PL3093345T3 - Wektory bakulowirusowe zawierające powtórzone sekwencje kodujące o zróżnicowanej preferencji kodonów - Google Patents

Wektory bakulowirusowe zawierające powtórzone sekwencje kodujące o zróżnicowanej preferencji kodonów

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Publication number
PL3093345T3
PL3093345T3 PL16176100T PL16176100T PL3093345T3 PL 3093345 T3 PL3093345 T3 PL 3093345T3 PL 16176100 T PL16176100 T PL 16176100T PL 16176100 T PL16176100 T PL 16176100T PL 3093345 T3 PL3093345 T3 PL 3093345T3
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PL
Poland
Prior art keywords
coding sequences
baculoviral vectors
repeated coding
codon biases
differential codon
Prior art date
Application number
PL16176100T
Other languages
English (en)
Inventor
Andrew Christian BAKKER
Wilhelmus Theodorus Johannes Maria Christiaan Hermens
Original Assignee
Uniqure Ip B.V.
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Publication date
Application filed by Uniqure Ip B.V. filed Critical Uniqure Ip B.V.
Publication of PL3093345T3 publication Critical patent/PL3093345T3/pl

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    • 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
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    • C12N2710/14111Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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PL16176100T 2007-07-26 2008-07-25 Wektory bakulowirusowe zawierające powtórzone sekwencje kodujące o zróżnicowanej preferencji kodonów PL3093345T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US95208107P 2007-07-26 2007-07-26
EP07113257 2007-07-26
EP16176100.2A EP3093345B8 (en) 2007-07-26 2008-07-25 Baculoviral vectors comprising repeated coding sequences with differential codon biases
PCT/NL2008/050512 WO2009014445A2 (en) 2007-07-26 2008-07-25 Baculoviral vectors comprising repeated coding sequences with differential codon biases
EP08779058.0A EP2173888B1 (en) 2007-07-26 2008-07-25 Baculoviral vectors comprising repeated coding sequences with differential codon biases

Publications (1)

Publication Number Publication Date
PL3093345T3 true PL3093345T3 (pl) 2020-03-31

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PL08779058T PL2173888T3 (pl) 2007-07-26 2008-07-25 Wektory bakulowirusowe zawierające powtórzone sekwencje kodujące o zróżnicowanej preferencji kodonów
PL16176100T PL3093345T3 (pl) 2007-07-26 2008-07-25 Wektory bakulowirusowe zawierające powtórzone sekwencje kodujące o zróżnicowanej preferencji kodonów

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PL08779058T PL2173888T3 (pl) 2007-07-26 2008-07-25 Wektory bakulowirusowe zawierające powtórzone sekwencje kodujące o zróżnicowanej preferencji kodonów

Country Status (20)

Country Link
US (5) US8697417B2 (pl)
EP (3) EP3093345B8 (pl)
JP (2) JP5634262B2 (pl)
KR (1) KR101597695B1 (pl)
CN (1) CN101868547B (pl)
AU (1) AU2008279883B2 (pl)
BR (1) BRPI0814459B1 (pl)
CA (1) CA2694406C (pl)
DK (2) DK2173888T3 (pl)
EA (1) EA022169B1 (pl)
ES (1) ES2602585T3 (pl)
HK (1) HK1143185A1 (pl)
HU (1) HUE029744T2 (pl)
IL (1) IL203535A (pl)
MX (1) MX2010000944A (pl)
NZ (1) NZ582881A (pl)
PL (2) PL2173888T3 (pl)
PT (1) PT2173888T (pl)
SI (2) SI3093345T1 (pl)
WO (1) WO2009014445A2 (pl)

