MX2018006682A - Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico. - Google Patents

Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico.

Info

Publication number
MX2018006682A
MX2018006682A MX2018006682A MX2018006682A MX2018006682A MX 2018006682 A MX2018006682 A MX 2018006682A MX 2018006682 A MX2018006682 A MX 2018006682A MX 2018006682 A MX2018006682 A MX 2018006682A MX 2018006682 A MX2018006682 A MX 2018006682A
Authority
MX
Mexico
Prior art keywords
aav
serum
vector
associated viral
cell culture
Prior art date
Application number
MX2018006682A
Other languages
English (en)
Inventor
Qu Guang
Fraser Wright John
Lu Lin
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 MX2018006682A publication Critical patent/MX2018006682A/es

Links

Classifications

    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/0018Culture media for cell or tissue culture
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0603Embryonic cells ; Embryoid bodies
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0684Cells of the urinary tract or kidneys
    • C12N5/0686Kidney cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/70Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
    • 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
    • C12N2510/00Genetically modified cells
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Reproductive Health (AREA)
  • Gynecology & Obstetrics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)

Abstract

Se dan a conocer métodos y composiciones para transfectar células con plásmidos. En ciertas modalidades, se dan a conocer métodos y composiciones en los cuales la eficiencia de transfección se incrementa significativamente al poner en contacto las células que se transducen con polietilenimina (PEI) que se encuentra libre de ácido nucleico durante el proceso de transfección. También se dan a conocer vectores virales adenoasociados terapéuticamente útiles, generados de acuerdo con los métodos y composiciones dados a conocer.
MX2018006682A 2015-12-01 2016-12-01 Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico. MX2018006682A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562261815P 2015-12-01 2015-12-01
PCT/US2016/064414 WO2017096039A1 (en) 2015-12-01 2016-12-01 Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use

Publications (1)

Publication Number Publication Date
MX2018006682A true MX2018006682A (es) 2018-09-26

Family

ID=58797705

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018006682A MX2018006682A (es) 2015-12-01 2016-12-01 Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico.
MX2023012498A MX2023012498A (es) 2015-12-01 2018-05-31 Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023012498A MX2023012498A (es) 2015-12-01 2018-05-31 Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico.

Country Status (16)

Country Link
US (1) US20190292561A1 (es)
EP (1) EP3384015A4 (es)
JP (2) JP7444521B2 (es)
KR (1) KR20180091863A (es)
CN (1) CN108603174A (es)
AU (2) AU2016362317B2 (es)
BR (1) BR112018011193A2 (es)
CA (1) CA3006309A1 (es)
CO (1) CO2018006699A2 (es)
IL (1) IL259595B2 (es)
MX (2) MX2018006682A (es)
PE (2) PE20181534A1 (es)
PH (1) PH12018501168A1 (es)
RU (1) RU2766583C2 (es)
SG (2) SG11201804400SA (es)
WO (1) WO2017096039A1 (es)

Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
BR112017009497A2 (pt) 2014-11-05 2018-02-06 Voyager Therapeutics, Inc. polinucleotídeos de aadc para o tratamento da doença de parkinson
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
BR112017010087A2 (pt) 2014-11-14 2018-06-05 Voyager Therapeutics, Inc. composições e métodos para tratar esclerose lateral amiotrófica (ela)
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
SG10202107733QA (en) 2015-09-28 2021-09-29 Univ North Carolina Chapel Hill Methods and compositions for antibody-evading virus vectors
EP3384015A4 (en) * 2015-12-01 2019-05-29 Spark Therapeutics, Inc. SCALABLE PROCESS FOR THE PREPARATION OF A RECOMBINANT ADENO ASSOCIATED VIRAL VECTOR IN A SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR THE CLINICAL APPLICATION
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
MX2018014154A (es) 2016-05-18 2019-05-06 Voyager Therapeutics Inc Polinucleotidos moduladores.
JP2019530737A (ja) 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
EP3831281A1 (en) 2016-08-30 2021-06-09 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
AU2018261790A1 (en) 2017-05-05 2019-11-28 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
EP3619308A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS
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
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
SG11202003479TA (en) * 2017-10-18 2020-05-28 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
WO2019113202A1 (en) * 2017-12-05 2019-06-13 Applied Genetic Technologies Corporation Formulation optimization for viral particles
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN108866011A (zh) * 2018-07-31 2018-11-23 深圳生生凡非基因技术有限公司 一种同步包装rAAV的方法
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CN110437317B (zh) * 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
US11746360B2 (en) * 2019-02-11 2023-09-05 Florian M. Wurm Eukaryotic cell transfection systems and related methods
MX2021011468A (es) 2019-03-21 2021-12-15 Vectores de virus adenoasociados recombinantes.
US11414785B2 (en) 2019-08-13 2022-08-16 Waters Technologies Corporation Affinity resins and sample preparation devices based on cartilaginous fish IgNAR derived binding domains
WO2021076925A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CN110894494B (zh) * 2019-11-22 2022-09-27 广西梧州制药(集团)股份有限公司 一种大规模高密度悬浮培养293细胞高产腺病毒的方法
CN110938654A (zh) * 2019-12-11 2020-03-31 和元生物技术(上海)股份有限公司 一种细胞转染试剂及其应用
CN115315518A (zh) * 2020-01-17 2022-11-08 阿斯克肋匹奥生物制药公司 重组aav的生产
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
WO2022043926A1 (en) * 2020-08-31 2022-03-03 Intas Pharmaceuticals Ltd. Process for preparation of recombinant adeno-associated virus particle
KR20230085170A (ko) 2020-10-15 2023-06-13 에프. 호프만-라 로슈 아게 동시 유전자 활성화를 위한 핵산 구조체
IL302046A (en) 2020-10-15 2023-06-01 Hoffmann La Roche Nucleic acid structures for va RNA transcription
AU2022267891A1 (en) * 2021-04-27 2023-11-09 Novartis Ag Viral vector production system
WO2023198685A1 (en) 2022-04-13 2023-10-19 F. Hoffmann-La Roche Ag Method for determining aav genomes
EP4279599A1 (en) * 2022-05-18 2023-11-22 Branca Bunus Limited Ordered formulation method to construct polyplex with core-aggregate structure
WO2023227438A1 (en) 2022-05-23 2023-11-30 F. Hoffmann-La Roche Ag Raman-based method for the differentiation of aav particle serotype and aav particle loading status
WO2023232922A1 (en) 2022-06-03 2023-12-07 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
WO2024013239A1 (en) 2022-07-14 2024-01-18 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
WO2024056561A1 (en) 2022-09-12 2024-03-21 F. Hoffmann-La Roche Ag Method for separating full and empty aav particles

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6989264B2 (en) * 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP2325299A3 (en) * 1997-09-05 2011-10-05 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant AAV vectors
US6593123B1 (en) * 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7829694B2 (en) * 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
RU2303064C2 (ru) * 2005-04-06 2007-07-20 ГУ НИИ вирусных препаратов им. О.Г. Анджапаридзе РАМН Молекулярный комплекс для трансфекции клеток млекопитающих, содержащий плазмидную днк, модифицированный полиэтиленимин и молекулы-лиганды
AU2013221212B2 (en) * 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
EP2970920B1 (en) * 2013-03-15 2018-04-25 The Children's Hospital of Philadelphia Scalable manufacturing process to produce recombinant lentiviral vectors in serum-free suspension cell culture system
EP3008191A2 (en) * 2013-06-13 2016-04-20 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
CN103329852B (zh) * 2013-06-19 2015-10-07 中国医学科学院病原生物学研究所 一种hbv持续性感染及纤维化小鼠模型建立
CA2916800C (en) * 2013-06-28 2022-10-25 Ethris Gmbh Compositions comprising a component with oligo(alkylene amine) moieties
CA3209883A1 (en) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
CA2926792C (en) * 2013-08-13 2021-12-07 Baylor College Of Medicine A novel plga-modified polyethylenimine self-assembly nanotechnology for nucleic acid and drug delivery
EP3384015A4 (en) * 2015-12-01 2019-05-29 Spark Therapeutics, Inc. SCALABLE PROCESS FOR THE PREPARATION OF A RECOMBINANT ADENO ASSOCIATED VIRAL VECTOR IN A SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR THE CLINICAL APPLICATION
JP2020524498A (ja) * 2017-06-07 2020-08-20 スパーク セラピューティクス インコーポレイテッドSpark Therapeutics, Inc. 細胞トランスフェクション及び/又はrAAVベクター産生の改善のための増強剤

