SA518400146B1 - rAAV عـمـلـيـة تـصـنـيـع قـابـل لـلـتوسـع والـتـقـلـص لأقـصـى درجـة أسـاسـهـا عـمـود - Google Patents

rAAV عـمـلـيـة تـصـنـيـع قـابـل لـلـتوسـع والـتـقـلـص لأقـصـى درجـة أسـاسـهـا عـمـود Download PDF

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Publication number
SA518400146B1
SA518400146B1 SA518400146A SA518400146A SA518400146B1 SA 518400146 B1 SA518400146 B1 SA 518400146B1 SA 518400146 A SA518400146 A SA 518400146A SA 518400146 A SA518400146 A SA 518400146A SA 518400146 B1 SA518400146 B1 SA 518400146B1
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SA
Saudi Arabia
Prior art keywords
column
product
adeno
aav
particles
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SA518400146A
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Arabic (ar)
English (en)
Inventor
كو جوانج
فراسر وريت جون
لو لين
اوه يونجهون
Original Assignee
.سبارك ثيرابيوتكس، انك
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Application filed by .سبارك ثيرابيوتكس، انك filed Critical .سبارك ثيرابيوتكس، انك
Publication of SA518400146B1 publication Critical patent/SA518400146B1/ar

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • 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
    • 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, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
    • 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, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
    • 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 and B01D15/30 - B01D15/36, e.g. affinity, ligand exchange or chiral chromatography
    • 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; 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
    • 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
    • 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)
  • 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)
SA518400146A 2016-03-31 2018-09-30 rAAV عـمـلـيـة تـصـنـيـع قـابـل لـلـتوسـع والـتـقـلـص لأقـصـى درجـة أسـاسـهـا عـمـود SA518400146B1 (ar)

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
SA518400146B1 true SA518400146B1 (ar) 2021-10-19

Family

ID=59966426

Family Applications (1)

Application Number Title Priority Date Filing Date
SA518400146A SA518400146B1 (ar) 2016-03-31 2018-09-30 rAAV عـمـلـيـة تـصـنـيـع قـابـل لـلـتوسـع والـتـقـلـص لأقـصـى درجـة أسـاسـهـا عـمـود

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)

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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

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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
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US5981716A (en) * 1995-06-07 1999-11-09 Gruppo Lepettit, S.P.A. Process for the purification of proteins
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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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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
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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
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
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
SG11201808354PA (en) 2018-10-30
JP2019509745A (ja) 2019-04-11
PH12018502104B1 (en) 2024-03-27
WO2017173283A1 (en) 2017-10-05
KR20190006951A (ko) 2019-01-21

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