IN2014MN00740A - - Google Patents

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Publication number
IN2014MN00740A
IN2014MN00740A IN740MUN2014A IN2014MN00740A IN 2014MN00740 A IN2014MN00740 A IN 2014MN00740A IN 740MUN2014 A IN740MUN2014 A IN 740MUN2014A IN 2014MN00740 A IN2014MN00740 A IN 2014MN00740A
Authority
IN
India
Prior art keywords
proteins
naglu
mixture
present
methods
Prior art date
Application number
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 IN2014MN00740A publication Critical patent/IN2014MN00740A/en

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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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • 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)
  • Anesthesiology (AREA)
  • Obesity (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Transplantation (AREA)

Abstract

The present invention provides compositions comprising an isolated mixture of recombinant human NaGlu proteins in which a substantial amount of the NaGlu proteins in the mixture has increased levels of phosphorylated mannose that confer the proteins to be efficiently internalized into human cells. The present invention also provides methods of producing such mixture of NaGlu proteins vectors used in transgenesis and expression host cells harboring such vectors and methods of isolating and purifying the mixture of NaGlu proteins. The invention further provides methods of treating NaGlu associated diseases.
IN740MUN2014 2011-10-12 2012-10-11 IN2014MN00740A (en)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
IN2014MN00740A true IN2014MN00740A (en) 2015-05-22

Family

ID=48082738

Family Applications (1)

Application Number Title Priority Date Filing Date
IN740MUN2014 IN2014MN00740A (en) 2011-10-12 2012-10-11

Country Status (18)

Country Link
US (3) US9579366B2 (en)
EP (1) EP2766477B1 (en)
JP (1) JP6329483B2 (en)
KR (1) KR20140108519A (en)
CN (1) CN103958676A (en)
AU (1) AU2012322811A1 (en)
BR (1) BR112014008873A2 (en)
CA (1) CA2852027A1 (en)
CL (1) CL2014000927A1 (en)
CO (1) CO7010783A2 (en)
HK (1) HK1200489A1 (en)
IL (1) IL232073A0 (en)
IN (1) IN2014MN00740A (en)
MX (1) MX2014004359A (en)
RU (1) RU2014117291A (en)
SG (2) SG10201510106PA (en)
TW (1) TW201321515A (en)
WO (1) WO2013055888A2 (en)

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JP5901877B2 (en) 2007-07-27 2016-04-13 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． Methods and compositions for increasing [alpha] -L-iduronidase activity in the central nervous system
ES2725200T3 (en) 2009-10-09 2019-09-20 Armagen Inc Methods and compositions to increase the activity of iduronate 2-sulfatase in the CNS
NZ715014A (en) 2010-09-09 2018-10-26 Synageva Biopharma Corp Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
WO2012112681A1 (en) 2011-02-15 2012-08-23 Shire Human Genetic Therapies, Inc. Methods for treating lysosomal acid lipase deficiency
AU2012322811A1 (en) * 2011-10-12 2014-05-01 Synageva Biopharma Corp. Recombinant human NaGlu protein and uses thereof
EP4338797A3 (en) 2011-12-02 2024-06-12 Armagen, Inc. Methods and compositions for increasing arylsulfatase a activity in the cns
CA2912678C (en) 2013-05-15 2023-10-10 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
TWI793159B (en) * 2013-10-23 2023-02-21 美商健臻公司 Recombinant glycoproteins and uses thereof
KR102446169B1 (en) * 2014-05-14 2022-09-22 에스테베 파마슈티칼스 에스에이 Adenoassociated virus vectors for the treatment of lysosomal storage disorders
US20170267986A1 (en) * 2014-06-25 2017-09-21 Shire Human Genetic Therapies, Inc. Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase
BR112017002741A2 (en) 2014-08-11 2018-02-27 Shire Human Genetic Therapies, Inc mannose-6-phosphate containing peptides fused to lysosomal enzymes
AU2015323966A1 (en) 2014-09-29 2017-03-16 Alexion Pharmaceuticals, Inc. Methods of treating mucopolysaccharidosis IIIB (MPSIIIB)
CN104524662B (en) * 2014-12-31 2017-02-22 迈得医疗工业设备股份有限公司 Feeding mechanism for medical needle base assembling
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
US20190083583A1 (en) * 2016-01-29 2019-03-21 Alexion Pharmaceuticals, Inc. Enzyme replacement therapy in mucopolysaccharidosis iiib patients
WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
US10633705B2 (en) * 2016-10-14 2020-04-28 Alexion Pharmaceuticals, Inc. N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods
WO2019060662A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
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Also Published As

Publication number Publication date
BR112014008873A2 (en) 2020-09-15
SG10201510106PA (en) 2016-01-28
US9579366B2 (en) 2017-02-28
NZ623690A (en) 2016-08-26
WO2013055888A3 (en) 2013-06-13
CN103958676A (en) 2014-07-30
RU2014117291A (en) 2015-11-20
US20170216412A1 (en) 2017-08-03
CA2852027A1 (en) 2013-04-18
CO7010783A2 (en) 2014-07-31
WO2013055888A2 (en) 2013-04-18
AU2012322811A1 (en) 2014-05-01
KR20140108519A (en) 2014-09-11
EP2766477B1 (en) 2018-09-26
HK1200489A1 (en) 2015-08-07
CL2014000927A1 (en) 2014-11-14
US20140255383A1 (en) 2014-09-11
JP2014534962A (en) 2014-12-25
JP6329483B2 (en) 2018-05-23
IL232073A0 (en) 2014-05-28
MX2014004359A (en) 2014-05-28
US20130095092A1 (en) 2013-04-18
TW201321515A (en) 2013-06-01
SG11201401457SA (en) 2014-05-29
EP2766477A4 (en) 2015-07-15
EP2766477A2 (en) 2014-08-20

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