JP4740868B2 - 膵線維症の治療のためのグルコシダーゼ阻害剤の使用 - Google Patents

膵線維症の治療のためのグルコシダーゼ阻害剤の使用 Download PDF

Info

Publication number
JP4740868B2
JP4740868B2 JP2006538890A JP2006538890A JP4740868B2 JP 4740868 B2 JP4740868 B2 JP 4740868B2 JP 2006538890 A JP2006538890 A JP 2006538890A JP 2006538890 A JP2006538890 A JP 2006538890A JP 4740868 B2 JP4740868 B2 JP 4740868B2
Authority
JP
Japan
Prior art keywords
cells
dnj
cftr
delf508
protein
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Fee Related
Application number
JP2006538890A
Other languages
English (en)
Japanese (ja)
Other versions
JP2007510699A5 (https=
JP2007510699A (ja
Inventor
ベック,フレデリック
ノレツ,カロリーヌ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Centre National de la Recherche Scientifique CNRS
Universite de Poitiers
Original Assignee
Centre National de la Recherche Scientifique CNRS
Universite de Poitiers
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 Centre National de la Recherche Scientifique CNRS, Universite de Poitiers filed Critical Centre National de la Recherche Scientifique CNRS
Publication of JP2007510699A publication Critical patent/JP2007510699A/ja
Publication of JP2007510699A5 publication Critical patent/JP2007510699A5/ja
Application granted granted Critical
Publication of JP4740868B2 publication Critical patent/JP4740868B2/ja
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
    • A61K31/352Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. methantheline 
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4375Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having nitrogen as a ring heteroatom, e.g. quinolizines, naphthyridines, berberine, vincamine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0031Rectum, anus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0046Ear
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • A61P11/12Mucolytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pulmonology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Steroid Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
JP2006538890A 2003-11-07 2004-11-05 膵線維症の治療のためのグルコシダーゼ阻害剤の使用 Expired - Fee Related JP4740868B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
FR0313134A FR2861991B1 (fr) 2003-11-07 2003-11-07 Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose
FR0313134 2003-11-07
PCT/FR2004/002858 WO2005046672A2 (fr) 2003-11-07 2004-11-05 Utilisation d’inhibiteurs de glucosidase pour une therapie de la mucoviscidose

Publications (3)

Publication Number Publication Date
JP2007510699A JP2007510699A (ja) 2007-04-26
JP2007510699A5 JP2007510699A5 (https=) 2007-11-22
JP4740868B2 true JP4740868B2 (ja) 2011-08-03

Family

ID=34508347

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2006538890A Expired - Fee Related JP4740868B2 (ja) 2003-11-07 2004-11-05 膵線維症の治療のためのグルコシダーゼ阻害剤の使用

Country Status (24)

Country Link
US (2) US7973054B2 (https=)
EP (1) EP1680105B1 (https=)
JP (1) JP4740868B2 (https=)
KR (2) KR100979276B1 (https=)
CN (1) CN100534432C (https=)
AT (1) ATE423561T1 (https=)
AU (1) AU2004289083B2 (https=)
BR (1) BRPI0416228A (https=)
CA (1) CA2545133C (https=)
CY (1) CY1110468T1 (https=)
DE (1) DE602004019674D1 (https=)
DK (1) DK1680105T3 (https=)
ES (1) ES2321725T3 (https=)
FR (1) FR2861991B1 (https=)
HR (1) HRP20090159T1 (https=)
IL (1) IL175453A (https=)
NO (1) NO336904B1 (https=)
NZ (1) NZ547261A (https=)
PL (1) PL1680105T3 (https=)
PT (1) PT1680105E (https=)
RU (1) RU2358725C2 (https=)
SI (1) SI1680105T1 (https=)
WO (1) WO2005046672A2 (https=)
ZA (1) ZA200603726B (https=)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2932982B1 (en) 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
AU2007241622A1 (en) * 2006-04-24 2007-11-01 Academisch Medisch Centrum Improved treatment of cystic fibrosis
ES2573498T3 (es) 2006-05-16 2016-06-08 Amicus Therapeutics, Inc. Opciones de tratamiento para la enfermedad de Fabry
EP2352828B1 (en) * 2008-09-11 2015-02-25 Galapagos N.V. Method for identifying compounds useful for increasing the functional activity and cell surface expression of cf-associated mutant cystic fibrosis transmembrane conductance regulator
ITFE20080032A1 (it) * 2008-10-23 2010-04-24 Azienda Ospedaliera Istituti Ospit Alieri Di Vero Effetto anti infiammatorio del miglustat cellule dell'epitelio bronchiale
GB201000499D0 (en) 2010-01-13 2010-03-03 Fleet George W J Treatment of cystic fibrosis
WO2011095772A2 (en) 2010-02-04 2011-08-11 Summit Corporation Plc Novel iminosugar therapeutics
GB201006434D0 (en) 2010-04-19 2010-06-02 Summit Corp Plc Novel iminosugar combinations
KR20200032244A (ko) 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
KR20160055916A (ko) * 2013-09-16 2016-05-18 이머전트 바이롤로지 엘엘씨 데옥시노지리마이신 유도체 및 그의 이용 방법
FR3017536B1 (fr) 2014-02-18 2017-05-26 Univ La Rochelle Compositions pour la prevention et/ou le traitement de pathologies liees a l'alpha-glucosidase
WO2016054231A1 (en) 2014-09-30 2016-04-07 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
IL325155A (en) 2016-03-30 2026-02-01 Amicus Therapeutics Inc Formulations containing recombinant acid alpha-glucosidase
EP3448410A1 (en) * 2016-04-29 2019-03-06 Institut Pasteur Methods and compositions for modifying cystic fibrosis transmembrane conductance regulator activity
US10478427B2 (en) * 2016-07-07 2019-11-19 National University Corporation Chiba University Therapeutic agent for fibrosis and inhibitor of nuclear translocation of phosphorylated smad
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
AU2018278247B2 (en) * 2017-06-01 2022-07-28 Idorsia Pharmaceuticals Ltd Crystalline form of N-butyldeoxygalactonojirimycin
CN108187051A (zh) * 2018-01-30 2018-06-22 中国药科大学 α-L-岩藻糖苷酶及抑制剂用于制备防治心肌缺血损伤的药物的用途

