TW201814044A - 多核苷酸、載體、藥物組成物及其等之用途、以及載體獲得方法 - Google Patents

多核苷酸、載體、藥物組成物及其等之用途、以及載體獲得方法 Download PDF

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TW201814044A
TW201814044A TW106133935A TW106133935A TW201814044A TW 201814044 A TW201814044 A TW 201814044A TW 106133935 A TW106133935 A TW 106133935A TW 106133935 A TW106133935 A TW 106133935A TW 201814044 A TW201814044 A TW 201814044A
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vector
cag
gns
aav
seq
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TW106133935A
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Chinese (zh)
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查爾斯 羅卡列洽
維吉尼亞A. 霍里戈特曼多卡
法蒂瑪 波斯奇圖伯
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西班牙商以斯提夫博士實驗室股份有限公司
西班牙巴塞隆納歐都諾瑪大學
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Publication of TW201814044A publication Critical patent/TW201814044A/zh

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    • 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
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/06Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06014N-Acetylglucosamine-6-sulfatase (3.1.6.14)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/035Animal model for multifactorial diseases
    • A01K2267/0362Animal model for lipid/glucose metabolism, e.g. obesity, type-2 diabetes
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/15Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • C12N2830/80Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/90Vector systems having a special element relevant for transcription from vertebrates avian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
TW106133935A 2016-09-30 2017-09-30 多核苷酸、載體、藥物組成物及其等之用途、以及載體獲得方法 TW201814044A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP16382450 2016-09-30
??16382450.1 2016-09-30

Publications (1)

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TW201814044A true TW201814044A (zh) 2018-04-16

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TW106133935A TW201814044A (zh) 2016-09-30 2017-09-30 多核苷酸、載體、藥物組成物及其等之用途、以及載體獲得方法

Country Status (10)

Country Link
US (1) US11338046B2 (OSRAM)
EP (1) EP3519569B1 (OSRAM)
JP (1) JP7059285B2 (OSRAM)
CN (1) CN109863243B (OSRAM)
AR (1) AR109752A1 (OSRAM)
AU (1) AU2017333336B2 (OSRAM)
ES (1) ES2859661T3 (OSRAM)
PT (1) PT3519569T (OSRAM)
TW (1) TW201814044A (OSRAM)
WO (1) WO2018060097A1 (OSRAM)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI841566B (zh) * 2018-05-30 2024-05-11 西班牙商艾斯提夫製藥股份有限公司 用於治療黏多醣症 iv a 型之腺相關病毒載體

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EP2605798A1 (en) 2010-07-12 2013-06-26 Universidad Autònoma de Barcelona Gene therapy composition for use in diabetes treatment
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
EP3622821A1 (en) * 2013-05-15 2020-03-18 Regents of the University of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
EP3194430A1 (en) 2014-09-16 2017-07-26 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
WO2016087678A1 (en) 2014-12-05 2016-06-09 Universitat Autònoma De Barcelona Viral vectors for the treatment of diabetes
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
RU2020134965A (ru) 2018-04-27 2022-04-27 Спейскрафт Севен, Ллк Генная терапия при дегенерации цнс
AU2019299970B2 (en) * 2018-07-12 2025-12-11 Spacecraft Seven, Llc Gene therapy vectors for treatment of Danon disease
EP3914720B9 (en) 2019-07-10 2023-09-27 Masonic Medical Research Laboratory Vgll4 with ucp-1 cis-regulatory element and method of use thereof
CN111718947B (zh) * 2020-06-18 2022-08-23 舒泰神(北京)生物制药股份有限公司 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
CN114480454B (zh) * 2020-10-27 2024-03-29 华东理工大学 用于治疗粘多糖贮积症ⅱ型的腺相关病毒载体、药物及其应用

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US7232670B2 (en) * 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
DK2325302T3 (en) * 2003-02-11 2016-03-14 Shire Human Genetic Therapies Cells expressing a sulfatase and a C-formylglycine-generating enzyme and methods and uses thereof
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
AU2013216920B2 (en) 2012-02-07 2016-09-22 Global Bio Therapeutics, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
CN105916990B (zh) * 2013-08-02 2020-02-04 巴塞罗那自治大学 用于转导脂肪组织的腺相关病毒载体
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
GB201322798D0 (en) * 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
AR100419A1 (es) 2014-05-14 2016-10-05 Esteve Labor Dr Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosómico
SG11201706445SA (en) 2015-02-10 2017-09-28 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
US9981021B1 (en) * 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI841566B (zh) * 2018-05-30 2024-05-11 西班牙商艾斯提夫製藥股份有限公司 用於治療黏多醣症 iv a 型之腺相關病毒載體

Also Published As

Publication number Publication date
AU2017333336B2 (en) 2023-11-09
US11338046B2 (en) 2022-05-24
EP3519569B1 (en) 2020-12-02
PT3519569T (pt) 2021-03-04
AR109752A1 (es) 2019-01-23
AU2017333336A1 (en) 2019-04-11
ES2859661T3 (es) 2021-10-04
EP3519569A1 (en) 2019-08-07
JP2019528793A (ja) 2019-10-17
US20190231900A1 (en) 2019-08-01
CN109863243B (zh) 2023-07-28
JP7059285B2 (ja) 2022-04-25
CN109863243A (zh) 2019-06-07
WO2018060097A1 (en) 2018-04-05

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