SG11201808354PA - Column-based fully scalable raav manufacturing process - Google Patents

Column-based fully scalable raav manufacturing process

Info

Publication number
SG11201808354PA
SG11201808354PA SG11201808354PA SG11201808354PA SG11201808354PA SG 11201808354P A SG11201808354P A SG 11201808354PA SG 11201808354P A SG11201808354P A SG 11201808354PA SG 11201808354P A SG11201808354P A SG 11201808354PA SG 11201808354P A SG11201808354P A SG 11201808354PA
Authority
SG
Singapore
Prior art keywords
international
rule
aiex
avb
pennsylvania
Prior art date
Application number
SG11201808354PA
Other languages
English (en)
Inventor
Guang Qu
Younghoon Oh
Lin Lu
John Fraser Wright
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 SG11201808354PA publication Critical patent/SG11201808354PA/en

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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
    • 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)
    • 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
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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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)
  • 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)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Water Supply & Treatment (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (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)
SG11201808354PA 2016-03-31 2017-03-31 Column-based fully scalable raav manufacturing process SG11201808354PA (en)

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
SG11201808354PA true SG11201808354PA (en) 2018-10-30

Family

ID=59966426

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201808354PA SG11201808354PA (en) 2016-03-31 2017-03-31 Column-based fully scalable raav manufacturing process

Country Status (23)

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

Families Citing this family (18)

* 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
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
ES2896080T3 (es) * 2016-03-31 2022-02-23 Spark Therapeutics Inc Procedimiento en columna totalmente escalable de preparación de VAAr
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
KR102877327B1 (ko) 2016-08-29 2025-10-27 웨인 스테이트 유니버시티 개선된 광 민감성을 갖는 채널옵신 변이체에서의 돌연변이의 확인 및 이의 용도
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
SG11201913157RA (en) * 2017-06-30 2020-01-30 Spark Therapeutics Inc Aav vector column purification methods
CN120505370A (zh) * 2017-11-08 2025-08-19 诺华股份有限公司 制备病毒载体的手段和方法及其用途
EP3784276A4 (en) * 2018-04-24 2022-03-16 Merck Sharp & Dohme Corp. SCALABLE CHROMATOGRAPHY PROCEDURE FOR PURIFICATION OF HUMAN CYTOMEGALOVIRUS
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US12228571B2 (en) 2018-06-08 2025-02-18 Novartis Ag Cell-based assay for measuring drug product potency
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
US20220267796A1 (en) * 2019-06-28 2022-08-25 Takeda Pharmaceutical Company Limited Adeno-associated virus purification methods
WO2021207132A1 (en) * 2020-04-06 2021-10-14 Brown University Formulations and processes for treating water from impacted sources
JPWO2022045055A1 (enExample) * 2020-08-24 2022-03-03
US20230183659A1 (en) * 2021-10-29 2023-06-15 Homology Medicines, Inc. Methods and compositions for the purification of adeno-associated virus

Family Cites Families (18)

* 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
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
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
WO1993003769A1 (en) 1991-08-20 1993-03-04 THE UNITED STATES OF AMERICA, represented by THE SECRETARY, DEPARTEMENT OF HEALTH AND HUMAN SERVICES 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
DK1944362T3 (en) * 1997-09-05 2016-01-25 Genzyme Corp Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer
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
US7261544B2 (en) * 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida Raav vector compositions having tyrosine-modified capsid proteins and methods for use
KR101812813B1 (ko) * 2009-06-16 2017-12-27 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
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
US20140248695A1 (en) 2011-10-05 2014-09-04 MolMed, S.p.A. Viral Vectors Purification System
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
CL2018002754A1 (es) 2019-02-01
US20200299650A1 (en) 2020-09-24
HUE056854T2 (hu) 2022-03-28
EP3436051A4 (en) 2019-10-16
NZ747126A (en) 2025-05-02
ES2896080T3 (es) 2022-02-23
JP7364306B2 (ja) 2023-10-18
EP3436051A1 (en) 2019-02-06
RU2018138164A (ru) 2020-04-30
CA3019133A1 (en) 2017-10-05
LT3436051T (lt) 2021-11-25
RU2754467C2 (ru) 2021-09-02
CO2018011151A2 (es) 2019-04-30
SA518400146B1 (ar) 2021-10-19
US11702639B2 (en) 2023-07-18
PE20190434A1 (es) 2019-03-21
KR102405250B1 (ko) 2022-06-02
IL261970B1 (en) 2025-06-01
CN109152821A (zh) 2019-01-04
RU2018138164A3 (enExample) 2020-06-16
AU2017240721B2 (en) 2023-10-12
PL3436051T3 (pl) 2022-02-07
MY195391A (en) 2023-01-18
AU2017240721A1 (en) 2018-10-11
JP7659523B2 (ja) 2025-04-09
IL261970B2 (en) 2025-10-01
IL261970A (en) 2018-10-31
DK3436051T3 (da) 2021-11-01
PT3436051T (pt) 2021-11-02
JP2022120011A (ja) 2022-08-17
PH12018502104A1 (en) 2019-07-29
EP3436051B1 (en) 2021-07-28
BR112018070256A2 (pt) 2019-01-29
JP2019509745A (ja) 2019-04-11
PH12018502104B1 (en) 2024-03-27
WO2017173283A1 (en) 2017-10-05
KR20190006951A (ko) 2019-01-21

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