CO7010783A2 - Proteína naglu humana recombinante y usos de la misma - Google Patents

Proteína naglu humana recombinante y usos de la misma

Info

Publication number
CO7010783A2
CO7010783A2 CO14098853A CO14098853A CO7010783A2 CO 7010783 A2 CO7010783 A2 CO 7010783A2 CO 14098853 A CO14098853 A CO 14098853A CO 14098853 A CO14098853 A CO 14098853A CO 7010783 A2 CO7010783 A2 CO 7010783A2
Authority
CO
Colombia
Prior art keywords
naglu
proteins
mixture
recombinant human
cells
Prior art date
Application number
CO14098853A
Other languages
English (en)
Inventor
Anthony Quinn
Markley C Leavitt
Xia Zhinan
Joseph Victor Rutkowski
Original Assignee
Synageva Biopharma Corp
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 Synageva Biopharma Corp filed Critical Synageva Biopharma Corp
Publication of CO7010783A2 publication Critical patent/CO7010783A2/es

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    • 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)
    • A61K38/47—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7024—Esters of saccharides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/02—Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/12—Antidiarrhoeals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/20—Hypnotics; Sedatives
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • 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)
    • C12N9/24—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)
    • 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)
    • C12N9/24—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Anesthesiology (AREA)
  • Transplantation (AREA)
  • Obesity (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)

Abstract

La presente invención proporciona composiciones que comprenden una mezcla aislada de proteínas NaGlu humanas recombinantes en la que una cantidad sustancial de las proteínas NaGlu de la mezcla tiene niveles superiores de manosa fosforilada que hacen que las proteínas se introduzcan eficazmente en las células humanas. La presente invención también proporciona métodos de producción de dicha mezcla de proteínas NaGlu, vectores usados en la transgénesis y expresión, células huésped que albergan dichos vectores y métodos de aislamiento y purificación de la mezcla de proteínas NaGlu. La invención proporciona además métodos de tratamiento de enfermedades asociadas con NaGlu.
CO14098853A 2011-10-12 2014-05-08 Proteína naglu humana recombinante y usos de la misma CO7010783A2 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201161546248P 2011-10-12 2011-10-12

Publications (1)

Publication Number Publication Date
CO7010783A2 true CO7010783A2 (es) 2014-07-31

Family

ID=48082738

Family Applications (1)

Application Number Title Priority Date Filing Date
CO14098853A CO7010783A2 (es) 2011-10-12 2014-05-08 Proteína naglu humana recombinante y usos de la misma

Country Status (18)

Country Link
US (3) US20140255383A1 (es)
EP (1) EP2766477B1 (es)
JP (1) JP6329483B2 (es)
KR (1) KR20140108519A (es)
CN (1) CN103958676A (es)
AU (1) AU2012322811A1 (es)
BR (1) BR112014008873A2 (es)
CA (1) CA2852027A1 (es)
CL (1) CL2014000927A1 (es)
CO (1) CO7010783A2 (es)
HK (1) HK1200489A1 (es)
IL (1) IL232073A0 (es)
IN (1) IN2014MN00740A (es)
MX (1) MX2014004359A (es)
RU (1) RU2014117291A (es)
SG (2) SG11201401457SA (es)
TW (1) TW201321515A (es)
WO (1) WO2013055888A2 (es)

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EP2485761B1 (en) 2009-10-09 2019-02-27 Armagen, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
AR082953A1 (es) 2010-09-09 2013-01-23 Synageva Biopharma Corp Metodos para tratar deficiencia de acido lisosomal lipasa en pacientes
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
AU2012322811A1 (en) * 2011-10-12 2014-05-01 Synageva Biopharma Corp. Recombinant human NaGlu protein and uses thereof
CA2857647C (en) 2011-12-02 2022-04-19 Armagen Technologies, Inc. Methods and compositions for increasing arylsulfatase a activity in the cns
SG11201509419QA (en) * 2013-05-15 2015-12-30 Univ Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
TWI642782B (zh) * 2013-10-23 2018-12-01 健臻公司 重組醣蛋白及其用途
RU2718248C2 (ru) * 2014-05-14 2020-03-31 Эстев Фармасьютикалс, С.А. Аденоассоциированные вирусные векторы для лечения лизосомных болезней накопления
EP3160494B1 (en) * 2014-06-25 2019-11-20 Shire Human Genetic Therapies, Inc. Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase
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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
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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
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Also Published As

Publication number Publication date
KR20140108519A (ko) 2014-09-11
NZ623690A (en) 2016-08-26
WO2013055888A3 (en) 2013-06-13
US20170216412A1 (en) 2017-08-03
SG11201401457SA (en) 2014-05-29
US9579366B2 (en) 2017-02-28
JP6329483B2 (ja) 2018-05-23
JP2014534962A (ja) 2014-12-25
RU2014117291A (ru) 2015-11-20
EP2766477A2 (en) 2014-08-20
AU2012322811A1 (en) 2014-05-01
MX2014004359A (es) 2014-05-28
BR112014008873A2 (pt) 2020-09-15
IN2014MN00740A (es) 2015-05-22
CA2852027A1 (en) 2013-04-18
US20130095092A1 (en) 2013-04-18
HK1200489A1 (en) 2015-08-07
US20140255383A1 (en) 2014-09-11
EP2766477B1 (en) 2018-09-26
SG10201510106PA (en) 2016-01-28
EP2766477A4 (en) 2015-07-15
WO2013055888A2 (en) 2013-04-18
CN103958676A (zh) 2014-07-30
TW201321515A (zh) 2013-06-01
CL2014000927A1 (es) 2014-11-14
IL232073A0 (en) 2014-05-28

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