Also Published As

Publication number Publication date
SG10202106287YA (en) 2021-07-29
IL259595B2 (en) 2024-01-01
AU2016362317A1 (en) 2018-06-14
KR20180091863A (ko) 2018-08-16
RU2018123502A (ru) 2020-01-14
CA3006309A1 (en) 2017-06-08
JP7444521B2 (ja) 2024-03-06
RU2766583C2 (ru) 2022-03-15
PE20181534A1 (es) 2018-09-26
PH12018501168A1 (en) 2019-01-21
IL259595A (en) 2018-07-31
SG11201804400SA (en) 2018-06-28
RU2018123502A3 (es) 2020-04-20
EP3384015A1 (en) 2018-10-10
IL259595B1 (en) 2023-09-01
US20190292561A1 (en) 2019-09-26
PE20240371A1 (es) 2024-03-05
CN108603174A (zh) 2018-09-28
AU2023200992A1 (en) 2023-05-18
AU2016362317B2 (en) 2023-03-16
BR112018011193A2 (pt) 2018-11-21
WO2017096039A1 (en) 2017-06-08
JP2018535682A (ja) 2018-12-06
CO2018006699A2 (es) 2018-09-20
EP3384015A4 (en) 2019-05-29
JP2022071049A (ja) 2022-05-13
MX2023012498A (es) 2023-11-03

Similar Documents

Publication Publication Date Title
PH12018501168A1 (en) Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
PH12019502774A1 (en) ENHANCING AGENTS FOR IMPROVED CELL TRANSFECTION AND/OR rAAV VECTOR PRODUCTION
MX2015012450A (es) Proceso de elaboracion escalable para producir vectores lentivirales recombinantes en un sistema de cultivo celular en suspension libre de suero.
MX2020005282A (es) Vector para la produccion de particulas aav.
BR112018005620A2 (pt) método para transferência de gene estável e de nível elevado em linfócitos
MX2021003188A (es) Composiciones y métodos para la manufactura de vectores de terapia génica.
WO2015168547A3 (en) In vivo gene engineering with adenoviral vectors
AU2016262093A8 (en) Enhancing endonuclease based gene editing in primary cells
MX2019002498A (es) Metodos y productos para la expresion de proteinas en celulas.
MX2023009050A (es) Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas.
WO2014077866A8 (en) Replicative minicircle vectors with improved expression
MX2021014525A (es) Adenovirus modificados.
MX2018010288A (es) Sistema de transposon y metodos de uso.
IL277883B1 (en) Optimized engineered nucleases with specificity for the human t-cell receptor alpha constant region gene
WO2016007570A3 (en) Engineered invariant natural killer t (inkt) cells and methods of making and using thereof
EA201492185A1 (ru) Способ получения рекомбинантной идуронат-2-сульфатазы
JP2016500260A5 (es)
PH12018500155A1 (en) Recombinant orf virus vector
ZA202207849B (en) Regulatory nucleic acid sequences
RU2018139201A (ru) Улучшенные способы доставки генов
CO2023017400A2 (es) Producción de vectores de aav recombinantes para tratamiento de distrofia muscular
JP2017524366A5 (es)
MX2023007527A (es) Metodos y sistemas para transfeccion celular mejorada.
FI3615674T3 (fi) Menetelmiä reumatoidin artriitin hoitoon HLA-geenin RNA-ohjattua genomin muokkausta käyttämällä
MX2015010887A (es) Formulaciones y metodos para aumentar la produccion de proteinas recombinantes.