AU2017333336B2 - Adenoassociated virus vectors for the treatment of mucopolysaccharidoses - Google Patents

Adenoassociated virus vectors for the treatment of mucopolysaccharidoses Download PDF

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Publication number
AU2017333336B2
AU2017333336B2 AU2017333336A AU2017333336A AU2017333336B2 AU 2017333336 B2 AU2017333336 B2 AU 2017333336B2 AU 2017333336 A AU2017333336 A AU 2017333336A AU 2017333336 A AU2017333336 A AU 2017333336A AU 2017333336 B2 AU2017333336 B2 AU 2017333336B2
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vector
cag
aav
gns
aav9
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AU2017333336A
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AU2017333336A1 (en
Inventor
Fàtima BOSCH TUBERT
Virginia A. HAURIGOT-MENDONÇA
Carles ROCA LECHA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Universitat Autonoma de Barcelona UAB
Esteve Pharmaceuticals SA
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Universitat Autonoma de Barcelona UAB
Esteve Pharmaceuticals SA
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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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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)
    • C12Y301/06014—N-Acetylglucosamine-6-sulfatase (3.1.6.14)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0362—Animal model for lipid/glucose metabolism, e.g. obesity, type-2 diabetes
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/90—Vector systems having a special element relevant for transcription from vertebrates avian

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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
  • Plant Pathology (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)
AU2017333336A 2016-09-30 2017-09-22 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses Active AU2017333336B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16382450.1 2016-09-30
EP16382450 2016-09-30
PCT/EP2017/074081 WO2018060097A1 (en) 2016-09-30 2017-09-22 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

Publications (2)

Publication Number Publication Date
AU2017333336A1 AU2017333336A1 (en) 2019-04-11
AU2017333336B2 true AU2017333336B2 (en) 2023-11-09

Family

ID=57123944

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2017333336A Active AU2017333336B2 (en) 2016-09-30 2017-09-22 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

Country Status (10)

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

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma 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
ES2900973T3 (es) 2015-01-07 2022-03-21 UNIV AUTòNOMA DE BARCELONA Constructo genético de vector individual que comprende genes de insulina y glucoquinasa
BR112020021903A2 (pt) 2018-04-27 2021-03-02 Rocket Pharmaceuticals, Ltd. terapia genética para degeneração do snc
EP3802803A1 (en) * 2018-05-30 2021-04-14 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a
CA3106010A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
WO2021007473A1 (en) 2019-07-10 2021-01-14 Masonic Medical Research Institute 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
CN116194154A (zh) 2020-08-07 2023-05-30 太空飞船七有限责任公司 使用aav载体的plakophilin-2(pkp2)基因疗法
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015060722A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
WO2016130591A2 (en) * 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US8227212B2 (en) * 2003-02-11 2012-07-24 Shire Human Genetic Therapies, Inc. Cell that expresses a sulfatase and a formylglycine generating enzyme
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
CN107982548A (zh) 2012-02-07 2018-05-04 全球生物疗法有限公司 核酸输送的区室化方法及其组合物和应用
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
GB201322798D0 (en) * 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
MX383227B (es) 2014-05-14 2025-03-13 UNIV AUTòNOMA DE BARCELONA Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico.
US9981021B1 (en) * 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015060722A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
WO2016130591A2 (en) * 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex

Also Published As

Publication number Publication date
AU2017333336A1 (en) 2019-04-11
JP2019528793A (ja) 2019-10-17
CN109863243B (zh) 2023-07-28
US11338046B2 (en) 2022-05-24
TW201814044A (zh) 2018-04-16
EP3519569B1 (en) 2020-12-02
CN109863243A (zh) 2019-06-07
ES2859661T3 (es) 2021-10-04
US20190231900A1 (en) 2019-08-01
AR109752A1 (es) 2019-01-23
EP3519569A1 (en) 2019-08-07
WO2018060097A1 (en) 2018-04-05
JP7059285B2 (ja) 2022-04-25
PT3519569T (pt) 2021-03-04

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