AU2009214648B2 - Method to predict response to pharmacological chaperone treatment of diseases - Google Patents

Method to predict response to pharmacological chaperone treatment of diseases Download PDF

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Publication number
AU2009214648B2
AU2009214648B2 AU2009214648A AU2009214648A AU2009214648B2 AU 2009214648 B2 AU2009214648 B2 AU 2009214648B2 AU 2009214648 A AU2009214648 A AU 2009214648A AU 2009214648 A AU2009214648 A AU 2009214648A AU 2009214648 B2 AU2009214648 B2 AU 2009214648B2
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gal
protein
gaa
activity
cells
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AU2009214648A1 (en
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Elfrida Benjamin
Hung V. Do
John Flanagan
Xiaoyang Wu
Brandon Wustman
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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Priority to AU2014221321A priority Critical patent/AU2014221321B2/en
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    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/46—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
    • G01N2333/47—Assays involving proteins of known structure or function as defined in the subgroups
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
    • G01N2333/94—Hydrolases (3) acting on glycosyl compounds (3.2) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/04—Endocrine or metabolic disorders
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/38—Pediatrics
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

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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)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Zoology (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Analytical Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Obesity (AREA)
  • Epidemiology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Food Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
AU2009214648A 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases Active AU2009214648B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2014221321A AU2014221321B2 (en) 2008-02-12 2014-09-10 Method to predict response to pharmacological chaperone treatment of diseases
AU2018220047A AU2018220047A1 (en) 2009-02-12 2018-08-22 A method for treatment of fabry disease

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US2814108P 2008-02-12 2008-02-12
US61/028,141 2008-02-12
US3568408P 2008-03-11 2008-03-11
US61/035,684 2008-03-11
US9363108P 2008-09-02 2008-09-02
US61/093,631 2008-09-02
US11349608P 2008-11-11 2008-11-11
US61/113,496 2008-11-11
PCT/US2009/033963 WO2009102895A2 (en) 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases

Related Child Applications (2)

Application Number Title Priority Date Filing Date
AU2014221321A Division AU2014221321B2 (en) 2008-02-12 2014-09-10 Method to predict response to pharmacological chaperone treatment of diseases
AU2018220047A Addition AU2018220047A1 (en) 2009-02-12 2018-08-22 A method for treatment of fabry disease

Publications (2)

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AU2009214648A1 AU2009214648A1 (en) 2009-08-20
AU2009214648B2 true AU2009214648B2 (en) 2014-11-13

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Application Number Title Priority Date Filing Date
AU2009214648A Active AU2009214648B2 (en) 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases
AU2014221321A Active AU2014221321B2 (en) 2008-02-12 2014-09-10 Method to predict response to pharmacological chaperone treatment of diseases
AU2016206297A Abandoned AU2016206297A1 (en) 2008-02-12 2016-07-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2017268649A Abandoned AU2017268649A1 (en) 2008-02-12 2017-11-30 Method to predict response to pharmacological chaperone treatment of diseases
AU2019219727A Active AU2019219727B2 (en) 2008-02-12 2019-08-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2021218172A Abandoned AU2021218172A1 (en) 2008-02-12 2021-08-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2024219997A Pending AU2024219997A1 (en) 2008-02-12 2024-09-24 Method to predict response to pharmacological chaperone treatment of diseases

Family Applications After (6)

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AU2014221321A Active AU2014221321B2 (en) 2008-02-12 2014-09-10 Method to predict response to pharmacological chaperone treatment of diseases
AU2016206297A Abandoned AU2016206297A1 (en) 2008-02-12 2016-07-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2017268649A Abandoned AU2017268649A1 (en) 2008-02-12 2017-11-30 Method to predict response to pharmacological chaperone treatment of diseases
AU2019219727A Active AU2019219727B2 (en) 2008-02-12 2019-08-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2021218172A Abandoned AU2021218172A1 (en) 2008-02-12 2021-08-20 Method to predict response to pharmacological chaperone treatment of diseases
AU2024219997A Pending AU2024219997A1 (en) 2008-02-12 2024-09-24 Method to predict response to pharmacological chaperone treatment of diseases

Country Status (16)

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US (8) US8592362B2 (enExample)
EP (4) EP3824900A1 (enExample)
JP (7) JP5844045B2 (enExample)
AU (7) AU2009214648B2 (enExample)
CA (1) CA2715407C (enExample)
CY (3) CY1116466T1 (enExample)
DK (3) DK3470077T3 (enExample)
ES (3) ES2836121T3 (enExample)
HR (3) HRP20150728T1 (enExample)
HU (3) HUE042882T2 (enExample)
LT (2) LT3470077T (enExample)
MX (1) MX2010008835A (enExample)
PL (3) PL2946785T3 (enExample)
PT (3) PT2946785T (enExample)
SI (3) SI2946785T1 (enExample)
WO (1) WO2009102895A2 (enExample)

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US20110136151A1 (en) * 2008-03-12 2011-06-09 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for pompe disease
TW201117810A (en) * 2009-10-01 2011-06-01 Baylor Res Inst Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease
US20120283290A1 (en) * 2011-05-06 2012-11-08 Amicus Therapeutics Inc. Quantitation of gl3 in urine
ES3017134T3 (en) 2014-09-30 2025-05-12 Amicus Therapeutics Inc Highly potent acid alpha-glucosidase with enhanced carbohydrates
JP6895892B2 (ja) * 2015-03-19 2021-06-30 トランスレイト バイオ， インコーポレイテッド ポンペ病のｍＲＮＡ治療
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
KR102747683B1 (ko) 2015-12-30 2024-12-31 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
LT3397273T (lt) 2015-12-30 2021-06-25 Amicus Therapeutics, Inc. Patobulinta rūgštinė alfa-gliukozidazė pompe ligos gydymui
CN109069494A (zh) 2016-03-22 2018-12-21 阿米库斯治疗学公司 治疗在gla基因中具有g9331a突变的患者的法布里病的方法
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US11219695B2 (en) * 2016-10-20 2022-01-11 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
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