CN101868547B - 包含具有差别密码子偏性的重复编码序列的杆状病毒载体 - Google Patents

包含具有差别密码子偏性的重复编码序列的杆状病毒载体 Download PDF

Info

Publication number
CN101868547B
CN101868547B CN200880108079.2A CN200880108079A CN101868547B CN 101868547 B CN101868547 B CN 101868547B CN 200880108079 A CN200880108079 A CN 200880108079A CN 101868547 B CN101868547 B CN 101868547B
Authority
CN
China
Prior art keywords
nucleotide sequence
sequence
insect cell
amino acid
codon
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CN200880108079.2A
Other languages
English (en)
Chinese (zh)
Other versions
CN101868547A (zh
Inventor
A·C·贝克
W·T·J·M·C·赫尔门斯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Uniqure IP BV
Original Assignee
Uniqure IP BV
Amsterdam Molecular Therapeutics AMT BV
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 Uniqure IP BV, Amsterdam Molecular Therapeutics AMT BV filed Critical Uniqure IP BV
Publication of CN101868547A publication Critical patent/CN101868547A/zh
Application granted granted Critical
Publication of CN101868547B publication Critical patent/CN101868547B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14041Use of virus, viral particle or viral elements as a vector
    • C12N2710/14044Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14111Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143Use 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
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14122New 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
    • 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
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/50Vectors for producing vectors

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN200880108079.2A 2007-07-26 2008-07-25 包含具有差别密码子偏性的重复编码序列的杆状病毒载体 Active CN101868547B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US95208107P 2007-07-26 2007-07-26
US60/952,081 2007-07-26
EP07113257 2007-07-26
EP07113257.5 2007-07-26
PCT/NL2008/050512 WO2009014445A2 (en) 2007-07-26 2008-07-25 Baculoviral vectors comprising repeated coding sequences with differential codon biases

Publications (2)

Publication Number Publication Date
CN101868547A CN101868547A (zh) 2010-10-20
CN101868547B true CN101868547B (zh) 2016-03-16

Family

ID=38753583

Family Applications (1)

Application Number Title Priority Date Filing Date
CN200880108079.2A Active CN101868547B (zh) 2007-07-26 2008-07-25 包含具有差别密码子偏性的重复编码序列的杆状病毒载体

Country Status (19)

Country Link
US (5) US8697417B2 (OSRAM)
EP (3) EP3561063A1 (OSRAM)
JP (2) JP5634262B2 (OSRAM)
KR (1) KR101597695B1 (OSRAM)
CN (1) CN101868547B (OSRAM)
AU (1) AU2008279883B2 (OSRAM)
BR (1) BRPI0814459B1 (OSRAM)
CA (1) CA2694406C (OSRAM)
DK (2) DK2173888T3 (OSRAM)
EA (1) EA022169B1 (OSRAM)
ES (1) ES2602585T3 (OSRAM)
HU (1) HUE029744T2 (OSRAM)
IL (1) IL203535A (OSRAM)
MX (1) MX2010000944A (OSRAM)
NZ (1) NZ582881A (OSRAM)
PL (2) PL2173888T3 (OSRAM)
PT (1) PT2173888T (OSRAM)
SI (2) SI2173888T1 (OSRAM)
WO (1) WO2009014445A2 (OSRAM)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2739596C2 (ru) * 2016-04-21 2020-12-28 Вайровек, Инк Продуцирование aav в клетках насекомых, способы и композиции для этого

