WO2024102739A3 - Adeno-associated virus (aav) production - Google Patents

Adeno-associated virus (aav) production Download PDF

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Publication number
WO2024102739A3
WO2024102739A3 PCT/US2023/078957 US2023078957W WO2024102739A3 WO 2024102739 A3 WO2024102739 A3 WO 2024102739A3 US 2023078957 W US2023078957 W US 2023078957W WO 2024102739 A3 WO2024102739 A3 WO 2024102739A3
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WIPO (PCT)
Prior art keywords
aav
adeno
associated virus
production
viral particle
Prior art date
Application number
PCT/US2023/078957
Other languages
French (fr)
Other versions
WO2024102739A2 (en
Inventor
Joseph J. HIGGINS
Ray TABIBIZAR
Feng Yao
Quan Karen ZHU
Original Assignee
Saliogen Therapeutics, Inc.
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Publication date
Application filed by Saliogen Therapeutics, Inc. filed Critical Saliogen Therapeutics, Inc.
Publication of WO2024102739A2 publication Critical patent/WO2024102739A2/en
Publication of WO2024102739A3 publication Critical patent/WO2024102739A3/en

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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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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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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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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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
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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
    • C12N2750/14151—Methods of production or purification of viral material
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/90—Vectors containing a transposable element
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
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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 RNAses, DNAses

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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

A method of making a viral particle packaging and producer ceil line is provided. The cell line is based on an adeno-associated virus (AAV) vector, the cell line is caused to expresses, in association with the viral particle, a desired transgene.
PCT/US2023/078957 2022-11-07 2023-11-07 Adeno-associated virus (aav) production WO2024102739A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263423220P 2022-11-07 2022-11-07
US63/423,220 2022-11-07

Publications (2)

Publication Number Publication Date
WO2024102739A2 WO2024102739A2 (en) 2024-05-16
WO2024102739A3 true WO2024102739A3 (en) 2024-07-04

Family

ID=91033437

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/078957 WO2024102739A2 (en) 2022-11-07 2023-11-07 Adeno-associated virus (aav) production

Country Status (1)

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WO (1) WO2024102739A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220145328A1 (en) * 2020-07-30 2022-05-12 Shape Therapeutics Inc. STABLE CELL LINES FOR INDUCIBLE PRODUCTION OF rAAV VIRIONS
US20220177854A1 (en) * 2018-10-17 2022-06-09 Glaxosmithkline Intellectual Property Development Limited Adeno-associated viral vector producer cell lines
WO2023173105A2 (en) * 2022-03-10 2023-09-14 Saliogen Therapeutics, Inc. Tetracycline inducer/repressor system and simplified viral particle production

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220177854A1 (en) * 2018-10-17 2022-06-09 Glaxosmithkline Intellectual Property Development Limited Adeno-associated viral vector producer cell lines
US20220145328A1 (en) * 2020-07-30 2022-05-12 Shape Therapeutics Inc. STABLE CELL LINES FOR INDUCIBLE PRODUCTION OF rAAV VIRIONS
WO2023173105A2 (en) * 2022-03-10 2023-09-14 Saliogen Therapeutics, Inc. Tetracycline inducer/repressor system and simplified viral particle production

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Publication number Publication date
WO2024102739A2 (en) 2024-05-16

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