JP7059285B2 - ムコ多糖症の治療のためのアデノ随伴ウイルスベクター - Google Patents

ムコ多糖症の治療のためのアデノ随伴ウイルスベクター Download PDF

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JP7059285B2
JP7059285B2 JP2019538714A JP2019538714A JP7059285B2 JP 7059285 B2 JP7059285 B2 JP 7059285B2 JP 2019538714 A JP2019538714 A JP 2019538714A JP 2019538714 A JP2019538714 A JP 2019538714A JP 7059285 B2 JP7059285 B2 JP 7059285B2
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JP2019528793A5 (enExample
JP2019528793A (ja
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レチャ，カルレス ロカ
ア． アウリゴト－メンドンカ，ビルヒニア
トゥベルト，ファティマ ボスチ
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Universitat Autonòma de Barcelona
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
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    • 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
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    • 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

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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)
  • 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 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)
  • Enzymes And Modification Thereof (AREA)
JP2019538714A 2016-09-30 2017-09-22 ムコ多糖症の治療のためのアデノ随伴ウイルスベクター Expired - Fee Related JP7059285B2 (ja)

Applications Claiming Priority (3)

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

Publications (3)

Publication Number Publication Date
JP2019528793A JP2019528793A (ja) 2019-10-17
JP2019528793A5 JP2019528793A5 (enExample) 2020-11-12
JP7059285B2 true JP7059285B2 (ja) 2022-04-25

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JP2019538714A Expired - Fee Related JP7059285B2 (ja) 2016-09-30 2017-09-22 ムコ多糖症の治療のためのアデノ随伴ウイルスベクター

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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 (15)

* 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
JP2016523835A (ja) * 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma 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
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (ｆｇｆ21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
MY204731A (en) * 2018-05-30 2024-09-11 Esteve Pharmaceuticals Sa Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a
EP3820537A4 (en) * 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
IL283452B2 (en) 2018-11-26 2026-04-01 Univ Barcelona Autonoma FIBROBLAST GROWTH FACTOR 21 (FGF21)-BASED GENE THERAPY
ES2948620T3 (es) 2019-07-10 2023-09-14 Masonic Medical Res Laboratory Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo
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
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
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 (5)

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JP2013531490A (ja) 2010-06-10 2013-08-08 ラボラトリオス・デル・ドクトル・エステベ・ソシエダッド・アノニマ 疾患の治療のためのベクター及び配列
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
JP2015513528A (ja) 2012-02-07 2015-05-14 グローバル・バイオ・セラピューティクス・ユーエスエイ・インコーポレイテッドＧｌｏｂａｌ Ｂｉｏ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｕｓａ，Ｉｎｃ． 核酸送達の区画化方法ならびにその組成物および使用
WO2015173308A1 (en) 2014-05-14 2015-11-19 Laboratorios Del Dr. Esteve S.A. Adenoassociated virus vectors for the treatment of lysosomal storage disorders
WO2016130591A2 (en) 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex

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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
CN110496230A (zh) * 2003-02-11 2019-11-26 夏尔人类遗传性治疗公司 使用甲酰-甘氨酸生成酶(fge)对多种硫酸酯酶缺乏症和其它病症进行诊断和治疗
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
US9981021B1 (en) * 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof

Patent Citations (5)

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Publication number Priority date Publication date Assignee Title
JP2013531490A (ja) 2010-06-10 2013-08-08 ラボラトリオス・デル・ドクトル・エステベ・ソシエダッド・アノニマ 疾患の治療のためのベクター及び配列
JP2015513528A (ja) 2012-02-07 2015-05-14 グローバル・バイオ・セラピューティクス・ユーエスエイ・インコーポレイテッドＧｌｏｂａｌ Ｂｉｏ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｕｓａ，Ｉｎｃ． 核酸送達の区画化方法ならびにその組成物および使用
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
WO2015173308A1 (en) 2014-05-14 2015-11-19 Laboratorios Del Dr. Esteve S.A. Adenoassociated virus vectors for the treatment of lysosomal storage disorders
WO2016130591A2 (en) 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex

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ES2859661T3 (es) 2021-10-04
PT3519569T (pt) 2021-03-04
WO2018060097A1 (en) 2018-04-05
CN109863243B (zh) 2023-07-28
AR109752A1 (es) 2019-01-23
TW201814044A (zh) 2018-04-16
AU2017333336A1 (en) 2019-04-11
US20190231900A1 (en) 2019-08-01
EP3519569A1 (en) 2019-08-07
EP3519569B1 (en) 2020-12-02
US11338046B2 (en) 2022-05-24
JP2019528793A (ja) 2019-10-17
AU2017333336B2 (en) 2023-11-09
CN109863243A (zh) 2019-06-07

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