Families Citing this family (108)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2173888T (pt) * 2007-07-26 2016-11-17 Uniqure Ip Bv Resumo
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US9228174B2 (en) * 2010-03-11 2016-01-05 Uniqure Ip B.V. Mutated rep encoding sequences for use in AAV production
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
WO2012145509A2 (en) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors, and viruses
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
ES2990174T3 (es) 2014-03-10 2024-11-29 Uniqure Ip Bv Vectores de AAV mejorados producidos en células de insecto
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
DK3224376T4 (da) 2014-11-28 2023-05-30 Uniqure Ip Bv DNA-forureninger i en sammensætning omfattende en parvoviral virion
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EA037696B1 (ru) 2014-12-24 2021-05-12 ЮНИКЕР АйПи Б.В. Супрессия гена гентингтина, индуцированная рнк-интерференцией
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
EP3491008A2 (en) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
CA3045551A1 (en) 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
IL271957B2 (en) 2017-07-10 2024-02-01 Uniqure Ip Bv Means and methods for AAV-based gene therapy
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN111527201A (zh) 2017-12-29 2020-08-11 优尼科Ip有限公司 修饰的病毒载体及其制备和使用方法
LT3740222T (lt) 2018-01-17 2023-09-11 Meiragtx Uk Ii Limited Modifikuotas raav kapsidės baltymas, skirtas genų terapijai
GB201800903D0 (en) 2018-01-19 2018-03-07 Oxford Genetics Ltd Vectors
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. パーキンソン病を治療するための組成物および方法
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒(aav)之組成物和方法
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
JP7565218B2 (ja) 2018-07-02 2024-10-10 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症および脊髄に関連する障害の治療
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CA3112007A1 (en) 2018-09-12 2020-03-19 Uniqure Ip B.V. Rnai induced c9orf72 suppression for the treatment of als/ftd
WO2020061418A1 (en) * 2018-09-20 2020-03-26 Tallgrass Biologics L.L.C. Treatment and control of colony collapse disorder
CN109295172A (zh) * 2018-09-26 2019-02-01 浙江海洋大学 大黄鱼清道夫受体家族基因的密码子偏好性分析方法
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3867389A1 (en) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
EP3884050A1 (en) 2018-11-19 2021-09-29 uniQure IP B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
WO2020104435A1 (en) 2018-11-19 2020-05-28 Uniqure Ip B.V. A companion diagnostic to monitor the effects of gene therapy
WO2020104480A1 (en) 2018-11-19 2020-05-28 Uniqure Biopharma B.V. Adeno-associated virus vectors for expressing fviii mimetics and uses thereof
AU2019382824A1 (en) 2018-11-19 2021-06-03 Uniqure Ip B.V. Method and means to deliver miRNA to target cells
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
EP3962536A1 (en) 2019-04-29 2022-03-09 Voyager Therapeutics, Inc. Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors
CA3146791A1 (en) 2019-07-15 2021-01-21 Meiragtx Uk Ii Limited Modified aav capsid proteins for treatment of arthritic disease
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202122582A (zh) 2019-08-26 2021-06-16 美商航海家醫療公司 病毒蛋白之控制表現
WO2021041375A1 (en) * 2019-08-26 2021-03-04 Rootpath Genomics, Inc. Compositions and methods for producing adeno-associated viral vectors
WO2021053018A1 (en) 2019-09-16 2021-03-25 Uniqure Ip B.V. Targeting misspliced transcripts in genetic disorders
WO2021113767A1 (en) * 2019-12-04 2021-06-10 Sangamo Therapeutics, Inc. Novel compositions and methods for producing recombinant aav
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
WO2021195491A2 (en) * 2020-03-26 2021-09-30 Asklepios Biopharmaceutical, Inc. Inducible promoter for viral vector production
EP4127146A1 (en) 2020-04-02 2023-02-08 uniQure biopharma B.V. Novel cell line
CA3169087A1 (en) * 2020-04-02 2021-10-07 David Johannes Francois DU PLESSIS Dual bifunctional vectors for aav production
WO2021204872A1 (en) 2020-04-07 2021-10-14 Uniqure Ip B.V. Gene constructs for silencing angiopoietin-like 3 (angptl3) and uses thereof
WO2021211753A1 (en) 2020-04-15 2021-10-21 Voyager Therapeutics, Inc. Tau binding compounds
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4189095A1 (en) 2020-07-27 2023-06-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4305157A1 (en) 2021-03-09 2024-01-17 Huidagene Therapeutics (Singapore) Pte. Ltd. Engineered crispr/cas13 system and uses thereof
WO2022214635A1 (en) 2021-04-08 2022-10-13 Stichting Vu Nucleic acid molecules for compensation of stxbp1 haploinsufficiency and their use in the treatment of stxbp1-related disorders
AU2022285138A1 (en) 2021-06-02 2023-11-30 Uniqure Biopharma B.V. Adeno-associated virus vectors modified to bind high-density lipoprotein
WO2022253955A2 (en) 2021-06-02 2022-12-08 Uniqure Biopharma B.V. Insect cell production of parvoviral vectors with modified capsid proteins
US20250382611A1 (en) 2021-06-21 2025-12-18 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
CN117836010A (zh) 2021-06-21 2024-04-05 优尼科生物制药有限公司 改进的裂解程序
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
AU2022317647A1 (en) * 2021-07-28 2024-01-25 Biomarin Pharmaceutical Inc. Large scale adeno-associated virus production systems
EP4392434A1 (en) 2021-08-26 2024-07-03 uniQure biopharma B.V. Insect cell-produced high potency aav vectors with cns-tropism
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4469585A2 (en) 2022-01-25 2024-12-04 Voyager Therapeutics, Inc. Baculovirus expression system
CN114703203B (zh) * 2022-02-11 2024-08-06 上海渤因生物科技有限公司 杆状病毒载体及其用途
JP2025513066A (ja) 2022-04-12 2025-04-22 ユニキュアー バイオファーマ ビー.ブイ. Apoeの核酸制御
US20250297252A1 (en) 2022-04-12 2025-09-25 Uniqure Biopharma B.V. Nucleic acid regulation of SNCA
US20250230437A1 (en) 2022-04-12 2025-07-17 Uniqure Biopharma B.V. Novel systems for nucleic acid regulation
WO2023198702A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of c9orf72
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024143440A1 (ja) 2022-12-28 2024-07-04 富士フイルム株式会社 アデノ随伴ウイルスの製造方法、およびベクターのセット
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024218192A1 (en) 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs
WO2024218190A1 (en) 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Generation of adeno-associated virus capsid libraries for insect cells
AU2024256392A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Gene delivery vehicles comprising rna and antibodies
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2024261220A1 (en) 2023-06-23 2024-12-26 Uniqure Biopharma B.V. Novel fragile x constructs
WO2025049993A1 (en) 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025051805A1 (en) 2023-09-04 2025-03-13 Uniqure Biopharma B.V. Novel neurotropic recombinant adeno-associated virus particles
WO2025078606A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Further novel systems for nucleic acid regulation
WO2025078598A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Novel nucleic acid regulation of huntingtin gene
WO2025078602A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Nucleic acid regulation of msh3 in repeat expansion disorders
WO2025114524A1 (en) 2023-11-30 2025-06-05 Uniqure Biopharma B.V. Formulations for viral drug products
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025219443A1 (en) 2024-04-19 2025-10-23 Uniqure Biopharma B.V. Nucleic acid for a1at regulation

