WO2000018954A3 - Use of pex in the treatment of metabolic bone diseases - Google Patents

Use of pex in the treatment of metabolic bone diseases Download PDF

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Publication number
WO2000018954A3
WO2000018954A3 PCT/CA1999/000895 CA9900895W WO0018954A3 WO 2000018954 A3 WO2000018954 A3 WO 2000018954A3 CA 9900895 W CA9900895 W CA 9900895W WO 0018954 A3 WO0018954 A3 WO 0018954A3
Authority
WO
WIPO (PCT)
Prior art keywords
bone diseases
metabolic bone
pex
treatment
patient
Prior art date
Application number
PCT/CA1999/000895
Other languages
French (fr)
Other versions
WO2000018954A2 (en
Inventor
Andrew C Karaplis
David Goltzman
Mark L Lipman
Janet E Henderson
Original Assignee
Univ Mcgill
Andrew C Karaplis
David Goltzman
Mark L Lipman
Janet E Henderson
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 Mcgill, Andrew C Karaplis, David Goltzman, Mark L Lipman, Janet E Henderson filed Critical Univ Mcgill
Priority to EP99945809A priority Critical patent/EP1117427A2/en
Priority to CA002343713A priority patent/CA2343713C/en
Priority to AU58450/99A priority patent/AU5845099A/en
Priority to JP2000572401A priority patent/JP2002525124A/en
Publication of WO2000018954A2 publication Critical patent/WO2000018954A2/en
Publication of WO2000018954A3 publication Critical patent/WO2000018954A3/en
Priority to US10/952,878 priority patent/US7393837B2/en

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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
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/473—Quinolines; Isoquinolines ortho- or peri-condensed with carbocyclic ring systems, e.g. acridines, phenanthridines
    • 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
    • A61K31/66—Phosphorus compounds
    • 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)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4886—Metalloendopeptidases (3.4.24), e.g. collagenase
    • 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
    • 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
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • 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
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/635—Parathyroid hormone, i.e. parathormone; Parathyroid hormone-related peptides
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6489—Metalloendopeptidases (3.4.24)
    • C12N9/6491—Matrix metalloproteases [MMP's], e.g. interstitial collagenase (3.4.24.7); Stromelysins (3.4.24.17; 3.2.1.22); Matrilysin (3.4.24.23)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • C12Q1/37—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase involving peptidase or proteinase
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Urology & Nephrology (AREA)
  • Analytical Chemistry (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Food Science & Technology (AREA)

Abstract

The present invention relates to a method for the diagnosis of metabolic bone diseases in a patient, which comprises the step of determining the level of PTHrP in a biological sample of a patient wherein an alteration of PTHrP levels from that of a normal individual is indicative of metabolic bone diseases and/or metabolic bone diseases predisposition. The present invention also relates to a method for the diagnosis of metabolic bone diseases in a patient, which comprises the step of determining the level of PTHrP in a biological sample of a patient wherein an alteration of PTHrP levels from that of a normal individual is indicative of metabolic bone diseases and/or metabolic bone diseases predisposition.
PCT/CA1999/000895 1998-09-28 1999-09-27 Use of pex in the treatment of metabolic bone diseases WO2000018954A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
EP99945809A EP1117427A2 (en) 1998-09-28 1999-09-27 USE OF $i(PEX) IN THE TREATMENT OF METABOLIC BONE DISEASES
CA002343713A CA2343713C (en) 1998-09-28 1999-09-27 Use of pex in the treatment of metabolic bone diseases
AU58450/99A AU5845099A (en) 1998-09-28 1999-09-27 Use of (pex) in the treatment of metabolic bone diseases
JP2000572401A JP2002525124A (en) 1998-09-28 1999-09-27 Use of PEX in the treatment of metabolic bone disease
US10/952,878 US7393837B2 (en) 1998-09-28 2004-09-30 Inhibition of PEX in the treatment of metabolic bone diseases

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CA002245903A CA2245903A1 (en) 1998-09-28 1998-09-28 Use of pex in the treatment of metabolic bone diseases
CA2,245,903 1998-09-28

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US09806110 A-371-Of-International 1999-09-27
US10/952,878 Continuation-In-Part US7393837B2 (en) 1998-09-28 2004-09-30 Inhibition of PEX in the treatment of metabolic bone diseases

Publications (2)

Publication Number Publication Date
WO2000018954A2 WO2000018954A2 (en) 2000-04-06
WO2000018954A3 true WO2000018954A3 (en) 2000-07-06

Family

ID=4162769

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/CA1999/000895 WO2000018954A2 (en) 1998-09-28 1999-09-27 Use of pex in the treatment of metabolic bone diseases

Country Status (6)

Country Link
US (1) US7393837B2 (en)
EP (1) EP1117427A2 (en)
JP (1) JP2002525124A (en)
AU (1) AU5845099A (en)
CA (1) CA2245903A1 (en)
WO (1) WO2000018954A2 (en)

Families Citing this family (25)

