CY1117249T1 - Ενωσεις ως διαμορφωτες μιας μεταλλαγμενης πρωτεϊνης cftr και χρησης αυτων για την θεραπεια νοσων που συνδεονται με δυσλειτουργια της πρωτεϊνης cftr - Google Patents

Ενωσεις ως διαμορφωτες μιας μεταλλαγμενης πρωτεϊνης cftr και χρησης αυτων για την θεραπεια νοσων που συνδεονται με δυσλειτουργια της πρωτεϊνης cftr

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Publication number
CY1117249T1
CY1117249T1 CY20151101051T CY151101051T CY1117249T1 CY 1117249 T1 CY1117249 T1 CY 1117249T1 CY 20151101051 T CY20151101051 T CY 20151101051T CY 151101051 T CY151101051 T CY 151101051T CY 1117249 T1 CY1117249 T1 CY 1117249T1
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CY
Cyprus
Prior art keywords
protein
cftr
constructors
compounds
disease treatment
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Application number
CY20151101051T
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English (en)
Inventor
Norbert Odolczyk
Piotr Zielenkiewicz
Grzegorz Wieczorek
Aleksander Edelman
Danielle Tondelier
Janine Fritsch
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Instytut Biochemii I Biofizyki Pan
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Priority claimed from PL392397A external-priority patent/PL221889B1/pl
Priority claimed from PL392396A external-priority patent/PL223680B1/pl
Application filed by Instytut Biochemii I Biofizyki Pan filed Critical Instytut Biochemii I Biofizyki Pan
Publication of CY1117249T1 publication Critical patent/CY1117249T1/el

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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
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    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
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    • C07F9/30—Phosphinic acids [R2P(=O)(OH)]; Thiophosphinic acids ; [R2P(=X1)(X2H) (X1, X2 are each independently O, S or Se)]
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    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D473/00—Heterocyclic compounds containing purine ring systems
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    • C07D473/22—Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6 one oxygen and one sulfur atom
    • 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
    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/496—Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
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    • 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
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    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52—Purines, e.g. adenine
    • 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
    • A61K31/662—Phosphorus acids or esters thereof having P—C bonds, e.g. foscarnet, trichlorfon
    • A61K31/663—Compounds having two or more phosphorus acid groups or esters thereof, e.g. clodronic acid, pamidronic acid
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    • C07C233/65—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals
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    • C07C235/70—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups and doubly-bound oxygen atoms bound to the same carbon skeleton
    • C07C235/84—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups and doubly-bound oxygen atoms bound to the same carbon skeleton with the carbon atom of at least one of the carboxamide groups bound to a carbon atom of a six-membered aromatic ring
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    • C07D401/12—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
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    • C07F9/30—Phosphinic acids [R2P(=O)(OH)]; Thiophosphinic acids ; [R2P(=X1)(X2H) (X1, X2 are each independently O, S or Se)]
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    • C07F9/30—Phosphinic acids [R2P(=O)(OH)]; Thiophosphinic acids ; [R2P(=X1)(X2H) (X1, X2 are each independently O, S or Se)]
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    • C07C2603/10—Ortho- or ortho- and peri-condensed systems containing three rings containing at least one ring with less than six ring members containing five-membered rings
    • C07C2603/12—Ortho- or ortho- and peri-condensed systems containing three rings containing at least one ring with less than six ring members containing five-membered rings only one five-membered ring
    • C07C2603/18—Fluorenes; Hydrogenated fluorenes

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Abstract

Η παρούσα εφεύρεση αναφέρεται σε νέους διαμορφωτές πρωτεϊνών ικανούς μετατροπής της λειτουργίας της μεταλλαγμένης πρωτεΐνης CFTR και στη χρησιμοποίηση τους για τη θεραπεία νόσων που συνδέονται με δυσλειτουργία της πρωτεΐνης CFTR. Η εφεύρεση παρέχει συνθέσεις, φαρμακευτικά παρασκευάσματα και μεθόδους διόρθωσης της κυτταρικής μετατροπής μιας μεταλλαγμένης πρωτεΐνης CFTR όπου η μετάλλαξη CFTR είναι μια μετάλλαξη ΔF508-CFTR, ή άλλη μετάλλαξη τάξης II.
CY20151101051T 2010-09-14 2015-11-20 Ενωσεις ως διαμορφωτες μιας μεταλλαγμενης πρωτεϊνης cftr και χρησης αυτων για την θεραπεια νοσων που συνδεονται με δυσλειτουργια της πρωτεϊνης cftr CY1117249T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
PL392397A PL221889B1 (pl) 2010-09-14 2010-09-14 Zastosowanie modulatora funkcji zmutowanego białka CFTR lub jego farmaceutycznie akceptowalnej pochodnej do wytwarzania leku do leczenia mukowiscydozy
PL392396A PL223680B1 (pl) 2010-09-14 2010-09-14 Zastosowanie modulatora funkcji zmutowanego białka CFTR
EP11736202.0A EP2616444B1 (en) 2010-09-14 2011-06-20 Compounds as modulators of a mutant cftr protein and their use for treating diseases associated with cftr protein malfunction

