2019-09-09 2020-09-09 Métodos e composições para modular a interação entre vírus adeno-associado (aav) e receptor de aav (aavr) para biodistribuição alterada de aav BR112022004183A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962897973P 2019-09-09 2019-09-09
US201962934996P 2019-11-13 2019-11-13
PCT/US2020/050027 WO2021050614A2 (en) 2019-09-09 2020-09-09 Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav

Publications (1)

Publication Number Publication Date
BR112022004183A2 true BR112022004183A2 (pt) 2022-05-31

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022004183A BR112022004183A2 (pt) 2019-09-09 2020-09-09 Métodos e composições para modular a interação entre vírus adeno-associado (aav) e receptor de aav (aavr) para biodistribuição alterada de aav

Country Status (10)

Country Link
US (1) US12570998B2 (https=)
EP (1) EP4028531A2 (https=)
JP (1) JP2022547197A (https=)
KR (1) KR20220054435A (https=)
CN (1) CN114667349A (https=)
AU (1) AU2020347165A1 (https=)
BR (1) BR112022004183A2 (https=)
CA (1) CA3153972A1 (https=)
IL (1) IL291136A (https=)
WO (1) WO2021050614A2 (https=)

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WO2022150634A2 (en) * 2021-01-08 2022-07-14 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
US20240352077A1 (en) * 2021-08-25 2024-10-24 Children's Medical Research Institute Modified aav capsids and vectors
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KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2023095034A1 (en) * 2021-11-24 2023-06-01 Novartis Ag Modulators of adeno-associated virus transduction and uses thereof
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WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2025179124A1 (en) * 2024-02-23 2025-08-28 The Board Of Regents Of The University Of Texas System Enhanced aav9 capsid protein
WO2025194042A1 (en) 2024-03-14 2025-09-18 Affinia Therapeutics Inc. Plasmid system for production of recombinant adenoassociated virus
WO2025235491A1 (en) 2024-05-07 2025-11-13 Affinia Therapeutics Inc. Recombinant aav for treatment of cardiac diseases

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Also Published As

Publication number Publication date
CA3153972A1 (en) 2021-03-18
IL291136A (en) 2022-05-01
US12570998B2 (en) 2026-03-10
AU2020347165A1 (en) 2022-03-31
WO2021050614A3 (en) 2021-05-20
EP4028531A2 (en) 2022-07-20
KR20220054435A (ko) 2022-05-02
CN114667349A (zh) 2022-06-24
WO2021050614A2 (en) 2021-03-18
US20220411820A1 (en) 2022-12-29
JP2022547197A (ja) 2022-11-10

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