JP2024525142A - Aavベクターカラム精製方法 - Google Patents

Aavベクターカラム精製方法 Download PDF

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Publication number
JP2024525142A
JP2024525142A JP2023576326A JP2023576326A JP2024525142A JP 2024525142 A JP2024525142 A JP 2024525142A JP 2023576326 A JP2023576326 A JP 2023576326A JP 2023576326 A JP2023576326 A JP 2023576326A JP 2024525142 A JP2024525142 A JP 2024525142A
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JP
Japan
Prior art keywords
particles
column
raav
complete
chromatography medium
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Pending
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JP2023576326A
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English (en)
Japanese (ja)
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JP2024525142A5 (https=
JPWO2022261663A5 (https=
Inventor
オンマル カナル
ヴィジェシュ クマール
ミ ジン
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Spark Therapeutics Inc
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Spark Therapeutics Inc
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Publication of JP2024525142A publication Critical patent/JP2024525142A/ja
Publication of JP2024525142A5 publication Critical patent/JP2024525142A5/ja
Publication of JPWO2022261663A5 publication Critical patent/JPWO2022261663A5/ja
Pending legal-status Critical Current

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    • 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
    • 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
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D15/00Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08Selective adsorption, e.g. chromatography
    • B01D15/26Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/36Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
    • B01D15/361Ion-exchange
    • B01D15/363Anion-exchange
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D15/00Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08Selective adsorption, e.g. chromatography
    • B01D15/42Selective adsorption, e.g. chromatography characterised by the development mode, e.g. by displacement or by elution
    • B01D15/422Displacement mode
    • 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
    • C12N2750/14151Methods of production or purification of viral material

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Analytical Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Treatment Of Liquids With Adsorbents In General (AREA)
JP2023576326A 2021-06-11 2022-06-10 Aavベクターカラム精製方法 Pending JP2024525142A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163209680P 2021-06-11 2021-06-11
US63/209,680 2021-06-11
US202263366094P 2022-06-09 2022-06-09
US63/366,094 2022-06-09
PCT/US2022/072859 WO2022261663A1 (en) 2021-06-11 2022-06-10 Aav vector column purification methods

Publications (3)

Publication Number Publication Date
JP2024525142A true JP2024525142A (ja) 2024-07-10
JP2024525142A5 JP2024525142A5 (https=) 2025-06-17
JPWO2022261663A5 JPWO2022261663A5 (https=) 2025-06-17

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ID=84426294

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023576326A Pending JP2024525142A (ja) 2021-06-11 2022-06-10 Aavベクターカラム精製方法

Country Status (13)

Country Link
US (1) US20240271159A1 (https=)
EP (1) EP4351755A4 (https=)
JP (1) JP2024525142A (https=)
KR (1) KR20240021231A (https=)
AU (1) AU2022289035A1 (https=)
BR (1) BR112023025999A2 (https=)
CA (1) CA3221540A1 (https=)
CL (1) CL2023003646A1 (https=)
CO (1) CO2023018191A2 (https=)
IL (1) IL309070A (https=)
MX (1) MX2023014809A (https=)
PE (1) PE20240359A1 (https=)
WO (1) WO2022261663A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024036251A1 (en) * 2022-08-12 2024-02-15 Ultragenyx Pharmaceutical Inc. Novel anion-exchange chromatography methods for separation of empty from full recombinant adeno-associated virus particles
EP4670812A1 (en) * 2024-06-27 2025-12-31 Sartorius Stedim Biotech GmbH CONTINUOUS POLISHING OF VIRAL CAPSIDES
WO2026064763A1 (en) * 2024-09-23 2026-03-26 Regeneron Pharmaceuticals, Inc. Partitioning anion exchange chromatography for purification of recombinant adeno-associated virus (raav)

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE602004030327D1 (de) * 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
US20120093775A1 (en) * 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
MY207534A (en) * 2017-06-30 2025-03-03 Spark Therapeutics Inc Aav vector column purification methods
US20230183659A1 (en) * 2021-10-29 2023-06-15 Homology Medicines, Inc. Methods and compositions for the purification of adeno-associated virus
EP4602172A1 (en) * 2022-10-11 2025-08-20 MeiraGTx UK II Limited Chromatography methods for purification of aav capsids

Also Published As

Publication number Publication date
IL309070A (en) 2024-02-01
US20240271159A1 (en) 2024-08-15
CO2023018191A2 (es) 2024-01-25
WO2022261663A1 (en) 2022-12-15
EP4351755A4 (en) 2025-04-23
EP4351755A1 (en) 2024-04-17
AU2022289035A1 (en) 2024-01-04
BR112023025999A2 (pt) 2024-02-27
PE20240359A1 (es) 2024-02-27
KR20240021231A (ko) 2024-02-16
CL2023003646A1 (es) 2024-10-25
CA3221540A1 (en) 2022-12-15
MX2023014809A (es) 2024-01-29

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