DK3436051T3 - Søjlebaseret fuldt skalérbar raav-fremstillingsfremgangsmåde. - Google Patents

Søjlebaseret fuldt skalérbar raav-fremstillingsfremgangsmåde. Download PDF

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Publication number
DK3436051T3
DK3436051T3 DK17776783.7T DK17776783T DK3436051T3 DK 3436051 T3 DK3436051 T3 DK 3436051T3 DK 17776783 T DK17776783 T DK 17776783T DK 3436051 T3 DK3436051 T3 DK 3436051T3
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DK
Denmark
Prior art keywords
pillar
fully scalable
based fully
manufacturing
raav manufacturing
Prior art date
Application number
DK17776783.7T
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English (en)
Inventor
Younghoon Oh
John Fraser Wright
Guang Qu
Lin Lu
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Spark Therapeutics Inc
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Publication of DK3436051T3 publication Critical patent/DK3436051T3/da

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • B01D15/362Cation-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
    • B01D15/36Selective 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
    • B01D15/26Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/38Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 and B01D15/30 - B01D15/36, e.g. affinity, ligand exchange or chiral chromatography
    • B01D15/3804Affinity chromatography
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D61/00Processes of separation using semi-permeable membranes, e.g. dialysis, osmosis or ultrafiltration; Apparatus, accessories or auxiliary operations specially adapted therefor
    • B01D61/14Ultrafiltration; Microfiltration
    • B01D61/145Ultrafiltration
    • 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
    • C12N1/00Microorganisms; Compositions thereof; Processes of propagating, maintaining or preserving microorganisms or compositions thereof; Processes of preparing or isolating a composition containing a microorganism; Culture media therefor
    • C12N1/06Lysis of microorganisms
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D2315/00Details relating to the membrane module operation
    • B01D2315/16Diafiltration
    • 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
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • 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
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Water Supply & Treatment (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
DK17776783.7T 2016-03-31 2017-03-31 Søjlebaseret fuldt skalérbar raav-fremstillingsfremgangsmåde. DK3436051T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662316252P 2016-03-31 2016-03-31
PCT/US2017/025396 WO2017173283A1 (en) 2016-03-31 2017-03-31 Column-based fully scalable raav manufacturing process

Publications (1)

Publication Number Publication Date
DK3436051T3 true DK3436051T3 (da) 2021-11-01

Family

ID=59966426

Family Applications (1)

Application Number Title Priority Date Filing Date
DK17776783.7T DK3436051T3 (da) 2016-03-31 2017-03-31 Søjlebaseret fuldt skalérbar raav-fremstillingsfremgangsmåde.

Country Status (24)

Country Link
US (1) US11702639B2 (da)
EP (1) EP3436051B1 (da)
JP (2) JP7364306B2 (da)
KR (1) KR102405250B1 (da)
CN (1) CN109152821A (da)
AU (1) AU2017240721B2 (da)
BR (1) BR112018070256A2 (da)
CA (1) CA3019133A1 (da)
CL (1) CL2018002754A1 (da)
CO (1) CO2018011151A2 (da)
DK (1) DK3436051T3 (da)
ES (1) ES2896080T3 (da)
HU (1) HUE056854T2 (da)
IL (1) IL261970B2 (da)
LT (1) LT3436051T (da)
MY (1) MY195391A (da)
PE (1) PE20190434A1 (da)
PH (1) PH12018502104B1 (da)
PL (1) PL3436051T3 (da)
PT (1) PT3436051T (da)
RU (1) RU2754467C2 (da)
SA (1) SA518400146B1 (da)
SG (1) SG11201808354PA (da)
WO (1) WO2017173283A1 (da)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
LT3436051T (lt) * 2016-03-31 2021-11-25 Spark Therapeutics, Inc. Kolonėlėmis pagrįstas visiškai keičiamo masto raav gavimo būdas
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3504227A1 (en) 2016-08-29 2019-07-03 Wayne State University Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
PE20200737A1 (es) * 2017-06-30 2020-07-23 Spark Therapeutics Inc Metodos de purificacion de columna de vector aav
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
WO2019209632A1 (en) 2018-04-24 2019-10-31 Merck Sharp & Dohme Corp. Scalable chromatography process for purification of human cytomegalovirus
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
EP3801638A1 (en) 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN114173827B (zh) * 2019-06-28 2025-01-28 武田药品工业株式会社 腺相关病毒纯化方法
US20230219075A1 (en) * 2020-04-06 2023-07-13 Brown University Formulations and processes for treating water from impacted sources
JPWO2022045055A1 (da) * 2020-08-24 2022-03-03
WO2023077086A1 (en) * 2021-10-29 2023-05-04 Homology Medicines, Inc. Methods and compositions for the purification of adeno-associated virus

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AU8200191A (en) 1990-07-09 1992-02-04 United States of America, as represented by the Secretary, U.S. Department of Commerce, The High efficiency packaging of mutant adeno-associated virus using amber suppressions
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DK1009808T3 (da) * 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
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ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
KR20180000341A (ko) * 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 aav 벡터에 대한 개선된 정제 방법
EP2325194A1 (en) * 2009-11-24 2011-05-25 Glycotope GmbH Process for the purification of glycoproteins
WO2011094198A1 (en) * 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
CN103842501B (zh) 2011-10-05 2017-12-08 莫尔梅德股份有限公司 病毒载体纯化系统
LT3436051T (lt) * 2016-03-31 2021-11-25 Spark Therapeutics, Inc. Kolonėlėmis pagrįstas visiškai keičiamo masto raav gavimo būdas

Also Published As

Publication number Publication date
JP2022120011A (ja) 2022-08-17
WO2017173283A1 (en) 2017-10-05
ES2896080T3 (es) 2022-02-23
US11702639B2 (en) 2023-07-18
CO2018011151A2 (es) 2019-04-30
CN109152821A (zh) 2019-01-04
AU2017240721A1 (en) 2018-10-11
IL261970B2 (en) 2025-10-01
PE20190434A1 (es) 2019-03-21
AU2017240721B2 (en) 2023-10-12
PL3436051T3 (pl) 2022-02-07
IL261970A (en) 2018-10-31
JP2019509745A (ja) 2019-04-11
PT3436051T (pt) 2021-11-02
KR20190006951A (ko) 2019-01-21
RU2018138164A3 (da) 2020-06-16
JP7364306B2 (ja) 2023-10-18
PH12018502104A1 (en) 2019-07-29
US20200299650A1 (en) 2020-09-24
IL261970B1 (en) 2025-06-01
EP3436051B1 (en) 2021-07-28
EP3436051A1 (en) 2019-02-06
RU2018138164A (ru) 2020-04-30
CA3019133A1 (en) 2017-10-05
CL2018002754A1 (es) 2019-02-01
SG11201808354PA (en) 2018-10-30
RU2754467C2 (ru) 2021-09-02
NZ747126A (en) 2025-05-02
SA518400146B1 (ar) 2021-10-19
KR102405250B1 (ko) 2022-06-02
PH12018502104B1 (en) 2024-03-27
HUE056854T2 (hu) 2022-03-28
LT3436051T (lt) 2021-11-25
BR112018070256A2 (pt) 2019-01-29
MY195391A (en) 2023-01-18
JP7659523B2 (ja) 2025-04-09
EP3436051A4 (en) 2019-10-16

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