MX2016014773A - Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico. - Google Patents

Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico.

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Publication number
MX2016014773A
MX2016014773A MX2016014773A MX2016014773A MX2016014773A MX 2016014773 A MX2016014773 A MX 2016014773A MX 2016014773 A MX2016014773 A MX 2016014773A MX 2016014773 A MX2016014773 A MX 2016014773A MX 2016014773 A MX2016014773 A MX 2016014773A
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MX
Mexico
Prior art keywords
treatment
virus vectors
lysosomal storage
storage disorders
adenoassociated virus
Prior art date
Application number
MX2016014773A
Other languages
English (en)
Inventor
Fàtima Bosch Tubert M
Virginia HAURIGOT MENDOÇA M
Ribera Sanche Albert
Original Assignee
Esteve Labor Dr
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 Esteve Labor Dr filed Critical Esteve Labor Dr
Publication of MX2016014773A publication Critical patent/MX2016014773A/es

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    • 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)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2474Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase
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    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105Alpha-N-acetylglucosaminidase (3.2.1.50)
    • 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
    • A61K48/0075Medicinal 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
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/10Vectors comprising a special translation-regulating system regulates levels of translation
    • C12N2840/105Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)

Abstract

La presente invención proporciona nuevos vectores de virus adenoasociados y composiciones farmacéuticas que contienen a los mismos para el tratamiento de trastornos de almacenamiento lisosómico y, en especial, para el tratamiento de la mucopolisacaridosis Tipo IIIB.
MX2016014773A 2014-05-14 2015-05-13 Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico. MX2016014773A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP14382171 2014-05-14
PCT/EP2015/060604 WO2015173308A1 (en) 2014-05-14 2015-05-13 Adenoassociated virus vectors for the treatment of lysosomal storage disorders

Publications (1)

Publication Number Publication Date
MX2016014773A true MX2016014773A (es) 2017-05-04

Family

ID=50771225

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016014773A MX2016014773A (es) 2014-05-14 2015-05-13 Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico.

Country Status (22)

Country Link
US (1) US11149285B2 (es)
EP (1) EP3143146B1 (es)
JP (1) JP6634073B2 (es)
KR (1) KR102446169B1 (es)
CN (1) CN107002095B (es)
AR (1) AR100419A1 (es)
AU (1) AU2015261519B2 (es)
BR (1) BR112016025819B1 (es)
CA (1) CA2947468C (es)
ES (1) ES2823948T3 (es)
IL (1) IL248538B (es)
MX (1) MX2016014773A (es)
MY (1) MY183472A (es)
NZ (1) NZ726316A (es)
PH (1) PH12016502248A1 (es)
PT (1) PT3143146T (es)
RU (1) RU2718248C2 (es)
SA (1) SA516380290B1 (es)
SG (1) SG11201609314XA (es)
TW (1) TWI701332B (es)
UA (1) UA123985C2 (es)
WO (1) WO2015173308A1 (es)

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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
MX2016001317A (es) 2013-07-29 2016-04-07 Danisco Us Inc Variantes de enzimas.
EP3194430A1 (en) 2014-09-16 2017-07-26 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3015134B1 (en) 2014-10-29 2020-08-26 Noxell Corporation Hair colouration with a cationic coloured polymer, method and kit thereof
EP3015135B1 (en) 2014-10-29 2020-09-09 Noxell Corporation Hair colouration with a cationic or anionic coloured polymer, method and kit thereof
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
US11458211B2 (en) 2016-07-12 2022-10-04 The University Of Manchester Gene therapy
AU2017333336B2 (en) * 2016-09-30 2023-11-09 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
MX2020005673A (es) * 2017-11-30 2020-12-03 Univ Pennsylvania Terapia génica para mucopolisacaridosis iiib.
JP2021522278A (ja) * 2018-04-27 2021-08-30 ザ メディカル カレッジ オブ ウィスコンシン インクThe Medical College Of Wisconsin, Inc. ライソゾーム病を改善するための、レンチウイルスベクターが形質導入されたt−rapa細胞の使用
SG11202011777PA (en) * 2018-05-30 2020-12-30 Esteve Pharmaceuticals Sa Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a
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
DK3688147T3 (da) * 2018-12-18 2022-12-19 Neuway Pharma Gmbh Hidtil ukendt lægemiddelindgivelsessystem og fremgangsmåder til tilvejebringelse heraf
US20220042043A1 (en) * 2018-12-20 2022-02-10 Esteve Pharmaceuticals, S.A. Recombinant vectors for the long term treatment of mucchopolysacharidosis
WO2021231491A1 (en) * 2020-05-11 2021-11-18 University Of Florida Research Foundation, Incorporated Disease correction by delivery of aav8 vectors expressing codon optimized naglu
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
IL300887A (en) * 2020-08-28 2023-04-01 Japan Chem Res α–N–ACETYLGLUCOSAMINIDASE MUTATION

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CN101522899B (zh) * 2006-10-10 2012-07-25 百疗医株式会社 安全性改善的表达载体
EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
US8775146B2 (en) * 2010-07-22 2014-07-08 Shire Human Genetic Therapies, Inc. Crystal structure of human alpha-N-acetylglucosaminidase
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
US9579366B2 (en) * 2011-10-12 2017-02-28 Alexion Pharmaceuticals, Inc. Recombinant human NaGlu protein and uses thereof
CN102618578A (zh) * 2012-03-16 2012-08-01 中国人民解放军第四军医大学 用于促进汉坦病毒融合蛋白g2s0.7有效提呈的重组腺病毒高表达载体
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EP3137497B1 (en) * 2014-05-02 2021-04-07 Genzyme Corporation Aav vectors for retinal and cns gene therapy
EP3645021A4 (en) * 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
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Also Published As

Publication number Publication date
CN107002095B (zh) 2021-03-02
TW201625793A (zh) 2016-07-16
PT3143146T (pt) 2020-08-27
CN107002095A (zh) 2017-08-01
AU2015261519A1 (en) 2016-11-24
AU2015261519A2 (en) 2017-02-02
EP3143146B1 (en) 2020-05-20
NZ726316A (en) 2023-07-28
AU2015261519B2 (en) 2021-08-05
RU2718248C2 (ru) 2020-03-31
CA2947468C (en) 2022-10-25
IL248538B (en) 2021-01-31
ES2823948T3 (es) 2021-05-10
SA516380290B1 (ar) 2021-05-17
EP3143146A1 (en) 2017-03-22
CA2947468A1 (en) 2015-11-19
UA123985C2 (uk) 2021-07-07
KR102446169B1 (ko) 2022-09-22
JP2017523802A (ja) 2017-08-24
US20170088859A1 (en) 2017-03-30
KR20170026358A (ko) 2017-03-08
RU2016149206A3 (es) 2019-04-25
SG11201609314XA (en) 2016-12-29
PH12016502248A1 (en) 2017-02-27
US11149285B2 (en) 2021-10-19
WO2015173308A1 (en) 2015-11-19
BR112016025819A2 (pt) 2018-07-03
RU2016149206A (ru) 2018-06-14
AR100419A1 (es) 2016-10-05
JP6634073B2 (ja) 2020-01-22
BR112016025819B1 (pt) 2022-08-16
TWI701332B (zh) 2020-08-11
MY183472A (en) 2021-02-19
IL248538A0 (en) 2016-12-29

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