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AU2001269923A1 - Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases - Google Patents

Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

Info

Publication number
AU2001269923A1
AU2001269923A1 AU2001269923A AU6992301A AU2001269923A1 AU 2001269923 A1 AU2001269923 A1 AU 2001269923A1 AU 2001269923 A AU2001269923 A AU 2001269923A AU 6992301 A AU6992301 A AU 6992301A AU 2001269923 A1 AU2001269923 A1 AU 2001269923A1
Authority
AU
Australia
Prior art keywords
therapy
small molecule
lysosomal storage
storage diseases
enzyme replacement
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2001269923A
Inventor
Seng H Cheng
David Meeker
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
Original Assignee
Genzyme 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 Genzyme Corp filed Critical Genzyme Corp
Publication of AU2001269923A1 publication Critical patent/AU2001269923A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2001269923A 2000-06-19 2001-06-19 Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases Abandoned AU2001269923A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US21237700P 2000-06-19 2000-06-19
US60/212,377 2000-06-19
PCT/US2001/019579 WO2001097829A2 (en) 2000-06-19 2001-06-19 Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

Publications (1)

Publication Number Publication Date
AU2001269923A1 true AU2001269923A1 (en) 2002-01-02

Family

ID=22790749

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001269923A Abandoned AU2001269923A1 (en) 2000-06-19 2001-06-19 Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

Country Status (3)

Country Link
US (4) US20020095135A1 (en)
AU (1) AU2001269923A1 (en)
WO (1) WO2001097829A2 (en)

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Also Published As

Publication number Publication date
WO2001097829A2 (en) 2001-12-27
US20110142818A1 (en) 2011-06-16
US20070280925A1 (en) 2007-12-06
US20020095135A1 (en) 2002-07-18
US7910545B2 (en) 2011-03-22
US20120183502A1 (en) 2012-07-19
US8168587B2 (en) 2012-05-01
WO2001097829A3 (en) 2002-12-27

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