Publications (1)

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CY1117249T1 true CY1117249T1 (el) 2017-04-05

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US (5) US20130303761A1 (el)
EP (4) EP2816034B1 (el)
JP (3) JP5926731B2 (el)
KR (1) KR20140023866A (el)
CN (4) CN104478882A (el)
AU (1) AU2011302715B2 (el)
CA (1) CA2811265C (el)
CY (1) CY1117249T1 (el)
DK (1) DK2616444T3 (el)
ES (1) ES2553628T3 (el)
HR (1) HRP20151224T1 (el)
HU (1) HUE026271T2 (el)
IL (4) IL225166A (el)
MX (2) MX356714B (el)
NZ (3) NZ700827A (el)
PL (1) PL2616444T3 (el)
PT (1) PT2616444E (el)
SI (1) SI2616444T1 (el)
WO (1) WO2012036573A2 (el)

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ITMI20122065A1 (it) 2012-12-03 2014-06-04 Univ Padova Uso dei correttori del cftr nel trattamento delle patologie del muscolo striato
CN105073728A (zh) 2013-03-15 2015-11-18 全球血液疗法股份有限公司 化合物及其用于调节血红蛋白的用途
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WO2017196843A1 (en) * 2016-05-09 2017-11-16 Proteostasis Therapeutics, Inc. Methods of identifying cftr modulators
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WO2020248075A1 (en) * 2019-06-12 2020-12-17 The Governors Of The University Of Alberta Targeting dna repair in tumor cells via inhibition of ercc1-xpf
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JP7495313B2 (ja) 2020-09-23 2024-06-04 株式会社Subaru 車両用エアガイド

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US20160000778A1 (en) 2016-01-07
SI2616444T1 (sl) 2016-03-31
US10398665B2 (en) 2019-09-03
PL2616444T3 (pl) 2016-04-29
CN103228632A (zh) 2013-07-31
CA2811265C (en) 2018-07-31
WO2012036573A2 (en) 2012-03-22
CN103228632B (zh) 2016-08-31
AU2011302715A1 (en) 2013-05-02
US10463639B2 (en) 2019-11-05
IL225166A (en) 2016-08-31
NZ609204A (en) 2015-05-29
ES2553628T3 (es) 2015-12-10
EP2826771A3 (en) 2015-02-18
JP2016029044A (ja) 2016-03-03
MX356714B (es) 2018-06-11
JP2013537225A (ja) 2013-09-30
US20130303761A1 (en) 2013-11-14
EP2816034A2 (en) 2014-12-24
AU2011302715B2 (en) 2016-08-04
EP2816035A2 (en) 2014-12-24
JP5926731B2 (ja) 2016-05-25
EP2826771A2 (en) 2015-01-21
HRP20151224T1 (hr) 2016-01-01
US20160101107A1 (en) 2016-04-14
EP2616444A2 (en) 2013-07-24
MX2013002895A (es) 2013-06-05
WO2012036573A3 (en) 2012-08-16
KR20140023866A (ko) 2014-02-27
CA2811265A1 (en) 2012-03-22
NZ700818A (en) 2016-05-27
IL235014A (en) 2017-10-31
IL235013A0 (en) 2014-11-30
JP2016028044A (ja) 2016-02-25
CN104496840A (zh) 2015-04-08
US20160113896A1 (en) 2016-04-28
EP2816034A3 (en) 2015-03-04
US20160095869A1 (en) 2016-04-07
CN104478882A (zh) 2015-04-01
JP6284914B2 (ja) 2018-02-28
IL235015A0 (en) 2014-11-30
JP6173393B2 (ja) 2017-08-02
EP2826771B1 (en) 2018-12-12
CN104478800A (zh) 2015-04-01
US10420738B2 (en) 2019-09-24
DK2616444T3 (en) 2015-11-30
EP2616444B1 (en) 2015-08-26
PT2616444E (pt) 2015-12-09
EP2816035A3 (en) 2015-02-25
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