BR112016015629A2 - produção de vetores virais adenoassociados de alto título - Google Patents

produção de vetores virais adenoassociados de alto título

Info

Publication number
BR112016015629A2
BR112016015629A2 BR112016015629A BR112016015629A BR112016015629A2 BR 112016015629 A2 BR112016015629 A2 BR 112016015629A2 BR 112016015629 A BR112016015629 A BR 112016015629A BR 112016015629 A BR112016015629 A BR 112016015629A BR 112016015629 A2 BR112016015629 A2 BR 112016015629A2
Authority
BR
Brazil
Prior art keywords
production
viral vectors
vectors
adenoassociated viral
cell lines
Prior art date
Application number
BR112016015629A
Other languages
English (en)
Inventor
Satkunanathan Stifani
Zhao Yuan
Original Assignee
The Sec Dep For Health
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 The Sec Dep For Health filed Critical The Sec Dep For Health
Publication of BR112016015629A2 publication Critical patent/BR112016015629A2/pt

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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
    • C12N15/63Introduction 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
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (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)

Abstract

a presente invenção refere-se a vetores virais adenoassociado (avv), a linhagens de células produtoras para a produção de vetores aav e a métodos de produção desses vetores. mais especificamente, a invenção refere-se a linhagens de células produtoras adaptadas para aumentar o título dos referidos vetores e a métodos de produção de vetores aav usando essas linhagens de células produtoras.
BR112016015629A 2014-01-31 2015-01-30 produção de vetores virais adenoassociados de alto título BR112016015629A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB201401707A GB201401707D0 (en) 2014-01-31 2014-01-31 Adeno-associated viral vectors
PCT/GB2015/050240 WO2015114365A1 (en) 2014-01-31 2015-01-30 High titer production of adeno-associated viral vectors

Publications (1)

Publication Number Publication Date
BR112016015629A2 true BR112016015629A2 (pt) 2017-10-24

Family

ID=50344213

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112016015629A BR112016015629A2 (pt) 2014-01-31 2015-01-30 produção de vetores virais adenoassociados de alto título

Country Status (13)

Country Link
US (1) US10035985B2 (pt)
EP (1) EP3099788A1 (pt)
JP (1) JP2017506885A (pt)
KR (1) KR20160137985A (pt)
CN (1) CN105980551A (pt)
AU (1) AU2015212563A1 (pt)
BR (1) BR112016015629A2 (pt)
CA (1) CA2934774A1 (pt)
GB (1) GB201401707D0 (pt)
IL (1) IL246588A0 (pt)
MX (1) MX2016009965A (pt)
SG (1) SG11201605848TA (pt)
WO (1) WO2015114365A1 (pt)

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
JP5963743B2 (ja) 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Cnsターゲティングaavベクターおよびその使用方法
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
EP3750907A3 (en) 2014-03-18 2021-04-28 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
JP7023108B2 (ja) 2014-10-21 2022-02-21 ユニバーシティ オブ マサチューセッツ 組み換えaavバリアントおよびその使用
US10335466B2 (en) 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
JP2017535266A (ja) 2014-11-14 2017-11-30 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)を治療する組成物および方法
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions and uses thereof
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US11253576B2 (en) 2015-10-22 2022-02-22 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US20190225955A1 (en) 2015-10-23 2019-07-25 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
ES2916325T3 (es) * 2015-11-05 2022-06-30 Massachusetts Gen Hospital Administración intratecal de secuencias de ácidos nucleicos codificantes de ABCD1 para el tratamiento de la adrenomieloneuropatía
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHODS FOR SELECTIVE INHIBITION OF EXPRESSION OF GRAINHEAD PROTEIN
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
JP7021191B2 (ja) 2016-08-15 2022-02-16 ジェンザイム・コーポレーション Aavを検出するための方法
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
IL269458B2 (en) 2017-03-23 2024-02-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
CN118006607A (zh) 2017-09-22 2024-05-10 马萨诸塞大学 Sod1双表达载体及其用途
KR20230110812A (ko) * 2017-10-10 2023-07-25 난트바이오 인코포레이티드 바이러스 생성 페이로드에 대해 독성이 낮은 변형된ec7 세포
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)
EP3697906A1 (en) 2017-10-16 2020-08-26 The Broad Institute, Inc. Uses of adenosine base editors
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
BR112021020391A2 (pt) * 2019-04-12 2022-02-08 Ultragenyx Pharmaceutical Inc Linhagens de células produtoras projetadas e métodos para preparar e usar as mesmas
EP3953478A4 (en) * 2019-04-12 2022-10-26 Massachusetts Eye and Ear Infirmary METHODS AND COMPOSITIONS FOR IMPROVING THE ARRANGEMENT OF ADENO-ASSOCIATED VIRUSES (AAVS)
US20220228129A1 (en) * 2019-04-26 2022-07-21 The Regents Of The University Of California Cells for enhanced production of adeno-associated virus
CN110184297A (zh) * 2019-05-27 2019-08-30 广州派真生物技术有限公司 一种表达shRNA的AAV辅助包装载体、构建筛选方法和应用
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003046142A2 (en) 2001-11-26 2003-06-05 Avigen, Inc. Methods for producing stocks of recombinant aav virions
DE602004030327D1 (de) * 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
US20080145313A1 (en) 2006-08-30 2008-06-19 Genesis Research & Development Corporation Limited Compositions and Methods for the Treatment and Prevention of Neoplastic Disorders
BR112012018899A2 (pt) 2010-01-28 2015-09-15 Philadelphia Children Hospital "método para purificar partículas de vetor de vírus adeno-associado."
WO2013033597A1 (en) * 2011-09-02 2013-03-07 Temple University - Of The Commonwealth System Of Higher Education Ssat mrna translation repression and activation
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products

Also Published As

Publication number Publication date
US20170029785A1 (en) 2017-02-02
CN105980551A (zh) 2016-09-28
CA2934774A1 (en) 2015-08-06
JP2017506885A (ja) 2017-03-16
GB201401707D0 (en) 2014-03-19
SG11201605848TA (en) 2016-08-30
MX2016009965A (es) 2017-06-14
EP3099788A1 (en) 2016-12-07
KR20160137985A (ko) 2016-12-02
WO2015114365A1 (en) 2015-08-06
IL246588A0 (en) 2016-08-31
AU2015212563A1 (en) 2016-07-14
US10035985B2 (en) 2018-07-31

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B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

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B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: EM VIRTUDE DO ARQUIVAMENTO PUBLICADO NA RPI 2616 DE 23-02-2021 E CONSIDERANDO AUSENCIA DE MANIFESTACAO DENTRO DOS PRAZOS LEGAIS, INFORMO QUE CABE SER MANTIDO O ARQUIVAMENTO DO PEDIDO DE PATENTE, CONFORME O DISPOSTO NO ARTIGO 12, DA RESOLUCAO 113/2013.