Families Citing this family (88)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2602585T3 (es) * 2007-07-26 2017-02-21 Uniqure Ip B.V. Vectores de baculovirus que comprenden secuencias de codificación repetidas con sesgos de codones diferenciales
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP3792348A3 (en) 2010-03-11 2021-06-23 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
US20160243260A1 (en) 2013-10-24 2016-08-25 Uniqure Ip B.V. Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins
AU2015230094B2 (en) * 2014-03-10 2021-05-27 Uniqure Ip B.V. Further improved AAV vectors produced in insect cells
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
JP6863891B2 (ja) 2014-11-14 2021-04-21 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. 調節性ポリヌクレオチド
CN107208142B (zh) 2014-11-28 2024-03-26 优尼科Ip有限公司 包含细小病毒病毒体的组合物中的dna杂质
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3540063A1 (en) 2014-12-24 2019-09-18 uniQure IP B.V. Rnai induced huntingtin gene suppression
KR20180134846A (ko) * 2016-04-21 2018-12-19 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
MX2019000962A (es) 2016-07-26 2019-08-01 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado.
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10286087B2 (en) 2016-12-16 2019-05-14 Uniqure Ip B.V. Immunoadsorption
CN110913866A (zh) 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3652326A1 (en) 2017-07-10 2020-05-20 uniQure IP B.V. Means and methods for aav gene therapy in humans
US11497576B2 (en) 2017-07-17 2022-11-15 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
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3697905A1 (en) 2017-10-16 2020-08-26 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
KR20200135768A (ko) 2018-01-17 2020-12-03 메이라지티엑스 유케이 Ii 리미티드 유전자 치료를 위한 변형된 rAAV 캡시드 단백질
EP3793616A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
US20210254103A1 (en) 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
EP3850098A1 (en) 2018-09-12 2021-07-21 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
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
EP3861107A1 (en) 2018-10-05 2021-08-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3116701A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
EP3884067A1 (en) 2018-11-19 2021-09-29 uniQure IP B.V. A companion diagnostic to monitor the effects of gene therapy
JP2022507283A (ja) 2018-11-19 2022-01-18 ユニキュアー アイピー ビー.ブイ. 脊髄小脳失調症3型の治療のためのアタキシン-3のrnai誘導低減
EP3883582A1 (en) 2018-11-19 2021-09-29 uniQure IP B.V. Method and means to deliver mirna to target cells
WO2020104480A1 (en) 2018-11-19 2020-05-28 Uniqure Biopharma B.V. Adeno-associated virus vectors for expressing fviii mimetics and uses thereof
TW202039858A (zh) 2019-01-18 2020-11-01 美商航海家醫療公司 用於生產aav顆粒之方法及系統
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
EP3999119A1 (en) 2019-07-15 2022-05-25 Meiragtx UK II Limited Modified aav capsid proteins for treatment of arthritic disease
EP4010465A1 (en) 2019-08-09 2022-06-15 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
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
WO2021195491A2 (en) * 2020-03-26 2021-09-30 Asklepios Biopharmaceutical, Inc. Inducible promoter for viral vector production
AU2021250656A1 (en) 2020-04-02 2022-09-15 Uniqure Biopharma B.V. Dual bifunctional vectors for AAV production
EP4127146A1 (en) 2020-04-02 2023-02-08 uniQure biopharma B.V. Novel cell line
JP2023521347A (ja) 2020-04-07 2023-05-24 ユニキュアー アイピー ビー.ブイ. アンジオポエチン様3(angptl3)をサイレンシングするための遺伝子構築物及びその使用
EP4135841A1 (en) 2020-04-15 2023-02-22 Voyager Therapeutics, Inc. Tau binding compounds
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202246516A (zh) 2021-03-03 2022-12-01 美商航海家醫療公司 病毒蛋白之控制表現
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022188797A1 (en) 2021-03-09 2022-09-15 Huigene Therapeutics Co., 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
EP4347798A2 (en) 2021-06-02 2024-04-10 uniQure biopharma B.V. Insect cell production of parvoviral vectors with modified capsid proteins
CA3219847A1 (en) 2021-06-02 2022-12-08 Sander Jan Hendrik Van Deventer Adeno-associated virus vectors modified to bind high-density lipoprotein
WO2022268835A1 (en) 2021-06-21 2022-12-29 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
WO2022268811A1 (en) 2021-06-21 2022-12-29 Uniqure Biopharma B.V. Improved lysis procedures
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
WO2023025920A1 (en) 2021-08-26 2023-03-02 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
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
CN114703203A (zh) * 2022-02-11 2022-07-05 上海渤因生物科技有限公司 杆状病毒载体及其用途
WO2023198745A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of apoe
WO2023198663A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of snca
WO2023198662A1 (en) 2022-04-12 2023-10-19 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

Family Cites Families (22)

* 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
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
EP1212411A2 (en) * 1999-08-20 2002-06-12 The Johns Hopkins University School Of Medicine Methods and compositions for the construction and use of fusion libraries
EP1228234A2 (en) * 2000-03-14 2002-08-07 Neurologix, Inc. Production of chimeric capsid vectors
US20030129203A1 (en) * 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
CA2467959C (en) 2001-11-09 2009-03-10 Robert M. Kotin 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
WO2003068804A2 (en) * 2002-02-14 2003-08-21 Novavax, Inc. Novel insect cell line
WO2003074714A1 (en) 2002-03-05 2003-09-12 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
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
ATE453716T1 (de) 2004-08-03 2010-01-15 Geneart Ag Verfahren zur modulation der genexpression durch änderung des cpg gehalts
DK1945779T3 (da) 2005-10-20 2013-06-03 Uniqure Ip Bv Forbedrede AAV vektorer frembragt i insektceller
US20090191597A1 (en) * 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
EP3023500B1 (en) * 2006-06-21 2020-02-12 uniQure IP B.V. Insect cells for the production of aav vectors
ES2602585T3 (es) * 2007-07-26 2017-02-21 Uniqure Ip B.V. Vectores de baculovirus que comprenden secuencias de codificación repetidas con sesgos de codones diferenciales

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