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 σαπερονηςInfo
- 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
Links
Classifications
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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
- G01N2800/38—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
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.
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 | |
EP09710178.6A EP2252313B1 (en) | 2008-02-12 | 2009-02-12 | Method to predict response to pharmacological chaperone treatment of diseases |
PCT/US2009/033963 WO2009102895A2 (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 |
Family
ID=40957500
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
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 |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20151100595T CY1116466T1 (el) | 2008-02-12 | 2015-07-08 | Μεθοδος για την προβλεψη αποκρισης σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης των ασθενειων |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20201101114T CY1123816T1 (el) | 2008-02-12 | 2020-11-25 | Μεθοδος για την προβλεψη αποκρισης των ασθενειων σε φαρμακολογικη θεραπευτικη αγωγη σαπερονης |
Country Status (16)
Country | Link |
---|---|
US (7) | US8592362B2 (el) |
EP (4) | EP3824900A1 (el) |
JP (6) | JP5844045B2 (el) |
AU (6) | AU2009214648B2 (el) |
CA (1) | CA2715407C (el) |
CY (3) | CY1116466T1 (el) |
DK (3) | DK3470077T3 (el) |
ES (3) | ES2716502T3 (el) |
HR (3) | HRP20150728T1 (el) |
HU (3) | HUE051377T2 (el) |
LT (2) | LT2946785T (el) |
MX (1) | MX2010008835A (el) |
PL (3) | PL2946785T3 (el) |
PT (3) | PT3470077T (el) |
SI (3) | SI2252313T1 (el) |
WO (1) | WO2009102895A2 (el) |
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DK2533050T6 (da) | 2006-05-16 | 2015-09-21 | Amicus Therapeutics Inc | Behandlingsmuligheder ved Fabrys sygdom |
HUE051377T2 (hu) | 2008-02-12 | 2021-03-01 | Amicus Therapeutics Inc | Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére |
MX2010009875A (es) * | 2008-03-12 | 2010-11-26 | Amicus Therapeutics Inc | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de pompe. |
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BR112017005810A2 (pt) | 2014-09-30 | 2018-02-27 | Amicus Therapeutics Inc | alfa-glucosidase ácida altamente potente com carboidratos melhorados |
WO2016149508A1 (en) * | 2015-03-19 | 2016-09-22 | Shire Human Genetic Therapies, Inc. | Mrna therapy for pompe disease |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 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 |
EP3432882B1 (en) | 2016-03-22 | 2021-09-01 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
SG11201808455VA (en) | 2016-03-30 | 2018-10-30 | Amicus Therapeutics Inc | Formulations comprising recombinant acid alpha-glucosidase |
BR112018070189A2 (pt) | 2016-03-30 | 2019-02-19 | Amicus Therapeutics, Inc. | método para seleção de proteínas recombinantes ricas em m6p |
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JP6546135B2 (ja) | 2016-08-24 | 2019-07-17 | 株式会社ノリタケカンパニーリミテド | 積層造形焼成体、積層造形焼成体の製造方法および積層造形焼成体製造用キット |
CA3039673A1 (en) * | 2016-10-20 | 2018-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
EA202290114A1 (ru) * | 2017-03-30 | 2022-03-29 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
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EP4324522A2 (en) | 2017-05-30 | 2024-02-21 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
KR102631805B1 (ko) | 2017-05-30 | 2024-01-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
KR20200044908A (ko) | 2017-08-28 | 2020-04-29 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 파브리병을 앓는 환자에서 심장 기능을 강화 및/또는 안정화시키는 방법 |
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US11833164B2 (en) | 2019-08-07 | 2023-12-05 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
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2009
- 2009-02-12 HU HUE18201960A patent/HUE051377T2/hu unknown
- 2009-02-12 LT LTEP15162630.6T patent/LT2946785T/lt unknown
- 2009-02-12 EP EP20192637.5A patent/EP3824900A1/en active Pending
- 2009-02-12 LT LTEP18201960.4T patent/LT3470077T/lt unknown
- 2009-02-12 PL PL15162630T patent/PL2946785T3/pl unknown
- 2009-02-12 PT PT182019604T patent/PT3470077T/pt unknown
- 2009-02-12 SI SI200931229T patent/SI2252313T1/sl unknown
- 2009-02-12 PT PT15162630T patent/PT2946785T/pt unknown
- 2009-02-12 MX MX2010008835A patent/MX2010008835A/es active IP Right Grant
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