CY1121386T1 - Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης - Google Patents

Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης

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Publication number
CY1121386T1
CY1121386T1 CY20191100100T CY191100100T CY1121386T1 CY 1121386 T1 CY1121386 T1 CY 1121386T1 CY 20191100100 T CY20191100100 T CY 20191100100T CY 191100100 T CY191100100 T CY 191100100T CY 1121386 T1 CY1121386 T1 CY 1121386T1
Authority
CY
Cyprus
Prior art keywords
saperone
gal
preventing disease
pharmacological treatment
disease response
Prior art date
Application number
CY20191100100T
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English (en)
Inventor
Elfrida Benjamin
Hung V. Do
Xiaoyang Wu
John Flanagan
Brandon Wustman
Original Assignee
Amicus Therapeutics, Inc.
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Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=40957500&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1121386(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Amicus Therapeutics, Inc. filed Critical Amicus Therapeutics, Inc.
Publication of CY1121386T1 publication Critical patent/CY1121386T1/el

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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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Urology & Nephrology (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Obesity (AREA)
  • Epidemiology (AREA)
  • Food Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Emergency Medicine (AREA)

Abstract

Μια μέθοδος σύνταξης ενός πίνακα αναφοράς θεραπευτικής αντιμετώπισης, ο οποίος υποδεικνύει την αποκρισιμότητα μιας ή περισσοτέρων ειδικών μεταλλάξεων ενός α-Gal Α σε μια ειδική φαρμακολογική σαπερόνη, με την μέθοδο να περιλαμβάνει: την ανάλυση in vitro της απόκρισης στην ειδική φαρμακολογική σαπερόνη εντός ενός κυττάρου ξενιστή, το οποίο έχει μετασχηματισθεί με έναν φορέα νουκλεϊνικού οξέος, ο οποίος κωδικοποιεί έναν πρώτο μεταλλάκτη α-Gal Α και τη σύνταξη ενός καταλόγου, υπό μορφή πίνακα, αποκρίσιμων και μη-αποκρίσιμων μεταλλαγμένων μορφών ενός α-Gal Α. Η ειδική φαρμακολογική σαπερόνη είναι 1-δεοξυγαλακτονοζιριμυκίνη ή ένα φαρμακευτικό άλας ή εστέρας και το πρώτο κύτταρο είναι ένα ΗΕΚ-293 MSR κύτταρο.
CY20191100100T 2008-02-12 2019-01-24 Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης CY1121386T1 (el)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US2814108P 2008-02-12 2008-02-12
US3568408P 2008-03-11 2008-03-11
US9363108P 2008-09-02 2008-09-02
US11349608P 2008-11-11 2008-11-11
PCT/US2009/033963 WO2009102895A2 (en) 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases
EP09710178.6A EP2252313B1 (en) 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases

Publications (1)

Publication Number Publication Date
CY1121386T1 true CY1121386T1 (el) 2020-05-29

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Family Applications (3)

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CY20151100595T CY1116466T1 (el) 2008-02-12 2015-07-08 Μεθοδος για την προβλεψη αποκρισης σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης των ασθενειων
CY20191100100T CY1121386T1 (el) 2008-02-12 2019-01-24 Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης
CY20201101114T CY1123816T1 (el) 2008-02-12 2020-11-25 Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης

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CY20151100595T CY1116466T1 (el) 2008-02-12 2015-07-08 Μεθοδος για την προβλεψη αποκρισης σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης των ασθενειων

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Country Status (16)

Country Link
US (8) US8592362B2 (el)
EP (4) EP2946785B1 (el)
JP (6) JP5844045B2 (el)
AU (6) AU2009214648B2 (el)
CA (1) CA2715407C (el)
CY (3) CY1116466T1 (el)
DK (3) DK3470077T3 (el)
ES (3) ES2836121T3 (el)
HR (3) HRP20150728T1 (el)
HU (3) HUE051377T2 (el)
LT (2) LT3470077T (el)
MX (1) MX2010008835A (el)
PL (3) PL3470077T3 (el)
PT (3) PT2252313E (el)
SI (3) SI2252313T1 (el)
WO (1) WO2009102895A2 (el)

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