CA3068622A1 - Aav vector column purification methods - Google Patents

Aav vector column purification methods Download PDF

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Publication number
CA3068622A1
CA3068622A1 CA3068622A CA3068622A CA3068622A1 CA 3068622 A1 CA3068622 A1 CA 3068622A1 CA 3068622 A CA3068622 A CA 3068622A CA 3068622 A CA3068622 A CA 3068622A CA 3068622 A1 CA3068622 A1 CA 3068622A1
Authority
CA
Canada
Prior art keywords
column
column eluate
vector particles
produce
raav vector
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
CA3068622A
Other languages
English (en)
French (fr)
Inventor
Younghoon Oh
Guang Qu
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.)
Spark Therapeutics Inc
Original Assignee
Spark Therapeutics 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 Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of CA3068622A1 publication Critical patent/CA3068622A1/en
Pending legal-status Critical Current

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Classifications

    • 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
    • B01D15/362Cation-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
    • B01D15/26Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/34Size-selective separation, e.g. size-exclusion chromatography; Gel filtration; Permeation
    • 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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Treatment Of Liquids With Adsorbents In General (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
CA3068622A 2017-06-30 2018-06-29 Aav vector column purification methods Pending CA3068622A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762527633P 2017-06-30 2017-06-30
US62/527,633 2017-06-30
US201762531744P 2017-07-12 2017-07-12
US62/531,744 2017-07-12
US201762567905P 2017-10-04 2017-10-04
US62/567,905 2017-10-04
PCT/US2018/040430 WO2019006390A1 (en) 2017-06-30 2018-06-29 AAV VECTOR COLUMN PURIFICATION METHOD

Publications (1)

Publication Number Publication Date
CA3068622A1 true CA3068622A1 (en) 2019-01-03

Family

ID=64742749

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3068622A Pending CA3068622A1 (en) 2017-06-30 2018-06-29 Aav vector column purification methods

Country Status (16)

Country Link
US (1) US20210079422A1 (enExample)
EP (1) EP3658250A4 (enExample)
JP (3) JP2020526190A (enExample)
KR (2) KR102669561B1 (enExample)
CN (1) CN111032176A (enExample)
AU (1) AU2018291023B2 (enExample)
CA (1) CA3068622A1 (enExample)
CL (1) CL2019003915A1 (enExample)
CO (1) CO2020000911A2 (enExample)
IL (1) IL271745A (enExample)
MX (2) MX2020000216A (enExample)
MY (1) MY207534A (enExample)
PE (2) PE20251545A1 (enExample)
PH (1) PH12020500044A1 (enExample)
SG (1) SG11201913157RA (enExample)
WO (1) WO2019006390A1 (enExample)

