CL2017003096A1 - Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis - Google Patents

Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis

Info

Publication number
CL2017003096A1
CL2017003096A1 CL2017003096A CL2017003096A CL2017003096A1 CL 2017003096 A1 CL2017003096 A1 CL 2017003096A1 CL 2017003096 A CL2017003096 A CL 2017003096A CL 2017003096 A CL2017003096 A CL 2017003096A CL 2017003096 A1 CL2017003096 A1 CL 2017003096A1
Authority
CL
Chile
Prior art keywords
treatment
mucopolysaccharidosis
adeno
viral vectors
associated viral
Prior art date
Application number
CL2017003096A
Other languages
English (en)
Inventor
Tubert Maria Fátima Bosch
Haurigot Virginia Areba
Mallol Sandra Motas
Original Assignee
Univ Barcelona Autonoma
Esteve Pharmaceuticals Sa
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 Univ Barcelona Autonoma, Esteve Pharmaceuticals Sa filed Critical Univ Barcelona Autonoma
Publication of CL2017003096A1 publication Critical patent/CL2017003096A1/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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

Landscapes

  • 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)

Abstract

La presente invención proporciona nuevos vectores derivados de virus adeno-asociados y composiciones farmacéuticas que los contienen para el tratamiento de trastornos de almacenamiento lisosómico y especialmente, para el tratamiento de mucopolisacaridosis Tipo II.
CL2017003096A 2015-06-05 2017-12-04 Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis CL2017003096A1 (es)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
CL2017003096A1 true CL2017003096A1 (es) 2018-08-10

Family

ID=53398018

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2017003096A CL2017003096A1 (es) 2015-06-05 2017-12-04 Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis

Country Status (21)

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

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US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma 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
US11085055B2 (en) 2014-12-05 2021-08-10 Universitat Autonoma 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
WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
WO2018191666A1 (en) * 2017-04-14 2018-10-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells
IL306098A (en) 2017-05-24 2023-11-01 Univ Barcelona Autonoma Viral expression vectors containing the coding sequence for FIBROBLAST GROWTH FACTOR 21 (FGF21)
WO2019060662A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE 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
TW202039857A (zh) * 2018-07-27 2020-11-01 美商銳進科斯生物股份有限公司 黏多醣病iva之治療
JP7462234B2 (ja) * 2018-11-15 2024-04-05 エステベ ファーマシューティカルズ，ソシエダッド アノニマ ムコ多糖症ｉｖａ型の動物モデル
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
CA3137622A1 (en) 2019-07-10 2021-01-14 Masonic Medical Research Institute Vgll4 with ucp-1 cis-regulatory element and method of use thereof
IL293684A (en) * 2019-12-10 2022-08-01 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease
MX2022012407A (es) * 2020-04-06 2022-10-28 Homology Medicines Inc Composiciones de virus adenoasociados para la transferencia del gen ids y sus metodos de uso.
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
WO2022173605A2 (en) 2021-02-10 2022-08-18 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
JP2024532176A (ja) * 2021-08-18 2024-09-05 ジョイント－ストック カンパニー “ジェネリウム” 療法酵素のターゲティング化送達

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Also Published As

Publication number Publication date
PH12017502192B1 (en) 2022-09-02
JP6850736B2 (ja) 2021-03-31
EP3303576B1 (en) 2020-02-12
MY184586A (en) 2021-04-06
AU2016273343A1 (en) 2017-12-21
UA125373C2 (uk) 2022-03-02
EP3101125A1 (en) 2016-12-07
ES2790834T3 (es) 2020-10-29
IL255939B (en) 2021-05-31
CN108138154A (zh) 2018-06-08
NZ738018A (en) 2025-03-28
EP3303576A1 (en) 2018-04-11
IL255939A (en) 2018-01-31
PT3303576T (pt) 2020-05-08
KR20180012843A (ko) 2018-02-06
CA2988201A1 (en) 2016-12-08
CA2988201C (en) 2024-01-16
PH12017502192A1 (en) 2018-06-11
CO2017012562A2 (es) 2018-02-28
CN108138154B (zh) 2022-01-18
US20180169272A1 (en) 2018-06-21
RU2017142006A (ru) 2019-07-09
JP2018517414A (ja) 2018-07-05
MX386615B (es) 2025-03-19
WO2016193431A1 (en) 2016-12-08
KR102609858B1 (ko) 2023-12-05
RU2744593C2 (ru) 2021-03-11
AU2016273343B2 (en) 2022-04-28
RU2017142006A3 (es) 2020-02-03
ZA201708039B (en) 2018-11-28
TN2017000506A1 (en) 2019-04-12
US10617771B2 (en) 2020-04-14
MX2017015725A (es) 2018-08-15
BR112017025892A2 (pt) 2018-08-14

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