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WO2023215851A3 - Plasmid optimized for packaging of aav vectors - Google Patents

Plasmid optimized for packaging of aav vectors Download PDF

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Publication number
WO2023215851A3
WO2023215851A3 PCT/US2023/066639 US2023066639W WO2023215851A3 WO 2023215851 A3 WO2023215851 A3 WO 2023215851A3 US 2023066639 W US2023066639 W US 2023066639W WO 2023215851 A3 WO2023215851 A3 WO 2023215851A3
Authority
WO
WIPO (PCT)
Prior art keywords
packaging
aav vectors
aav vector
plasmid
plasmid optimized
Prior art date
Application number
PCT/US2023/066639
Other languages
French (fr)
Other versions
WO2023215851A2 (en
Inventor
Scott Loiler
Original Assignee
Apic Bio, 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 Apic Bio, Inc. filed Critical Apic Bio, Inc.
Publication of WO2023215851A2 publication Critical patent/WO2023215851A2/en
Publication of WO2023215851A3 publication Critical patent/WO2023215851A3/en

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

The present invention includes an AAV vector packaging platform. The invention further includes helper plasmids useful for the production of AAV vector particles, as well as methods of producing and using AAV vector particles to introduce transgenes into target cells and treat diseases in subjects in need thereof.
PCT/US2023/066639 2022-05-06 2023-05-05 Plasmid optimized for packaging of aav vectors WO2023215851A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263339338P 2022-05-06 2022-05-06
US63/339,338 2022-05-06

Publications (2)

Publication Number Publication Date
WO2023215851A2 WO2023215851A2 (en) 2023-11-09
WO2023215851A3 true WO2023215851A3 (en) 2024-03-14

Family

ID=88647219

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/066639 WO2023215851A2 (en) 2022-05-06 2023-05-05 Plasmid optimized for packaging of aav vectors

Country Status (1)

Country Link
WO (1) WO2023215851A2 (en)

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6329181B1 (en) * 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US20090042297A1 (en) * 2007-06-01 2009-02-12 George Jr Alfred L Piggybac transposon-based vectors and methods of nucleic acid integration
US20100028880A1 (en) * 2008-08-04 2010-02-04 National Tsing Hua University Method for sustained expression of an exogenous gene
WO2017165859A1 (en) * 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
US20210047650A1 (en) * 2018-02-02 2021-02-18 University Of Massachusetts Campaign-ready series of recombinant adeno-associated virus (raav) complementing plasmids
US20210113615A1 (en) * 2018-04-13 2021-04-22 Ludwig Institute For Cancer Research Ltd. Heterodimeric inactivatable chimeric antigen receptors
WO2021188892A1 (en) * 2020-03-19 2021-09-23 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
US20210363498A1 (en) * 2013-08-30 2021-11-25 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
WO2022026409A1 (en) * 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6329181B1 (en) * 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US20090042297A1 (en) * 2007-06-01 2009-02-12 George Jr Alfred L Piggybac transposon-based vectors and methods of nucleic acid integration
US20100028880A1 (en) * 2008-08-04 2010-02-04 National Tsing Hua University Method for sustained expression of an exogenous gene
US20210363498A1 (en) * 2013-08-30 2021-11-25 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
WO2017165859A1 (en) * 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
US20210047650A1 (en) * 2018-02-02 2021-02-18 University Of Massachusetts Campaign-ready series of recombinant adeno-associated virus (raav) complementing plasmids
US20210113615A1 (en) * 2018-04-13 2021-04-22 Ludwig Institute For Cancer Research Ltd. Heterodimeric inactivatable chimeric antigen receptors
WO2021188892A1 (en) * 2020-03-19 2021-09-23 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
WO2022026409A1 (en) * 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency

Also Published As

Publication number Publication date
WO2023215851A2 (en) 2023-11-09

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