JP6329483B2 - 組換えヒトnagluタンパク質およびその利用 - Google Patents

組換えヒトnagluタンパク質およびその利用 Download PDF

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JP6329483B2
JP6329483B2 JP2014535865A JP2014535865A JP6329483B2 JP 6329483 B2 JP6329483 B2 JP 6329483B2 JP 2014535865 A JP2014535865 A JP 2014535865A JP 2014535865 A JP2014535865 A JP 2014535865A JP 6329483 B2 JP6329483 B2 JP 6329483B2
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rhnaglu
naglu
protein
composition
cells
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JP2014534962A (ja
JP2014534962A5 (enExample
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クィン,アンソニー
リービット,マークリー,シー.
シア,ジーナン
ルトコウスキー,ジョセフ,ビクター
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Synageva Biopharma Corp
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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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7024Esters of saccharides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
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    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
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    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/02Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
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    • 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
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/20Hypnotics; Sedatives
    • AHUMAN NECESSITIES
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • AHUMAN NECESSITIES
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/06Linear peptides containing only normal peptide links having 5 to 11 amino acids
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    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
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    • C07K7/08Linear peptides containing only normal peptide links having 12 to 20 amino acids
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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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    • 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)

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  • Health & Medical Sciences (AREA)
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  • Life Sciences & Earth Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Obesity (AREA)
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JP2014535865A 2011-10-12 2012-10-11 組換えヒトnagluタンパク質およびその利用 Expired - Fee Related JP6329483B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161546248P 2011-10-12 2011-10-12
US61/546,248 2011-10-12
PCT/US2012/059708 WO2013055888A2 (en) 2011-10-12 2012-10-11 Recombinant human naglu protein and uses thereof

Publications (3)

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JP2014534962A JP2014534962A (ja) 2014-12-25
JP2014534962A5 JP2014534962A5 (enExample) 2015-11-26
JP6329483B2 true JP6329483B2 (ja) 2018-05-23

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Country Link
US (3) US20140255383A1 (enExample)
EP (1) EP2766477B1 (enExample)
JP (1) JP6329483B2 (enExample)
KR (1) KR20140108519A (enExample)
CN (1) CN103958676A (enExample)
AU (1) AU2012322811A1 (enExample)
BR (1) BR112014008873A2 (enExample)
CA (1) CA2852027A1 (enExample)
CL (1) CL2014000927A1 (enExample)
CO (1) CO7010783A2 (enExample)
HK (1) HK1200489A1 (enExample)
IL (1) IL232073A0 (enExample)
IN (1) IN2014MN00740A (enExample)
MX (1) MX2014004359A (enExample)
RU (1) RU2014117291A (enExample)
SG (2) SG10201510106PA (enExample)
TW (1) TW201321515A (enExample)
WO (1) WO2013055888A2 (enExample)

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JP5959795B2 (ja) 2006-08-18 2016-08-02 アーメイゲン・テクノロジーズ・インコーポレイテッドArmagen Technologies, Inc. 血液脳関門送達のための物質
EP2997976A1 (en) 2007-07-27 2016-03-23 Armagen Technologies, Inc. Methods and compositions for increasing alpha-l-iduronidase activity in the cns
SI2485761T1 (sl) 2009-10-09 2019-05-31 Armagen, Inc. Postopki in sestavki za povečanje aktivnosti iduronat-2-sulfataze v CŽS
DK2613798T4 (en) 2010-09-09 2018-04-16 Synageva Biopharma Corp USING LYSOSOMAL SUR LIPASE TO TREAT PATIENTS WITHOUT LYSOSOMAL SUR LIPASE
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
CN103958676A (zh) * 2011-10-12 2014-07-30 辛那杰瓦生物制药股份有限公司 重组人naglu蛋白质及其用途
US8486399B2 (en) 2011-12-02 2013-07-16 Armagen Technologies, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
KR20160010526A (ko) * 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
TWI642782B (zh) * 2013-10-23 2018-12-01 健臻公司 重組醣蛋白及其用途
US11149285B2 (en) * 2014-05-14 2021-10-19 Universitat Autónoma De Barcelona Adenoassociated virus vectors for the treatment of lysosomal storage disorders
EP3160494B1 (en) * 2014-06-25 2019-11-20 Shire Human Genetic Therapies, Inc. Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase
MX2017001898A (es) * 2014-08-11 2017-04-11 Shire Human Genetic Therapies Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales.
EP3200816A1 (en) * 2014-09-29 2017-08-09 Alexion Pharmaceuticals, Inc. Methods of treating mucopolysaccharidosis iiib (mpsiiib)
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US10538589B2 (en) * 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
WO2017132675A1 (en) * 2016-01-29 2017-08-03 Alexion Pharmaceuticals, Inc. Enzyme replacement therapy in mucopolysaccharidosis iiib patients
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WO2021194915A1 (en) * 2020-03-23 2021-09-30 The University Of North Carolina At Chapel Hill Aav-naglu vectors for treatment of mucopolysaccharidosis iiib
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

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CN103958676A (zh) * 2011-10-12 2014-07-30 辛那杰瓦生物制药股份有限公司 重组人naglu蛋白质及其用途

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IN2014MN00740A (enExample) 2015-05-22
US9579366B2 (en) 2017-02-28
EP2766477B1 (en) 2018-09-26
HK1200489A1 (en) 2015-08-07
US20130095092A1 (en) 2013-04-18
CA2852027A1 (en) 2013-04-18
WO2013055888A3 (en) 2013-06-13
US20140255383A1 (en) 2014-09-11
TW201321515A (zh) 2013-06-01
CL2014000927A1 (es) 2014-11-14
JP2014534962A (ja) 2014-12-25
SG10201510106PA (en) 2016-01-28
AU2012322811A1 (en) 2014-05-01
SG11201401457SA (en) 2014-05-29
EP2766477A2 (en) 2014-08-20
US20170216412A1 (en) 2017-08-03
NZ623690A (en) 2016-08-26
EP2766477A4 (en) 2015-07-15
CO7010783A2 (es) 2014-07-31
KR20140108519A (ko) 2014-09-11
IL232073A0 (en) 2014-05-28
WO2013055888A2 (en) 2013-04-18
CN103958676A (zh) 2014-07-30
BR112014008873A2 (pt) 2020-09-15
MX2014004359A (es) 2014-05-28
RU2014117291A (ru) 2015-11-20

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