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Publication number Priority date Publication date Assignee Title
EP1293568A4 (en) * 2000-06-21 2005-10-26 Takeda Pharmaceutical Novel protein and dna thereof
AU2003207512B2 (en) 2002-01-10 2008-06-12 Osteotrophin Llc Treatment of bone disorders with skeletal anabolic drugs
US20040005619A1 (en) * 2002-05-31 2004-01-08 Mcgill University PTHrP-based prediction and diagnosis of bone disease
ES2365023T3 (en) 2004-04-21 2011-09-20 Enobia Pharma Inc. CONJUGATES OF BONE ADMINISTRATION AND METHOD OF USE OF THE SAME TO DIRECT BONE PROTEINS.
ES2568259T3 (en) 2004-05-13 2016-04-28 Alza Corporation Apparatus and method for transdermal administration of parathyroid hormone agents
US20070081984A1 (en) 2005-10-11 2007-04-12 Shunji Tomatsu Compositions and methods for treating hypophosphatasia
US20080171319A1 (en) * 2006-02-06 2008-07-17 Mickey Urdea Osteoporosis associated markers and methods of use thereof
JP2010540534A (en) 2007-09-28 2010-12-24 イントレキソン コーポレーション Therapeutic gene switch constructs and bioreactors for the expression of biotherapeutic molecules and uses thereof
BR112012027765A2 (en) 2010-04-30 2019-09-24 Enobia Pharma Inc methods, compositions and kits for treating matrix mineralization disorders.
EP2658979B1 (en) 2010-12-27 2018-02-14 Alexion Pharmaceuticals, Inc. Compositions comprising natriuretic peptides and methods of use thereof
US10052366B2 (en) 2012-05-21 2018-08-21 Alexion Pharmaceuticsl, Inc. Compositions comprising alkaline phosphatase and/or natriuretic peptide and methods of use thereof
WO2016007873A1 (en) 2014-07-11 2016-01-14 The Regents Of The University Of Michigan Compositions and methods for treating craniosynostosis
US10449236B2 (en) 2014-12-05 2019-10-22 Alexion Pharmaceuticals, Inc. Treating seizure with recombinant alkaline phosphatase
CA2973883A1 (en) 2015-01-28 2016-08-04 Alexion Pharmaceuticals, Inc. Methods of treating a subject with an alkaline phosphatase deficiency
US11352612B2 (en) 2015-08-17 2022-06-07 Alexion Pharmaceuticals, Inc. Manufacturing of alkaline phosphatases
WO2017058822A1 (en) 2015-09-28 2017-04-06 Alexion Pharmaceuticals, Inc. Identifying effective dosage regimens for tissue non-specific alkaline phosphatase (tnsalp)-enzyme replacement therapy of hypophosphatasia
EP3368062A4 (en) 2015-10-30 2019-07-03 Alexion Pharmaceuticals, Inc. Methods for treating craniosynostosis in a patient
WO2017155569A1 (en) 2016-03-08 2017-09-14 Alexion Pharmaceuticals, Inc. Methods for treating hypophosphatasia in children
WO2017173395A1 (en) 2016-04-01 2017-10-05 Alexion Pharmaceuticals, Inc. Methods for treating hypophosphatasia in adolescents and adults
CA3019726A1 (en) 2016-04-01 2017-10-05 Alexion Pharmaceuticals, Inc. Treating muscle weakness with alkaline phosphatases
US10988744B2 (en) 2016-06-06 2021-04-27 Alexion Pharmaceuticals, Inc. Method of producing alkaline phosphatase
EP3500289B1 (en) 2016-08-18 2024-10-09 Alexion Pharmaceuticals, Inc. Asfotase alfa for use in treating tracheobronchomalacia
CN110719786A (en) 2017-03-31 2020-01-21 阿雷克森制药公司 Methods for treating Hypophosphatasia (HPP) in adults and adolescents
EP3773684A1 (en) 2018-03-30 2021-02-17 Alexion Pharmaceuticals, Inc. Manufacturing of glycoproteins
CA3173631A1 (en) 2021-02-12 2022-08-18 Walter C. Voegtli Alkaline phosphatase polypeptides and methods of use thereof

Citations (1)

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Publication number Priority date Publication date Assignee Title
WO1998010078A2 (en) * 1996-09-05 1998-03-12 Karaplis Andrew C CLONING OF FULL-LENGTH HUMAN PEX cDNA

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MXPA00011272A (en) * 1998-05-18 2003-04-22 Univ London A novel polypeptide hormone phosphatonin.
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Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO1998010078A2 (en) * 1996-09-05 1998-03-12 Karaplis Andrew C CLONING OF FULL-LENGTH HUMAN PEX cDNA

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
GUISE THERESA A ET AL: "Evidence for a causal role of parathyroid hormone-related protein in the pathogenesis of human breast cancer-mediated osteolysis.", JOURNAL OF CLINICAL INVESTIGATION 1996, vol. 98, no. 7, 1996, pages 1544 - 1549, XP002134629, ISSN: 0021-9738 *
LIPMAN MARK L ET AL: "Cloning of human PEX cDNA: Expression, subcellular localization, and endopeptidase activity.", JOURNAL OF BIOLOGICAL CHEMISTRY MAY 29, 1998, vol. 273, no. 22, 29 May 1998 (1998-05-29), pages 13729 - 13737, XP002134628, ISSN: 0021-9258 *
SCHNEIDER HANS-GERHARD ET AL: "Parathyroid hormone-related protein mRNA and protein expressions in multiple myeloma: A case report.", JOURNAL OF BONE AND MINERAL RESEARCH OCT., 1998, vol. 13, no. 10, October 1998 (1998-10-01), pages 1640 - 1643, XP000891605, ISSN: 0884-0431 *

Also Published As

Publication number Publication date
WO2000018954A2 (en) 2000-04-06
CA2245903A1 (en) 2000-03-28
JP2002525124A (en) 2002-08-13
AU5845099A (en) 2000-04-17
US20050113303A1 (en) 2005-05-26
US7393837B2 (en) 2008-07-01
EP1117427A2 (en) 2001-07-25

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