KR102609858B1 - 점액다당류증의 치료를 위한 아데노-관련 바이러스 벡터 - Google Patents

점액다당류증의 치료를 위한 아데노-관련 바이러스 벡터 Download PDF

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KR102609858B1
KR102609858B1 KR1020187000048A KR20187000048A KR102609858B1 KR 102609858 B1 KR102609858 B1 KR 102609858B1 KR 1020187000048 A KR1020187000048 A KR 1020187000048A KR 20187000048 A KR20187000048 A KR 20187000048A KR 102609858 B1 KR102609858 B1 KR 102609858B1
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cag
vector
aav9
seq
plasmid
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KR20180012843A (ko
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마리아 파티마 보쉬 투베르트
마리아 파티마 보쉬 투베르트
버지니아 아레바 하우리고트
산드라 모타스 마욜
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에스테베 파마슈티칼스 에스에이
유니버시타트 아우토노마 데 바르셀로나
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0066—Manipulation 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal 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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020187000048A 2015-06-05 2016-06-03 점액다당류증의 치료를 위한 아데노-관련 바이러스 벡터 Active KR102609858B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP15382297.8A EP3101125A1 (en) 2015-06-05 2015-06-05 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
EP15382297.8 2015-06-05
PCT/EP2016/062655 WO2016193431A1 (en) 2015-06-05 2016-06-03 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

Publications (2)

Publication Number Publication Date
KR20180012843A KR20180012843A (ko) 2018-02-06
KR102609858B1 true KR102609858B1 (ko) 2023-12-05

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KR1020187000048A Active KR102609858B1 (ko) 2015-06-05 2016-06-03 점액다당류증의 치료를 위한 아데노-관련 바이러스 벡터

Country Status (21)

Country Link
US (1) US10617771B2 (enExample)
EP (2) EP3101125A1 (enExample)
JP (1) JP6850736B2 (enExample)
KR (1) KR102609858B1 (enExample)
CN (1) CN108138154B (enExample)
AU (1) AU2016273343B2 (enExample)
CA (1) CA2988201C (enExample)
CL (1) CL2017003096A1 (enExample)
CO (1) CO2017012562A2 (enExample)
ES (1) ES2790834T3 (enExample)
IL (1) IL255939B (enExample)
MX (1) MX386615B (enExample)
MY (1) MY184586A (enExample)
NZ (1) NZ738018A (enExample)
PH (1) PH12017502192B1 (enExample)
PT (1) PT3303576T (enExample)
RU (1) RU2744593C2 (enExample)
TN (1) TN2017000506A1 (enExample)
UA (1) UA125373C2 (enExample)
WO (1) WO2016193431A1 (enExample)
ZA (1) ZA201708039B (enExample)

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WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma 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
CA2960912A1 (en) 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
ES2824457T3 (es) 2014-12-05 2021-05-12 UNIV AUTòNOMA DE BARCELONA Vectores virales para el tratamiento de la diabetes
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
SG11201808176TA (en) 2016-04-15 2018-10-30 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
TW202532642A (zh) 2017-04-14 2025-08-16 美商銳進科斯生物股份有限公司 使用由人類神經或膠細胞產生的重組人類艾杜糖醛酸鹽 (iduronate)-2-硫酸酯酶 (ids) 之黏多醣病ii之治療
CA3076036A1 (en) * 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
CN108715867A (zh) * 2018-05-31 2018-10-30 深圳市免疫基因治疗研究院 一种Sanfilippo B综合症慢病毒载体、慢病毒及其制备方法和应用
CN108795985A (zh) * 2018-05-31 2018-11-13 深圳市免疫基因治疗研究院 一种黏多糖贮积症慢病毒载体、慢病毒及其制备方法和应用
SG11202100781VA (en) * 2018-07-27 2021-02-25 Regenxbio Inc Treatment of mucopolysaccharidosis iva
WO2020099548A1 (en) * 2018-11-15 2020-05-22 Esteve Pharmaceuticals, S.A. Animal model of mucopolysaccharidoses type iva
TW202039856A (zh) * 2019-03-28 2020-11-01 西班牙商艾斯提夫製藥股份有限公司 製造重組病毒載體之方法
US11319354B2 (en) 2019-07-10 2022-05-03 Masonic Medical Research Laboratory VGLL4 with UCP-1 cis-regulatory element and method of use thereof
US11981912B2 (en) * 2019-12-10 2024-05-14 Takeda Pharma ceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
KR20220164743A (ko) * 2020-04-06 2022-12-13 호몰로지 메디슨, 인크. Ids 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
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
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
EP4291249A2 (en) 2021-02-10 2023-12-20 RegenxBio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
WO2022221421A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions with high brain expression for treating mucopolysaccharidosis ii
US20240350653A1 (en) * 2021-08-18 2024-10-24 Joint-Stock Company "Generium" Targeted delivery of therapeutic enzymes

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Publication number Publication date
EP3303576B1 (en) 2020-02-12
JP2018517414A (ja) 2018-07-05
ES2790834T3 (es) 2020-10-29
KR20180012843A (ko) 2018-02-06
MX386615B (es) 2025-03-19
WO2016193431A1 (en) 2016-12-08
AU2016273343A1 (en) 2017-12-21
RU2744593C2 (ru) 2021-03-11
TN2017000506A1 (en) 2019-04-12
CA2988201A1 (en) 2016-12-08
CO2017012562A2 (es) 2018-02-28
EP3101125A1 (en) 2016-12-07
CN108138154B (zh) 2022-01-18
MY184586A (en) 2021-04-06
US20180169272A1 (en) 2018-06-21
MX2017015725A (es) 2018-08-15
RU2017142006A (ru) 2019-07-09
US10617771B2 (en) 2020-04-14
IL255939A (en) 2018-01-31
PT3303576T (pt) 2020-05-08
IL255939B (en) 2021-05-31
BR112017025892A2 (pt) 2018-08-14
PH12017502192B1 (en) 2022-09-02
CN108138154A (zh) 2018-06-08
CL2017003096A1 (es) 2018-08-10
CA2988201C (en) 2024-01-16
EP3303576A1 (en) 2018-04-11
PH12017502192A1 (en) 2018-06-11
AU2016273343B2 (en) 2022-04-28
NZ738018A (en) 2025-03-28
UA125373C2 (uk) 2022-03-02
ZA201708039B (en) 2018-11-28
JP6850736B2 (ja) 2021-03-31
RU2017142006A3 (enExample) 2020-02-03

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