KR20250004745A - 개선된 rAAV 역가를 위한 HEK293 현탁 플랫폼의 최적화 - Google Patents

개선된 rAAV 역가를 위한 HEK293 현탁 플랫폼의 최적화 Download PDF

Info

Publication number
KR20250004745A
KR20250004745A KR1020247037355A KR20247037355A KR20250004745A KR 20250004745 A KR20250004745 A KR 20250004745A KR 1020247037355 A KR1020247037355 A KR 1020247037355A KR 20247037355 A KR20247037355 A KR 20247037355A KR 20250004745 A KR20250004745 A KR 20250004745A
Authority
KR
South Korea
Prior art keywords
plasmid
cells
aav
raav
rep
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.)
Pending
Application number
KR1020247037355A
Other languages
English (en)
Korean (ko)
Inventor
알리 아가자니레파
Original Assignee
애드베룸 바이오테크놀로지스, 인코포레이티드
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 애드베룸 바이오테크놀로지스, 인코포레이티드 filed Critical 애드베룸 바이오테크놀로지스, 인코포레이티드
Publication of KR20250004745A publication Critical patent/KR20250004745A/ko
Pending legal-status Critical Current

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
    • 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
    • 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
    • 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
    • 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

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020247037355A 2022-04-11 2023-04-07 개선된 rAAV 역가를 위한 HEK293 현탁 플랫폼의 최적화 Pending KR20250004745A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263329856P 2022-04-11 2022-04-11
US63/329,856 2022-04-11
US202263331438P 2022-04-15 2022-04-15
US63/331,438 2022-04-15
PCT/US2023/017838 WO2023200679A1 (en) 2022-04-11 2023-04-07 OPTIMIZATION OF HEK293 SUSPENSION PLATFORM FOR IMPROVED rAAV TITERS

Publications (1)

Publication Number Publication Date
KR20250004745A true KR20250004745A (ko) 2025-01-08

Family

ID=88330208

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247037355A Pending KR20250004745A (ko) 2022-04-11 2023-04-07 개선된 rAAV 역가를 위한 HEK293 현탁 플랫폼의 최적화

Country Status (9)

Country Link
US (1) US20250250547A1 (https=)
EP (1) EP4508198A4 (https=)
JP (1) JP2025512382A (https=)
KR (1) KR20250004745A (https=)
CN (1) CN119053692A (https=)
AU (1) AU2023254746A1 (https=)
CA (1) CA3245930A1 (https=)
MX (1) MX2024012608A (https=)
WO (1) WO2023200679A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4683041A1 (en) 2024-02-29 2026-01-21 LG Energy Solution, Ltd. Method for regenerating cathode active material, and regenerated cathode active material prepared thereby

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026013235A1 (en) * 2024-07-11 2026-01-15 Ferring International Center S.A. Methods of producing recombinant adenovirus vectors

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7208315B2 (en) * 2000-01-05 2007-04-24 Fred Hutchinson Cancer Research Center Compositions and methods for efficient AAV vector production
US20140315294A1 (en) * 2011-11-24 2014-10-23 Genethon Scalable lentiviral vector production system compatible with industrial pharmaceutical applications
CA3130108A1 (en) * 2019-02-22 2020-08-27 Life Technologies Corporation Suspension system for adeno associated virus production

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4683041A1 (en) 2024-02-29 2026-01-21 LG Energy Solution, Ltd. Method for regenerating cathode active material, and regenerated cathode active material prepared thereby

Also Published As

Publication number Publication date
EP4508198A1 (en) 2025-02-19
EP4508198A4 (en) 2026-04-22
CA3245930A1 (en) 2023-10-19
US20250250547A1 (en) 2025-08-07
WO2023200679A1 (en) 2023-10-19
JP2025512382A (ja) 2025-04-17
CN119053692A (zh) 2024-11-29
MX2024012608A (es) 2024-11-08
AU2023254746A1 (en) 2024-10-10

Similar Documents

Publication Publication Date Title
EP4349852A1 (en) Recombinant adeno-associated virus having variant capsid, and application thereof
KR102373765B1 (ko) 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법
EP3393522B1 (en) Improved hybrid dual recombinant aav vector systems for gene therapy
CN115103710A (zh) 用于治疗遗传性听力损失的腺相关病毒(aav)系统
WO2011122950A1 (en) Monomeric duplex aav vectors
US20250250547A1 (en) OPTIMIZATION OF HEK293 SUSPENSION PLATFORM FOR IMPROVED rAAV TITERS
JP7785389B2 (ja) 新規なイントロン断片
WO2023278305A1 (en) Methods and compositions for tau reduction gene therapy
IL323167A (en) Method for producing recombinant AAV particle preparations
WO2024013239A1 (en) Method for producing recombinant aav particles
CN117736274A (zh) 一种腺相关病毒衣壳蛋白及其用途
US20260103729A1 (en) rAAV PRODUCTION IN INSECT CELLS
WO2026080176A1 (en) Scfv-and fab-modified aav capsids and uses thereof
CN118146317A (zh) 一种腺相关病毒衣壳蛋白及其用途
CN119101132A (zh) 靶向视网膜的aav衣壳蛋白变体及其用途
BR112022024345B1 (pt) Polinucleotídeo isolado, vetor, célula procariótica in vitro, método para produzir uma partícula de virus recombinante, partícula de virus recombinante adenoassociado e uso
BR122025008690A2 (pt) Polinucleotídeo, vetor, célula in vitro, método para produzir uma partícula de virus recombinante, partícula de virus recombinante e uso

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000