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ZA200701943B - Compositions and their uses for lysosomal enzyme deficiencies - Google Patents

Compositions and their uses for lysosomal enzyme deficiencies Download PDF

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Publication number
ZA200701943B
ZA200701943B ZA200701943A ZA200701943A ZA200701943B ZA 200701943 B ZA200701943 B ZA 200701943B ZA 200701943 A ZA200701943 A ZA 200701943A ZA 200701943 A ZA200701943 A ZA 200701943A ZA 200701943 B ZA200701943 B ZA 200701943B
Authority
ZA
South Africa
Prior art keywords
protein
ygp
disease
payload
composition
Prior art date
Application number
ZA200701943A
Other languages
English (en)
Inventor
Edward I Ginns
Gary R Ostroff
Original Assignee
Univ Massachusetts
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 Univ Massachusetts filed Critical Univ Massachusetts
Publication of ZA200701943B publication Critical patent/ZA200701943B/en

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005Wall or coating material
    • A61K9/5063Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5068Cell membranes or bacterial membranes enclosing drugs
    • 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
    • A61K31/715Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/716Glucans
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6901Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Botany (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
ZA200701943A 2004-09-17 2007-03-06 Compositions and their uses for lysosomal enzyme deficiencies ZA200701943B (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US61087204P 2004-09-17 2004-09-17

Publications (1)

Publication Number Publication Date
ZA200701943B true ZA200701943B (en) 2008-07-30

Family

ID=36060733

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA200701943A ZA200701943B (en) 2004-09-17 2007-03-06 Compositions and their uses for lysosomal enzyme deficiencies

Country Status (14)

Country Link
US (2) US8007814B2 (fr)
EP (1) EP1802289B1 (fr)
JP (1) JP5302537B2 (fr)
KR (1) KR20070054246A (fr)
CN (1) CN101052383B (fr)
AT (1) ATE536175T1 (fr)
AU (1) AU2005284727A1 (fr)
BR (1) BRPI0515332A (fr)
CA (1) CA2580537C (fr)
ES (1) ES2375995T3 (fr)
HK (1) HK1105587A1 (fr)
IL (1) IL181748A (fr)
WO (1) WO2006032039A2 (fr)
ZA (1) ZA200701943B (fr)

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US7740861B2 (en) 2004-06-16 2010-06-22 University Of Massachusetts Drug delivery product and methods
CN101052383B (zh) 2004-09-17 2013-01-30 马萨诸塞大学 用于溶酶体酶缺乏症的组合物和其用途
US20090209624A1 (en) * 2005-10-24 2009-08-20 University Of Massachusetts Compositions and their uses for gene therapy of bone conditions
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US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
WO2010078536A1 (fr) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition de pcsk9 par arni
JP5292118B2 (ja) * 2009-01-30 2013-09-18 富士フイルム株式会社 蛍光内視鏡システム及び蛍光内視鏡システムの作動方法
WO2012024229A1 (fr) 2010-08-14 2012-02-23 University Of Massachusetts Particule de paroi cellulaire de levures pour administrer des nanoparticules ciblées sur le récepteur
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Also Published As

Publication number Publication date
CN101052383A (zh) 2007-10-10
ES2375995T3 (es) 2012-03-08
JP2008513484A (ja) 2008-05-01
AU2005284727A1 (en) 2006-03-23
EP1802289A2 (fr) 2007-07-04
JP5302537B2 (ja) 2013-10-02
CN101052383B (zh) 2013-01-30
KR20070054246A (ko) 2007-05-28
WO2006032039A3 (fr) 2007-04-05
US8007814B2 (en) 2011-08-30
BRPI0515332A (pt) 2008-07-22
EP1802289B1 (fr) 2011-12-07
US8637045B2 (en) 2014-01-28
IL181748A (en) 2013-12-31
CA2580537A1 (fr) 2006-03-23
US20120039929A1 (en) 2012-02-16
ATE536175T1 (de) 2011-12-15
IL181748A0 (en) 2008-04-13
CA2580537C (fr) 2015-04-21
HK1105587A1 (en) 2008-02-22
WO2006032039A2 (fr) 2006-03-23
US20060083718A1 (en) 2006-04-20

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