SG11201609314XA - Adenoassociated virus vectors for the treatment of lysosomal storage disorders - Google Patents

Adenoassociated virus vectors for the treatment of lysosomal storage disorders

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Publication number
SG11201609314XA
SG11201609314XA SG11201609314XA SG11201609314XA SG11201609314XA SG 11201609314X A SG11201609314X A SG 11201609314XA SG 11201609314X A SG11201609314X A SG 11201609314XA SG 11201609314X A SG11201609314X A SG 11201609314XA SG 11201609314X A SG11201609314X A SG 11201609314XA
Authority
SG
Singapore
Prior art keywords
treatment
virus vectors
lysosomal storage
storage disorders
adenoassociated virus
Prior art date
Application number
SG11201609314XA
Other languages
English (en)
Inventor
Tubert M Fàtima Bosch
Mendoça M Virginia Haurigot
Sanchez Albert Ribera
Original Assignee
Esteve Labor Dr
Uni Autònoma De Barcelona
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, Uni Autònoma De Barcelona filed Critical Esteve Labor Dr
Publication of SG11201609314XA publication Critical patent/SG11201609314XA/en

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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
    • 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
    • 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)
    • C12N9/24Hydrolases (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)
    • 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)
    • C12N9/24Hydrolases (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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • 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
    • 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
    • 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
    • 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
    • C12N2830/008Vector 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

Landscapes

  • 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)
SG11201609314XA 2014-05-14 2015-05-13 Adenoassociated virus vectors for the treatment of lysosomal storage disorders SG11201609314XA (en)

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
SG11201609314XA true SG11201609314XA (en) 2016-12-29

Family

ID=50771225

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201609314XA SG11201609314XA (en) 2014-05-14 2015-05-13 Adenoassociated virus vectors for the treatment of lysosomal storage disorders

Country Status (22)

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

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* Cited by examiner, † Cited by third party
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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
BR112016002045A2 (pt) 2013-07-29 2017-08-29 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
CA2969599A1 (en) 2014-12-05 2016-06-09 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
US11458211B2 (en) 2016-07-12 2022-10-04 The University Of Manchester Gene therapy
EP3519569B1 (en) 2016-09-30 2020-12-02 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
CA3083761A1 (en) * 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iiib
CN112513072A (zh) * 2018-04-27 2021-03-16 威斯康星州医药大学股份有限公司 慢病毒载体转化的t-rapa细胞在改善溶酶体贮积症中的应用
CN113474453B (zh) * 2018-05-30 2024-01-12 埃斯蒂维制药有限公司 用于治疗iv a型粘多糖的腺相关病毒载体
US20220007621A1 (en) * 2018-11-15 2022-01-13 Esteve Pharmaceuticals, S.A. Animal model of mucopolysaccharidoses type iva
US11279938B2 (en) * 2018-12-18 2022-03-22 Neuway Pharma Gmbh Drug delivery system and methods providing thereof
WO2020127678A1 (en) * 2018-12-20 2020-06-25 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
MX2023002346A (es) * 2020-08-28 2023-03-22 Japan Chem Res Mutante de a-n-acetilglucosaminidasa.

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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
WO2012012742A2 (en) 2010-07-22 2012-01-26 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
KR20140108519A (ko) * 2011-10-12 2014-09-11 시나게바 바이오파르마, 코포레이션 재조합 인간 naglu 단백질 및 이의 용도
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
US20140010861A1 (en) * 2012-04-02 2014-01-09 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
PT3137497T (pt) * 2014-05-02 2021-07-12 Genzyme Corp Vetores de aav para terapia genética na retina e snc
EP3645021A4 (en) * 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
CN109762831B (zh) * 2019-01-24 2021-04-27 北京瑞希罕见病基因治疗技术研究所 用于治疗3a型粘多糖贮积症的基因药物构建体

Also Published As

Publication number Publication date
CN107002095A (zh) 2017-08-01
AR100419A1 (es) 2016-10-05
JP6634073B2 (ja) 2020-01-22
AU2015261519A2 (en) 2017-02-02
EP3143146B1 (en) 2020-05-20
IL248538A0 (en) 2016-12-29
CA2947468C (en) 2022-10-25
KR20170026358A (ko) 2017-03-08
TW201625793A (zh) 2016-07-16
NZ726316A (en) 2023-07-28
MY183472A (en) 2021-02-19
CN107002095B (zh) 2021-03-02
CA2947468A1 (en) 2015-11-19
US20170088859A1 (en) 2017-03-30
WO2015173308A1 (en) 2015-11-19
BR112016025819B1 (pt) 2022-08-16
ES2823948T3 (es) 2021-05-10
PH12016502248A1 (en) 2017-02-27
KR102446169B1 (ko) 2022-09-22
JP2017523802A (ja) 2017-08-24
RU2016149206A3 (pt) 2019-04-25
PT3143146T (pt) 2020-08-27
UA123985C2 (uk) 2021-07-07
AU2015261519B2 (en) 2021-08-05
EP3143146A1 (en) 2017-03-22
RU2016149206A (ru) 2018-06-14
US11149285B2 (en) 2021-10-19
SA516380290B1 (ar) 2021-05-17
AU2015261519A1 (en) 2016-11-24
IL248538B (en) 2021-01-31
TWI701332B (zh) 2020-08-11
MX2016014773A (es) 2017-05-04
BR112016025819A2 (pt) 2018-07-03
RU2718248C2 (ru) 2020-03-31

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