BRPI0515332A - composições e seus usos para deficiências das enzimas lisossÈmicas - Google Patents

composições e seus usos para deficiências das enzimas lisossÈmicas

Info

Publication number
BRPI0515332A
BRPI0515332A BRPI0515332-8A BRPI0515332A BRPI0515332A BR PI0515332 A BRPI0515332 A BR PI0515332A BR PI0515332 A BRPI0515332 A BR PI0515332A BR PI0515332 A BRPI0515332 A BR PI0515332A
Authority
BR
Brazil
Prior art keywords
payload
molecule
lysosomal enzyme
compositions
enzyme deficiencies
Prior art date
Application number
BRPI0515332-8A
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 BRPI0515332A publication Critical patent/BRPI0515332A/pt

Links

Classifications

    • 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
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/5005—Wall or coating material
    • A61K9/5063—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5068—Cell membranes or bacterial membranes enclosing drugs
    • 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
    • A61K31/715—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/716—Glucans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/69—Medicinal 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
    • A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • 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
    • A61K48/0008—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 '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
    • 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
    • A61K48/0008—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 '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
    • A61K48/0025—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 '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
    • A61K48/0041—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 '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
    • 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
    • A61P43/00—Drugs 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Botany (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)

Abstract

COMPOSIçõES E SEUS USOS PARA DEFICIêNCIAS DAS ENZIMAS LISOSSÈMICAS. A presente invenção refere-se a uma composição que compreende uma parede celular de levedura extraída compreendendo beta-glucano, uma molécula de carga útil de captura e uma molécula de carga útil, onde a molécula de carga útil e a molécula de carga útil de captura são solúveis no mesmo sistema de solvente, onde a molécula de carga útil suplementa a função da enzima lisossómica deficiente. A invenção adicionalmente proporciona métodos de preparar e métodos de utilizar a composição.
BRPI0515332-8A 2004-09-17 2005-09-19 composições e seus usos para deficiências das enzimas lisossÈmicas BRPI0515332A (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US61087204P 2004-09-17 2004-09-17
PCT/US2005/033300 WO2006032039A2 (en) 2004-09-17 2005-09-19 Compositions and their uses for lysosomal enzyme deficiencies

Publications (1)

Publication Number Publication Date
BRPI0515332A true BRPI0515332A (pt) 2008-07-22

Family

ID=36060733

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0515332-8A BRPI0515332A (pt) 2004-09-17 2005-09-19 composições e seus usos para deficiências das enzimas lisossÈmicas

Country Status (13)

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

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Also Published As

Publication number Publication date
US8007814B2 (en) 2011-08-30
CA2580537A1 (en) 2006-03-23
JP5302537B2 (ja) 2013-10-02
JP2008513484A (ja) 2008-05-01
ZA200701943B (en) 2008-07-30
AU2005284727A1 (en) 2006-03-23
IL181748A0 (en) 2008-04-13
US20060083718A1 (en) 2006-04-20
WO2006032039A2 (en) 2006-03-23
CN101052383A (zh) 2007-10-10
EP1802289B1 (en) 2011-12-07
EP1802289A2 (en) 2007-07-04
US8637045B2 (en) 2014-01-28
US20120039929A1 (en) 2012-02-16
ES2375995T3 (es) 2012-03-08
HK1105587A1 (en) 2008-02-22
CN101052383B (zh) 2013-01-30
ATE536175T1 (de) 2011-12-15
CA2580537C (en) 2015-04-21
WO2006032039A3 (en) 2007-04-05
KR20070054246A (ko) 2007-05-28
IL181748A (en) 2013-12-31

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