BR112018070256A2 - processo de fabricação de raav totalmente escalável à base de colunas - Google Patents

processo de fabricação de raav totalmente escalável à base de colunas

Info

Publication number
BR112018070256A2
BR112018070256A2 BR112018070256A BR112018070256A BR112018070256A2 BR 112018070256 A2 BR112018070256 A2 BR 112018070256A2 BR 112018070256 A BR112018070256 A BR 112018070256A BR 112018070256 A BR112018070256 A BR 112018070256A BR 112018070256 A2 BR112018070256 A2 BR 112018070256A2
Authority
BR
Brazil
Prior art keywords
manufacturing process
fully scalable
scalable column
based raav
raav manufacturing
Prior art date
Application number
BR112018070256A
Other languages
English (en)
Inventor
Qu Guang
Fraser Wright John
Lu Lin
Oh Younghoon
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 BR112018070256A2 publication Critical patent/BR112018070256A2/pt

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    • 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
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/34—Size selective separation, e.g. size exclusion chromatography, gel filtration, permeation
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/36—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction
    • B01D15/361—Ion-exchange
    • B01D15/362—Cation-exchange
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/36—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction
    • B01D15/361—Ion-exchange
    • B01D15/363—Anion-exchange
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/38—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 - B01D15/36
    • B01D15/3804—Affinity chromatography
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D61/00—Processes of separation using semi-permeable membranes, e.g. dialysis, osmosis or ultrafiltration; Apparatus, accessories or auxiliary operations specially adapted therefor
    • B01D61/14—Ultrafiltration; Microfiltration
    • B01D61/145—Ultrafiltration
    • 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
    • C12N1/00—Microorganisms, 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
    • C12N1/06—Lysis of microorganisms
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D2315/00—Details relating to the membrane module operation
    • B01D2315/16—Diafiltration
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • 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

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Water Supply & Treatment (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Mycology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

de acordo com a invenção, são aqui fornecidos métodos para purificar partículas de vetor adeno-associadas recombinantes (raav).
BR112018070256A 2016-03-31 2017-03-31 processo de fabricação de raav totalmente escalável à base de colunas BR112018070256A2 (pt)

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
BR112018070256A2 true BR112018070256A2 (pt) 2019-01-29

Family

ID=59966426

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018070256A BR112018070256A2 (pt) 2016-03-31 2017-03-31 processo de fabricação de raav totalmente escalável à base de colunas

Country Status (24)

Country Link
US (1) US11702639B2 (pt)
EP (1) EP3436051B1 (pt)
JP (2) JP7364306B2 (pt)
KR (1) KR102405250B1 (pt)
CN (1) CN109152821A (pt)
AU (1) AU2017240721B2 (pt)
BR (1) BR112018070256A2 (pt)
CA (1) CA3019133A1 (pt)
CL (1) CL2018002754A1 (pt)
CO (1) CO2018011151A2 (pt)
DK (1) DK3436051T3 (pt)
ES (1) ES2896080T3 (pt)
HU (1) HUE056854T2 (pt)
IL (1) IL261970A (pt)
LT (1) LT3436051T (pt)
MY (1) MY195391A (pt)
PE (1) PE20190434A1 (pt)
PH (1) PH12018502104A1 (pt)
PL (1) PL3436051T3 (pt)
PT (1) PT3436051T (pt)
RU (1) RU2754467C2 (pt)
SA (1) SA518400146B1 (pt)
SG (1) SG11201808354PA (pt)
WO (1) WO2017173283A1 (pt)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
DK3218386T3 (da) 2014-11-14 2021-06-07 Voyager Therapeutics Inc Modulatorisk polynukleotid
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc modulatory polynucleotides
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3068622A1 (en) * 2017-06-30 2019-01-03 Spark Therapeutics, Inc. Aav vector column purification methods
CA3082136A1 (en) * 2017-11-08 2019-05-16 Avexis, Inc. Means and method for preparing viral vectors and uses of same
US20210047626A1 (en) * 2018-04-24 2021-02-18 Merck Sharp & Dohme Corp. Scalable chromatography process for 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
US20230183658A1 (en) * 2021-10-29 2023-06-15 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus

Family Cites Families (17)

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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
CA2115742A1 (en) 1991-08-20 1993-03-04 Ronald G. Crystal Adenovirus mediated transfer of genes to the gastrointestinal tract
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
CA2995542A1 (en) * 1997-09-05 1999-03-11 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
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
SI2277996T1 (sl) 2003-05-21 2014-12-31 Genzyme Corporation Postopki za izdelavo pripravkov rekombinantnih AAV virionov v bistvu brez praznih kapsid
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use
BRPI1015176B1 (pt) * 2009-06-16 2019-10-29 Genzyme Corp método para isolar uma população de partículas de vírus adenoassociado recombinante (raav) de impurezas de processo em uma corrente de alimentação
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
PT3436051T (pt) 2021-11-02
JP2022120011A (ja) 2022-08-17
AU2017240721A1 (en) 2018-10-11
RU2018138164A3 (pt) 2020-06-16
US20200299650A1 (en) 2020-09-24
KR102405250B1 (ko) 2022-06-02
CL2018002754A1 (es) 2019-02-01
EP3436051A1 (en) 2019-02-06
DK3436051T3 (da) 2021-11-01
EP3436051B1 (en) 2021-07-28
WO2017173283A1 (en) 2017-10-05
SA518400146B1 (ar) 2021-10-19
CA3019133A1 (en) 2017-10-05
PL3436051T3 (pl) 2022-02-07
ES2896080T3 (es) 2022-02-23
CO2018011151A2 (es) 2019-04-30
KR20190006951A (ko) 2019-01-21
CN109152821A (zh) 2019-01-04
JP2019509745A (ja) 2019-04-11
AU2017240721B2 (en) 2023-10-12
HUE056854T2 (hu) 2022-03-28
MY195391A (en) 2023-01-18
RU2754467C2 (ru) 2021-09-02
JP7364306B2 (ja) 2023-10-18
LT3436051T (lt) 2021-11-25
IL261970A (en) 2018-10-31
EP3436051A4 (en) 2019-10-16
PE20190434A1 (es) 2019-03-21
PH12018502104A1 (en) 2019-07-29
US11702639B2 (en) 2023-07-18
SG11201808354PA (en) 2018-10-30
RU2018138164A (ru) 2020-04-30

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