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US12565645B2 (en) * 2019-04-11 2026-03-03 Regenxbio Inc. Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions
US12403164B2 (en) 2019-09-30 2025-09-02 Bioverativ Therapeutics Inc. Lentiviral vector formulations
AU2021262609A1 (en) 2020-05-01 2022-12-22 Kashiv Biosciences, Llc An improved process of purification of protein
EP3919613B1 (en) * 2020-06-05 2024-08-07 Sartorius BIA Separations d.o.o. Enhanced purification of adeno-associated virus to more effectively remove contaminating dna
KR20230074703A (ko) * 2020-06-24 2023-05-31 바이오버라티브 테라퓨틱스 인크. 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법
US20220033782A1 (en) * 2020-07-29 2022-02-03 Pall Corporation Adenovirus-associated viruses separation method
EP4229204A1 (en) 2020-10-15 2023-08-23 F. Hoffmann-La Roche AG Nucleic acid constructs for simultaneous gene activation
KR20230085929A (ko) 2020-10-15 2023-06-14 에프. 호프만-라 로슈 아게 Va rna 전사를 위한 핵산 구조체
JP7767029B2 (ja) * 2021-05-28 2025-11-11 ダイセン・メンブレン・システムズ株式会社 細胞外小胞の分離精製方法
JP7716233B2 (ja) * 2021-05-28 2025-07-31 ダイセン・メンブレン・システムズ株式会社 微小有用物質の分離精製方法と分離精製装置
PE20240359A1 (es) * 2021-06-11 2024-02-27 Spark Therapeutics Inc Metodos de purificacion de columna vectorial aav
AU2022358612A1 (en) * 2021-09-28 2024-04-11 Kashiv Biosciences, Llc An improved process of purification of fusion protein
CN114250222A (zh) * 2021-12-08 2022-03-29 苏州博腾生物制药有限公司 一种从动物组织中提取aav dna的方法
GB202117844D0 (en) * 2021-12-09 2022-01-26 Oxford Biomedica Ltd Purification method
JP2023141423A (ja) * 2022-03-24 2023-10-05 国立大学法人 東京大学 ウイルスの精製方法
WO2023198685A1 (en) 2022-04-13 2023-10-19 F. Hoffmann-La Roche Ag Method for determining aav genomes
CN119256220A (zh) 2022-05-23 2025-01-03 豪夫迈·罗氏有限公司 用于区分aav颗粒血清型和aav颗粒荷载状态的基于拉曼的方法
EP4532737A1 (en) 2022-06-03 2025-04-09 F. Hoffmann-La Roche AG Method for producing recombinant aav particles
KR20250036743A (ko) 2022-07-14 2025-03-14 에프. 호프만-라 로슈 아게 재조합 aav 입자를 생산하는 방법
JPWO2024024337A1 (enExample) * 2022-07-28 2024-02-01
EP4744752A1 (en) 2022-09-12 2026-05-20 Hoffmann La Roche METHOD FOR SEPARING SOLID AND EMPTY AAV PARTICLES
EP4385618A1 (en) 2022-12-16 2024-06-19 Chiral Technologies Europe SAS Composite material for bioseparations
IL322001A (en) 2023-02-07 2025-09-01 Hoffmann La Roche Method for detecting antibodies to anti-AAV particles
EP4683676A1 (en) 2023-03-21 2026-01-28 F. Hoffmann-La Roche AG Method for the production of recombinant aav particle preparations
KR20250172636A (ko) * 2023-04-12 2025-12-09 씨티바 바이오프로세스 알&디 에이비 크로마토그래피 시스템, 이의 용도, 및 아데노-연관 캡시드를 분리하는 방법
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles
TW202603164A (zh) * 2024-04-07 2026-01-16 美商星火治療公司 用於腺相關病毒殼體變異體純化之組成物及方法
WO2025252480A1 (en) 2024-06-07 2025-12-11 F. Hoffmann-La Roche Ag Method for purifying plasmid dna
WO2026022064A1 (en) 2024-07-22 2026-01-29 F. Hoffmann-La Roche Ag Novel aav rep orfs and rep polypeptides
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles

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DK1944362T3 (en) * 1997-09-05 2016-01-25 Genzyme Corp Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer
US6593123B1 (en) * 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US7261544B2 (en) * 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
KR101812813B1 (ko) * 2009-06-16 2017-12-27 젠자임 코포레이션 재조합 aav 벡터에 대한 개선된 정제 방법
ES2680915T3 (es) * 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
MX2018006682A (es) * 2015-12-01 2018-09-26 Spark Therapeutics Inc Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico.
US11015173B2 (en) * 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
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ES2896080T3 (es) * 2016-03-31 2022-02-23 Spark Therapeutics Inc Procedimiento en columna totalmente escalable de preparación de VAAr

Also Published As

Publication number Publication date
WO2019006390A1 (en) 2019-01-03
EP3658250A4 (en) 2021-10-27
JP2023029832A (ja) 2023-03-07
JP2025038906A (ja) 2025-03-19
CL2019003915A1 (es) 2020-06-19
PE20251545A1 (es) 2025-06-05
KR102879640B1 (ko) 2025-10-30
MX2024007103A (es) 2024-06-24
MY207534A (en) 2025-03-03
KR20240093848A (ko) 2024-06-24
BR112019028299A2 (pt) 2020-07-14
CO2020000911A2 (es) 2020-06-19
MX2020000216A (es) 2020-09-03
CN111032176A (zh) 2020-04-17
AU2018291023B2 (en) 2023-11-02
EP3658250A1 (en) 2020-06-03
KR20200040749A (ko) 2020-04-20
PH12020500044A1 (en) 2020-09-14
JP2020526190A (ja) 2020-08-31
US20210079422A1 (en) 2021-03-18
KR102669561B1 (ko) 2024-05-24
SG11201913157RA (en) 2020-01-30
IL271745A (en) 2020-02-27
RU2020103743A (ru) 2021-07-30
RU2020103743A3 (enExample) 2021-09-30
AU2018291023A1 (en) 2020-02-06
PE20200737A1 (es) 2020-07-23

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