JP2014506787A - シアル酸産生を増加させて、シアル酸に関連した病状を治療する方法および組成物 - Google Patents

シアル酸産生を増加させて、シアル酸に関連した病状を治療する方法および組成物 Download PDF

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JP2014506787A
JP2014506787A JP2013552609A JP2013552609A JP2014506787A JP 2014506787 A JP2014506787 A JP 2014506787A JP 2013552609 A JP2013552609 A JP 2013552609A JP 2013552609 A JP2013552609 A JP 2013552609A JP 2014506787 A JP2014506787 A JP 2014506787A
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gne
nucleic acid
cell
seq
peptide
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JP2014506787A5 (https=
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ダービッシュ，ダニエル
ヴァルレス―アヨウブ，ヤディラ
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エイチアイビーエム リサーチ グループ，インコーポレイテッド
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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
    • 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
    • A61K38/52—Isomerases (5)
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/42—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests having means for desensitising skin, for protruding skin to facilitate piercing, or for locating point where body is to be pierced
    • A61M5/425—Protruding skin to facilitate piercing, e.g. vacuum cylinders, vein immobilising means
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00—General protective or antinoxious agents
    • A61P39/06—Free radical scavengers or antioxidants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Vascular Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Toxicology (AREA)
  • Communicable Diseases (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Cardiology (AREA)
  • Obesity (AREA)
  • Oncology (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Anesthesiology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2013552609A 2011-02-01 2012-02-01 シアル酸産生を増加させて、シアル酸に関連した病状を治療する方法および組成物 Pending JP2014506787A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161438585P 2011-02-01 2011-02-01
US61/438,585 2011-02-01
PCT/US2012/023536 WO2012106465A2 (en) 2011-02-01 2012-02-01 Methods and compositions for increasing sialic acid production and treating sialic related disease conditions

Related Child Applications (1)

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JP2017092571A Division JP2017158577A (ja) 2011-02-01 2017-05-08 シアル酸産生を増加させて、シアル酸に関連した病状を治療する方法および組成物

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JP2014506787A true JP2014506787A (ja) 2014-03-20
JP2014506787A5 JP2014506787A5 (https=) 2016-07-28

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JP2013552609A Pending JP2014506787A (ja) 2011-02-01 2012-02-01 シアル酸産生を増加させて、シアル酸に関連した病状を治療する方法および組成物
JP2017092571A Pending JP2017158577A (ja) 2011-02-01 2017-05-08 シアル酸産生を増加させて、シアル酸に関連した病状を治療する方法および組成物

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US (2) US20130030040A1 (https=)
EP (2) EP3202425A1 (https=)
JP (2) JP2014506787A (https=)
KR (2) KR20140034150A (https=)
AU (2) AU2012212162A1 (https=)
CA (1) CA2863578C (https=)
IL (1) IL227773B (https=)
MX (1) MX365095B (https=)
WO (1) WO2012106465A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12559533B2 (en) 2017-05-24 2026-02-24 Universitat Autònoma De Barcelona Viral expression construct comprising a fibroblast growth factor 21 (FGF21) coding sequence

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014031837A1 (en) * 2012-08-22 2014-02-27 The Regents Of The University Of California Compositions and methods for enhancing sialic acid levels in tissue
WO2014077863A1 (en) * 2012-11-19 2014-05-22 Nature Technology Corporation Replicative minicircle vectors with improved expression
US20140256639A1 (en) * 2013-02-14 2014-09-11 The Regents Of The University Of California Peptoid neutralizing agents
CN103088090B (zh) * 2013-03-01 2014-06-04 南京工业大学 一种n-乙酰葡萄糖胺异构酶在生产n-乙酰甘露糖胺中的应用
FI3218005T3 (fi) 2014-11-12 2023-03-31 Seagen Inc Glykaanin kanssa vuorovaikutteisia yhdisteitä ja käyttömenetelmiä
CA3002097A1 (en) 2015-11-12 2017-05-18 Siamab Therapeutics, Inc. Glycan-interacting compounds and methods of use
WO2018094143A1 (en) 2016-11-17 2018-05-24 Siamab Therapeutics, Inc. Glycan-interacting compounds and methods of use
WO2018160909A1 (en) 2017-03-03 2018-09-07 Siamab Therapeutics, Inc. Glycan-interacting compounds and methods of use
CN108285886A (zh) * 2018-01-30 2018-07-17 江南大学 重组枯草芽孢杆菌全细胞转化生产n-乙酰神经氨酸的方法
US11865165B2 (en) 2018-09-18 2024-01-09 Daniel DARVISH GNE as a therapeutic agent

