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WO2023225455A3 - Génomes d'aav synthétiques pour une distribution de gène améliorée - Google Patents

Génomes d'aav synthétiques pour une distribution de gène améliorée Download PDF

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Publication number
WO2023225455A3
WO2023225455A3 PCT/US2023/066873 US2023066873W WO2023225455A3 WO 2023225455 A3 WO2023225455 A3 WO 2023225455A3 US 2023066873 W US2023066873 W US 2023066873W WO 2023225455 A3 WO2023225455 A3 WO 2023225455A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene delivery
improved gene
synthetic
aav genomes
genomes
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.)
Ceased
Application number
PCT/US2023/066873
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English (en)
Other versions
WO2023225455A2 (fr
Inventor
Christopher Chavez
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.)
Visgenx Inc
Original Assignee
Visgenx Inc
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 Visgenx Inc filed Critical Visgenx Inc
Publication of WO2023225455A2 publication Critical patent/WO2023225455A2/fr
Publication of WO2023225455A3 publication Critical patent/WO2023225455A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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
    • 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
    • C12N2750/14141—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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

L'invention concerne des procédés de production de génomes viraux adéno-associés synthétiques (AAV) pour une distribution de gènes améliorée. La présente invention comprend également des génomes d'AAV synthétiques sans limitation de taille de charge utile et aptes à recevoir des cassettes d'expression de n'importe quelle taille. L'invention concerne également des compositions et des procédés pour administrer un génome d'AAV synthétique à une cellule afin de traiter une ou plusieurs maladies ou un ou plusieurs états chez un sujet.
PCT/US2023/066873 2022-05-18 2023-05-11 Génomes d'aav synthétiques pour une distribution de gène améliorée Ceased WO2023225455A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263343287P 2022-05-18 2022-05-18
US63/343,287 2022-05-18

Publications (2)

Publication Number Publication Date
WO2023225455A2 WO2023225455A2 (fr) 2023-11-23
WO2023225455A3 true WO2023225455A3 (fr) 2023-12-21

Family

ID=88836237

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/066873 Ceased WO2023225455A2 (fr) 2022-05-18 2023-05-11 Génomes d'aav synthétiques pour une distribution de gène améliorée

Country Status (1)

Country Link
WO (1) WO2023225455A2 (fr)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018170473A1 (fr) * 2017-03-17 2018-09-20 Adverum Biotechnologies, Inc. Compositions et procédés d'amplification d'expression génique
WO2021011842A1 (fr) * 2019-07-17 2021-01-21 Generation Bio Co. Production synthétique de vecteurs de type adn viraux adéno-associés simple brin

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018170473A1 (fr) * 2017-03-17 2018-09-20 Adverum Biotechnologies, Inc. Compositions et procédés d'amplification d'expression génique
WO2021011842A1 (fr) * 2019-07-17 2021-01-21 Generation Bio Co. Production synthétique de vecteurs de type adn viraux adéno-associés simple brin

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ADACHI KUMI, TOMONO TARO, OKADA HIRONORI, SHIOZAWA YUSUKE, YAMAMOTO MOTOKO, MIYAGAWA YOSHITAKA, OKADA TAKASHI: "A PCR-amplified transgene fragment flanked by a single copy of a truncated inverted terminal repeat for recombinant adeno-associated virus production prevents unnecessary plasmid DNA packaging", GENE THERAPY, NATURE PUBLISHING GROUP, LONDON, GB, 11 October 2021 (2021-10-11), GB , XP055943083, ISSN: 0969-7128, DOI: 10.1038/s41434-021-00299-x *
MELANIE D. WHITE, RUTH V. J. MILNE, MATTHEW F. NOLAN: "A Molecular Toolbox for Rapid Generation of Viral Vectors to Up- or Down-Regulate Neuronal Gene Expression in vivo", FRONTIERS IN MOLECULAR NEUROSCIENCE, vol. 4, XP055718467, DOI: 10.3389/fnmol.2011.00008 *

Also Published As

Publication number Publication date
WO2023225455A2 (fr) 2023-11-23

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