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
Other languages
English (en)
Inventor
Younghoon Oh
John Fraser Wright
Guang Qu
Lin Lu
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
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Publication of DK3436051T3 publication Critical patent/DK3436051T3/da

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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
    • 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
    • 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
    • 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
    • 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 - B01D15/36
    • 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, e.g. protozoa; 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
    • 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
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Water Supply & Treatment (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Mycology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Tropical Medicine & Parasitology (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)
  • Saccharide Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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) IL261970A (da)
LT (1) LT3436051T (da)
MY (1) MY195391A (da)
PE (1) PE20190434A1 (da)
PH (1) PH12018502104A1 (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)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
SG10202001102XA (en) 2014-11-14 2020-03-30 Voyager Therapeutics Inc Modulatory polynucleotides
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
CN110214187B (zh) 2016-05-18 2024-01-30 沃雅戈治疗公司 调节性多核苷酸
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018291023B2 (en) * 2017-06-30 2023-11-02 Spark Therapeutics, Inc. AAV vector column purification methods
EP3707264A1 (en) * 2017-11-08 2020-09-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
EP3784276A4 (en) * 2018-04-24 2022-03-16 Merck Sharp & Dohme Corp. SCALABLE CHROMATOGRAPHY METHOD FOR THE PURIFICATION OF HUMAN CYTOMEGALOVIRUS
WO2022045055A1 (ja) * 2020-08-24 2022-03-03 タカラバイオ株式会社 pHの違いによる非エンベロープウイルスベクター粒子の調製方法
WO2023077086A1 (en) * 2021-10-29 2023-05-04 Homology Medicines, Inc. Methods and compositions for the purification of adeno-associated virus

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1992001070A1 (en) 1990-07-09 1992-01-23 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
ATE237694T1 (de) 1991-08-20 2003-05-15 Us Gov Health & Human Serv Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
US5981716A (en) * 1995-06-07 1999-11-09 Gruppo Lepettit, S.P.A. Process for the purification of proteins
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
ES2557997T3 (es) * 1997-09-05 2016-02-01 Genzyme Corporation Métodos para generar preparados de vectores AAV de título elevado sin virus auxiliar
WO2001083797A2 (en) 2000-04-28 2001-11-08 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
PT2277996E (pt) * 2003-05-21 2014-12-16 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use
HUE054940T2 (hu) * 2009-06-16 2021-10-28 Genzyme Corp Javított eljárások rekombináns AAV-vektorok tisztítására
EP2325194A1 (en) * 2009-11-24 2011-05-25 Glycotope GmbH Process for the purification of glycoproteins
EP2529020B1 (en) * 2010-01-28 2018-04-25 The Children's Hospital of Philadelphia A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
BR112014008225A2 (pt) * 2011-10-05 2017-04-11 Molmed Spa sistemas de purificação de vetores virais

Also Published As

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

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