KR102446169B1 - 리소좀 저장 장애의 치료를 위한 아데노연관 바이러스 벡터 - Google Patents

리소좀 저장 장애의 치료를 위한 아데노연관 바이러스 벡터 Download PDF

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KR102446169B1
KR102446169B1 KR1020167034776A KR20167034776A KR102446169B1 KR 102446169 B1 KR102446169 B1 KR 102446169B1 KR 1020167034776 A KR1020167034776 A KR 1020167034776A KR 20167034776 A KR20167034776 A KR 20167034776A KR 102446169 B1 KR102446169 B1 KR 102446169B1
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seq
cag
vector
acetylglucosaminidase
aav9
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KR1020167034776A
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Korean (ko)
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KR20170026358A (ko
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엠 파티마 보쉬 투베르트
멘도카 엠 버지니아 하우리고트
알버트 리베라 산체스
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에스테베 파마슈티칼스 에스에이
유니버시타트 아우토노마 데 바르셀로나
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    • 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
    • A—HUMAN NECESSITIES
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
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    • 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
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
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    • A—HUMAN NECESSITIES
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    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2474—Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
    • C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Psychology (AREA)
  • Diabetes (AREA)
  • Urology & Nephrology (AREA)
KR1020167034776A 2014-05-14 2015-05-13 리소좀 저장 장애의 치료를 위한 아데노연관 바이러스 벡터 KR102446169B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14382171.8 2014-05-14
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 (2)

Publication Number Publication Date
KR20170026358A KR20170026358A (ko) 2017-03-08
KR102446169B1 true KR102446169B1 (ko) 2022-09-22

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

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CA3128549A1 (en) 2010-07-12 2012-01-19 Universitat 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
US10081802B2 (en) 2013-07-29 2018-09-25 Danisco Us Inc. Variant Enzymes
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3015135B1 (en) 2014-10-29 2020-09-09 Noxell Corporation Hair colouration with a cationic or anionic coloured polymer, method and kit thereof
EP3015134B1 (en) 2014-10-29 2020-08-26 Noxell Corporation Hair colouration with a cationic coloured polymer, method and kit thereof
EP3227449B1 (en) 2014-12-05 2020-07-15 Universitat Autònoma de Barcelona Viral vectors for the treatment of diabetes
EP3978614A3 (en) 2015-01-07 2022-07-27 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
WO2018011572A1 (en) * 2016-07-12 2018-01-18 The University Of Manchester Gene therapy
JP7059285B2 (ja) * 2016-09-30 2022-04-25 エステベ プアルマセウティカルス， エッセ．ア． 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
MX2020005673A (es) * 2017-11-30 2020-12-03 Univ Pennsylvania Terapia génica para mucopolisacaridosis iiib.
SG11202010645VA (en) * 2018-04-27 2020-11-27 Medical College Wisconsin Inc Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
UA128788C2 (uk) * 2018-05-30 2024-10-23 Естеве Фармасьютікалз, С.А. Вектор аденоасоційованого вірусу для лікування мукополісахаридозу типу iva
WO2020099548A1 (en) * 2018-11-15 2020-05-22 Esteve Pharmaceuticals, S.A. Animal model of mucopolysaccharidoses type iva
CN111587289B (zh) * 2018-12-18 2024-04-12 纽卫制药有限公司 一种新型药品递送系统及其提供方法
EP3897695A1 (en) * 2018-12-20 2021-10-27 Esteve Pharmaceuticals, S.A. Recombinant vectors for the long term treatment of mucchopolysacharidosis
US20230183741A1 (en) * 2020-05-11 2023-06-15 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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PH12016502248A1 (en) 2017-02-27
EP3143146B1 (en) 2020-05-20
CA2947468A1 (en) 2015-11-19
TWI701332B (zh) 2020-08-11
IL248538B (en) 2021-01-31
AU2015261519A1 (en) 2016-11-24
RU2016149206A (ru) 2018-06-14
SG11201609314XA (en) 2016-12-29
TW201625793A (zh) 2016-07-16
WO2015173308A1 (en) 2015-11-19
CA2947468C (en) 2022-10-25
US11149285B2 (en) 2021-10-19
US20170088859A1 (en) 2017-03-30
BR112016025819A2 (pt) 2018-07-03
AU2015261519B2 (en) 2021-08-05
BR112016025819B1 (pt) 2022-08-16
IL248538A0 (en) 2016-12-29
AR100419A1 (es) 2016-10-05
AU2015261519A2 (en) 2017-02-02
MX2016014773A (es) 2017-05-04
JP6634073B2 (ja) 2020-01-22
NZ726316A (en) 2023-07-28
JP2017523802A (ja) 2017-08-24
PT3143146T (pt) 2020-08-27
CN107002095B (zh) 2021-03-02
CN107002095A (zh) 2017-08-01
EP3143146A1 (en) 2017-03-22
RU2718248C2 (ru) 2020-03-31
UA123985C2 (uk) 2021-07-07
ES2823948T3 (es) 2021-05-10
KR20170026358A (ko) 2017-03-08
SA516380290B1 (ar) 2021-05-17
RU2016149206A3 (pt-PT) 2019-04-25
MY183472A (en) 2021-02-19

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