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070073264A1 (en) * 2003-09-26 2007-03-29 The Trustees Of The University Of Pennsylvania Methods, compositions and apparatus for delivering heterologous molecules to cells
US20090298112A1 (en) * 2008-05-28 2009-12-03 Hibm Research Group, Inc. Mouse model and treatment of hereditary inclusion body myopathy
JP2010519184A (ja) * 2007-02-07 2010-06-03 グラダリス インク． シアル酸産生を調節し、遺伝性封入体ミオパチーを治療する方法及び組成物
US20100184158A1 (en) * 2009-01-21 2010-07-22 James Arthur Williams DNA Plasmids with improved copy number

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070173465A9 (en) 1995-10-11 2007-07-26 Monahan Sean D Expression of zeta negative and zeta positive nucleic acids using a dystrophin gene
AU2001236739A1 (en) * 2000-02-08 2001-08-20 Genentech Inc. Improved sialylation of glycoproteins
WO2004071177A2 (en) * 2003-02-11 2004-08-26 Arizona Board Of Regents, Acting For And On Behalf Of, Arizona State University Sialylation of glycoproteins in plants
WO2008001023A1 (en) 2006-06-27 2008-01-03 Gigabeam Corporation Cellular communication system
WO2008124934A1 (en) 2007-04-17 2008-10-23 National Research Council Of Canada Constructs for enhancement of gene expression in muscle
EP2324110A4 (en) 2008-09-09 2013-01-09 Univ California ELIMINATION OF A CONTAMINATING NON-HUMAN SIALIC ACID BY METABOLISM COMPETITION
WO2011096750A2 (en) * 2010-02-08 2011-08-11 Korea Advanced Institute Of Science And Technology Method for preparing recombinant glycoproteins with high sialic acid content

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070073264A1 (en) * 2003-09-26 2007-03-29 The Trustees Of The University Of Pennsylvania Methods, compositions and apparatus for delivering heterologous molecules to cells
JP2010519184A (ja) * 2007-02-07 2010-06-03 グラダリス インク． シアル酸産生を調節し、遺伝性封入体ミオパチーを治療する方法及び組成物
US20090298112A1 (en) * 2008-05-28 2009-12-03 Hibm Research Group, Inc. Mouse model and treatment of hereditary inclusion body myopathy
US20100184158A1 (en) * 2009-01-21 2010-07-22 James Arthur Williams DNA Plasmids with improved copy number

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12559533B2 (en) 2017-05-24 2026-02-24 Universitat Autònoma De Barcelona Viral expression construct comprising a fibroblast growth factor 21 (FGF21) coding sequence

Also Published As

Publication number Publication date
KR102257741B1 (ko) 2021-05-28
IL227773B (en) 2021-03-25
CA2863578A1 (en) 2012-08-09
AU2012212162A1 (en) 2013-08-29
IL227773A0 (en) 2013-09-30
EP2670442A2 (en) 2013-12-11
WO2012106465A3 (en) 2014-04-24
KR20190108178A (ko) 2019-09-23
CA2863578C (en) 2021-10-26
EP3202425A1 (en) 2017-08-09
US20130030040A1 (en) 2013-01-31
KR20140034150A (ko) 2014-03-19
MX2013008948A (es) 2014-02-18
EP2670442A4 (en) 2015-07-29
US10098969B2 (en) 2018-10-16
US20140275230A1 (en) 2014-09-18
MX365095B (es) 2019-05-22
WO2012106465A2 (en) 2012-08-09
JP2017158577A (ja) 2017-09-14
AU2017203027B2 (en) 2019-08-15
AU2017203027A1 (en) 2017-07-20

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