DK3219801T3 - Konstruktion - Google Patents

Konstruktion Download PDF

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Publication number
DK3219801T3
DK3219801T3 DK17161437.3T DK17161437T DK3219801T3 DK 3219801 T3 DK3219801 T3 DK 3219801T3 DK 17161437 T DK17161437 T DK 17161437T DK 3219801 T3 DK3219801 T3 DK 3219801T3
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DK
Denmark
Prior art keywords
construction
Prior art date
Application number
DK17161437.3T
Other languages
English (en)
Inventor
Kyriacos Mitrophanous
Scott Ralph
Hannah Stewart
Alan Kingsman
Original Assignee
Oxford Biomedica Ltd
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Publication of DK3219801T3 publication Critical patent/DK3219801T3/da

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    • 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
    • C12N15/867Retroviral 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
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/54Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0071Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88Lyases (4.)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P13/00Preparation of nitrogen-containing organic compounds
    • C12P13/001Amines; Imines
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16002Tyrosine 3-monooxygenase (1.14.16.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04016GTP cyclohydrolase I (3.5.4.16)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y401/00Carbon-carbon lyases (4.1)
    • C12Y401/01Carboxy-lyases (4.1.1)
    • C12Y401/01028Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use 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
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
DK17161437.3T 2011-10-28 2012-10-26 Konstruktion DK3219801T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161552581P 2011-10-28 2011-10-28
GBGB1118636.8A GB201118636D0 (en) 2011-10-28 2011-10-28 Nucleotide sequence
EP12778779.4A EP2771471B1 (en) 2011-10-28 2012-10-26 Construct

Publications (1)

Publication Number Publication Date
DK3219801T3 true DK3219801T3 (da) 2021-02-22

Family

ID=45373567

Family Applications (2)

Application Number Title Priority Date Filing Date
DK12778779.4T DK2771471T3 (da) 2011-10-28 2012-10-26 Konstruktion
DK17161437.3T DK3219801T3 (da) 2011-10-28 2012-10-26 Konstruktion

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DK12778779.4T DK2771471T3 (da) 2011-10-28 2012-10-26 Konstruktion

Country Status (11)

Country Link
US (1) US11279954B2 (da)
EP (2) EP2771471B1 (da)
JP (1) JP6296987B2 (da)
CN (2) CN107523581B (da)
AU (1) AU2012328166B2 (da)
CA (2) CA2849241C (da)
DK (2) DK2771471T3 (da)
ES (2) ES2628419T3 (da)
GB (1) GB201118636D0 (da)
PL (2) PL3219801T3 (da)
WO (1) WO2013061076A1 (da)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
RU2018104098A (ru) * 2015-08-03 2019-09-06 Майодопа Лимитед Системный синтез и регуляция l-дофа
GB2562774A (en) * 2017-05-25 2018-11-28 Mcdonald Michael Genetic construct
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN108841868A (zh) * 2018-05-31 2018-11-20 康霖生物科技(杭州)有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体
JP2022525136A (ja) * 2019-03-10 2022-05-11 サイオ ジーン セラピーズ インコーポレイテッド パーキンソン病を処置するための遺伝子治療組成物および方法
CN109971729B (zh) * 2019-04-19 2021-07-16 上海信致医药科技有限公司 一种酶组合物
GB2587319A (en) 2019-08-12 2021-03-31 Healing Genes Llc Genetic construct
CN116555351A (zh) * 2020-03-13 2023-08-08 康霖生物科技(杭州)有限公司 一种核酸构建体
GB2601752A (en) 2020-12-08 2022-06-15 Maavrx Ltd Expression vector
WO2023111592A1 (en) 2021-12-17 2023-06-22 Imperial College Innovations Limited Rna construct
CN114317610A (zh) * 2022-01-27 2022-04-12 上海本导基因技术有限公司 一种适用于帕金森疾病基因治疗的慢病毒载体
CN117106825A (zh) * 2023-08-28 2023-11-24 康霖生物科技(杭州)有限公司 用于治疗帕金森病的基因治疗载体及其用途

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
DE4006630A1 (de) 1990-03-03 1991-09-12 Behringwerke Ag Humane monoklonale antikoerper gegen tollwutviren, ihre herstellung und verwendung
US5512421A (en) 1991-02-19 1996-04-30 The Regents Of The University Of California Generation, concentration and efficient transfer of VSV-G pseudotyped retroviral vectors
DE69232353T2 (de) 1991-06-21 2002-11-14 Phillips Petroleum Co Fluorwasserstoffsäure und Sulfone enthaltender Alkylierungskatalysator
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
AU1073797A (en) * 1995-11-14 1997-06-05 Somatix Therapy Corporation Joint expression of gtp cyclohydrolase and tyrosine hydroxylase
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
ES2153654T3 (es) 1996-10-17 2001-03-01 Oxford Biomedica Ltd Vectores retrovirales.
GB9622500D0 (en) * 1996-10-29 1997-01-08 Oxford Biomedica Ltd Therapeutic gene
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
CN1325653C (zh) 1998-05-22 2007-07-11 牛津生物医学(英国)有限公司 逆转录病毒传递系统
GB9904905D0 (en) 1999-03-03 1999-04-28 Oxford Biomedica Ltd Cells
US6857241B1 (en) 1999-07-13 2005-02-22 Carlos Fradera Pellicer Building panel and plant for the manufacture thereof
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
GB0024550D0 (da) * 2000-10-06 2000-11-22 Oxford Biomedica Ltd
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
DK1504108T3 (da) 2002-02-01 2013-06-10 Oxford Biomedica Ltd Lentivirusvektor
SE0302509D0 (sv) 2003-09-19 2003-09-19 Amersham Biosciences Ab Matrix for separation of polyethers and method of separation
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
GB0526210D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Vectors
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
CN102124115B (zh) 2008-06-18 2020-03-03 牛津生物医学(英国)有限公司 病毒纯化
WO2010055290A1 (en) * 2008-11-11 2010-05-20 Oxford Biomedica (Uk) Limited Method
EP2575894B1 (en) * 2010-05-28 2015-02-25 Oxford Biomedica (UK) Ltd Delivery of lentiviral vectors to the brain

Also Published As

Publication number Publication date
ES2628419T3 (es) 2017-08-02
CA2849241C (en) 2019-12-31
WO2013061076A1 (en) 2013-05-02
CN107523581B (zh) 2021-08-17
EP3219801B1 (en) 2020-12-16
GB201118636D0 (en) 2011-12-07
JP6296987B2 (ja) 2018-03-20
ES2857003T3 (es) 2021-09-28
CA3052407A1 (en) 2013-05-02
CN104011213B (zh) 2017-10-03
US20200040361A1 (en) 2020-02-06
EP2771471A1 (en) 2014-09-03
CA3052407C (en) 2020-03-31
EP2771471B1 (en) 2017-05-03
US11279954B2 (en) 2022-03-22
AU2012328166A1 (en) 2014-06-12
CN107523581A (zh) 2017-12-29
PL3219801T3 (pl) 2021-06-14
CN104011213A (zh) 2014-08-27
JP2014532402A (ja) 2014-12-08
PL2771471T3 (pl) 2017-10-31
DK2771471T3 (da) 2017-08-21
CA2849241A1 (en) 2013-05-02
EP3219801A1 (en) 2017-09-20
AU2012328166B2 (en) 2018-03-08

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