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
US5871974A (en) * 1990-09-28 1999-02-16 Ixsys Inc. Surface expression libraries of heteromeric receptors
NZ308772A (en) * 1995-05-17 1999-04-29 Du Pont Recombinant baculovirus insecticides
FR2774699B1 (fr) 1997-11-17 2003-10-03 Rhone Poulenc Rorer Sa Methode de reduction des evenements de recombinaison homologue
AU6490699A (en) 1998-09-24 2000-04-10 Ultrasonus Ab A mixing method and a mixing chamber
US6103526A (en) 1998-10-08 2000-08-15 Protein Sciences Corporation Spodoptera frugiperda single cell suspension cell line in serum-free media, methods of producing and using
ATE404217T1 (de) 1999-06-24 2008-08-15 Univ British Columbia Therapie mit lipoproteinlipase (lpl) variant
WO2001014539A2 (en) * 1999-08-20 2001-03-01 Johns Hopkins University School Of Medicine Methods and compositions for the construction and use of fusion libraries
WO2001068888A2 (en) * 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
US20030129203A1 (en) * 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
CA2369985A1 (en) * 2002-01-18 2003-07-18 Duke University Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
AU2003217413A1 (en) * 2002-02-14 2003-09-04 Novavax, Inc. Method for isolation and purification of expressed gene products in vitro
AU2003212708A1 (en) 2002-03-05 2003-09-16 Stichting Voor De Technische Wetenschappen Baculovirus expression system
DE10260805A1 (de) 2002-12-23 2004-07-22 Geneart Gmbh Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins
AU2003272868A1 (en) * 2003-10-15 2005-04-27 Agtc Gene Technology Company Ltd. Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors
US20060166363A1 (en) * 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
AU2005270430B2 (en) 2004-08-03 2008-06-12 Geneart Ag Method for modulating gene expression by modifying the CpG content
PL1945779T3 (pl) 2005-10-20 2013-08-30 Uniqure Ip Bv Ulepszone wektory AAV produkowane w komórkach owadzich
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
CA2655957C (en) 2006-06-21 2016-05-03 Amsterdam Molecular Therapeutics (Amt) B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
PT2173888T (pt) * 2007-07-26 2016-11-17 Uniqure Ip Bv Resumo

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Erik Kohlbrenner et al.Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system.《Molecular Therapy》.2005,第12卷全文. *
M Urabe et al.Insert cells as a factory to produce adeno-associated virus type2 vectors.《Human gene therapy》.2002,全文. *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2739596C2 (ru) * 2016-04-21 2020-12-28 Вайровек, Инк Продуцирование aav в клетках насекомых, способы и композиции для этого

Also Published As

Publication number Publication date
SI3093345T1 (sl) 2019-08-30
EP3561063A1 (en) 2019-10-30
MX2010000944A (es) 2010-08-31
ES2602585T3 (es) 2017-02-21
AU2008279883A1 (en) 2009-01-29
US8697417B2 (en) 2014-04-15
US20100261254A1 (en) 2010-10-14
JP5634262B2 (ja) 2014-12-03
IL203535A0 (en) 2011-07-31
JP2010534472A (ja) 2010-11-11
US20220251520A1 (en) 2022-08-11
WO2009014445A3 (en) 2009-04-30
HK1143185A1 (zh) 2010-12-24
BRPI0814459A2 (pt) 2014-10-21
US12486496B2 (en) 2025-12-02
EP3093345B1 (en) 2019-04-24
IL203535A (en) 2016-02-29
US11306291B2 (en) 2022-04-19
EP2173888A2 (en) 2010-04-14
EP3093345A1 (en) 2016-11-16
US9580691B2 (en) 2017-02-28
EP2173888B1 (en) 2016-08-17
CN101868547A (zh) 2010-10-20
CA2694406C (en) 2019-03-26
DK2173888T3 (en) 2016-11-28
AU2008279883B2 (en) 2013-12-05
EP3093345B8 (en) 2019-07-24
DK3093345T3 (da) 2019-06-24
SI2173888T1 (sl) 2017-01-31
WO2009014445A2 (en) 2009-01-29
US20140186926A1 (en) 2014-07-03
PL2173888T3 (pl) 2017-02-28
PL3093345T3 (pl) 2020-03-31
CA2694406A1 (en) 2009-01-29
BRPI0814459B1 (pt) 2023-01-24
US20190203183A1 (en) 2019-07-04
EA022169B1 (ru) 2015-11-30
JP2014239686A (ja) 2014-12-25
HUE029744T2 (en) 2017-04-28
US20160032254A1 (en) 2016-02-04
KR20100065284A (ko) 2010-06-16
US10190098B2 (en) 2019-01-29
EA201070184A1 (ru) 2010-08-30
NZ582881A (en) 2012-04-27
KR101597695B1 (ko) 2016-02-25
PT2173888T (pt) 2016-11-17

Similar Documents

Publication Publication Date Title
US12486496B2 (en) AAV vectors produced by insect cells comprising Rep52 and Rep78 coding sequences with differential codon biases
US10865423B2 (en) Vectors with modified initiation codon for the translation of AAV-REP78 useful for production of AAV
CN101287837B (zh) 昆虫细胞中生产的改进的aav载体
AU2014201171B9 (en) Baculoviral vectors comprising repeated coding sequences with differential codon biases
HK40014371A (en) Baculoviral vectors comprising repeated coding sequences with differential codon biases
DK2035564T3 (en) VECTORS WITH MODIFIED initiation codon TO THE TRANSLATION OF AAV rep 78 FOR PRODUCTION OF AAV IN INSECT CELLS
HK1231125B (en) Baculoviral vectors comprising repeated coding sequences with differential codon biases
HK1231125A1 (en) Baculoviral vectors comprising repeated coding sequences with differential codon biases
HK40037260A (en) Aav vectors with improved rep coding sequences for production in insect cells
HK1143185B (en) Baculoviral vectors comprising repeated coding sequences with differential codon biases
HK1224336B (en) Insect cells for the production of aav vectors
HK1127083B (en) Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C53 Correction of patent of invention or patent application
CB02 Change of applicant information

Address after: Amsterdam, The Netherlands

Applicant after: UNIQURE IP B.V.

Address before: Amsterdam, The Netherlands

Applicant before: Amsterdam molecular therapy (AMT) Co.,Ltd.

Address after: Amsterdam, The Netherlands

Applicant after: Amsterdam molecular therapy (AMT) Co.,Ltd.

Address before: Amsterdam, The Netherlands

Applicant before: Amsterdam Molecular Therapeutics B.V.

COR Change of bibliographic data

Free format text: CORRECT: APPLICANT; FROM: AMSTERDAM MOLECULAR THERAPEUTI TO: AMSTERDAM MOLECULAR THERAPEUTICS BV

Free format text: CORRECT: APPLICANT; FROM: AMSTERDAM MOLECULAR THERAPEUTICS BV TO: JURNIKL BIOPHARMACEUTICAL CO., LTD.

ASS Succession or assignment of patent right

Owner name: UNIQURE IP B. V.

Free format text: FORMER OWNER: JURNIKL BIOPHARMACEUTICAL CO., LTD.

Effective date: 20150618

C41 Transfer of patent application or patent right or utility model
TA01 Transfer of patent application right

Effective date of registration: 20150618

Address after: Amsterdam, The Netherlands

Applicant after: UNIQURE IP B.V.

Address before: Amsterdam, The Netherlands

Applicant before: UNIQURE IP B.V.

C14 Grant of patent or utility model
GR01 Patent grant