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NO154918C (no) * 1977-08-27 1987-01-14 Bayer Ag Analogifremgangsmaate til fremstilling av terapeutisk aktive derivater av 3,4,5-trihydroksypiperidin.
TW275621B (https=) * 1992-10-27 1996-05-11 Merck & Co Inc
WO1998020123A2 (en) * 1996-11-04 1998-05-14 Institut Curie Stable cell lines expressing the cftr protein or a mutant of this protein, tool for selecting molecules having an effect on the intracellular transport of these proteins
ID21411A (id) * 1997-12-10 1999-06-10 Takeda Chemical Industries Ltd Agen untuk mengobati daya tahan glukosa yang berisiko tinggi rusak
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
AU5799500A (en) * 1999-06-29 2001-01-22 Mcgill University Human alpha 1,2-mannosidase
US6476194B1 (en) * 1999-06-29 2002-11-05 National Research Council Of Canada Method for folding unfolded proteins
AU7398900A (en) * 1999-09-23 2001-04-24 Hospital For Sick Children, The Three-dimensional structure and crystal of a class i alpha1,2-mannosidase, and methods of use thereof
US20050053625A1 (en) * 2001-10-30 2005-03-10 Block Timothy M Method of treating viral infections
US7081260B2 (en) * 2002-10-29 2006-07-25 Council Of Scientific And Industrial Research α-Glucosidase inhibitors from a natural source
PT2441467E (pt) 2003-01-31 2015-10-12 Sinai School Medicine Terapia combinada para o tratamento de transtornos associados a uma deficiência proteica
US7071341B2 (en) * 2003-02-28 2006-07-04 Council Of Scientific And Industrial Research α-Glucosidase inhibitors and their synthesis from a natural source

Also Published As

Publication number Publication date
CA2545133A1 (fr) 2005-05-26
AU2004289083B2 (en) 2009-12-03
FR2861991A1 (fr) 2005-05-13
PL1680105T3 (pl) 2009-07-31
PT1680105E (pt) 2009-05-20
NO336904B1 (no) 2015-11-23
ZA200603726B (en) 2007-12-27
DK1680105T3 (da) 2009-06-29
KR20060130058A (ko) 2006-12-18
FR2861991B1 (fr) 2008-01-18
NO20062617L (no) 2006-07-25
EP1680105A2 (fr) 2006-07-19
KR20100038452A (ko) 2010-04-14
IL175453A (en) 2012-09-24
KR100979276B1 (ko) 2010-08-31
ATE423561T1 (de) 2009-03-15
US8242136B2 (en) 2012-08-14
SI1680105T1 (sl) 2009-08-31
DE602004019674D1 (de) 2009-04-09
ES2321725T3 (es) 2009-06-10
WO2005046672A2 (fr) 2005-05-26
CA2545133C (fr) 2013-04-16
CY1110468T1 (el) 2015-04-29
IL175453A0 (en) 2008-03-20
US20070213357A1 (en) 2007-09-13
CN1897933A (zh) 2007-01-17
RU2006119914A (ru) 2007-12-20
NZ547261A (en) 2009-11-27
EP1680105B1 (fr) 2009-02-25
HRP20090159T1 (en) 2009-05-31
JP2007510699A (ja) 2007-04-26
AU2004289083A1 (en) 2005-05-26
US20110269795A1 (en) 2011-11-03
US7973054B2 (en) 2011-07-05
RU2358725C2 (ru) 2009-06-20
WO2005046672A3 (fr) 2005-09-15
BRPI0416228A (pt) 2007-01-02
CN100534432C (zh) 2009-09-02

Similar Documents

Publication Publication Date Title
JP4740868B2 (ja) 膵線維症の治療のためのグルコシダーゼ阻害剤の使用
Danahay et al. TMEM16A potentiation: a novel therapeutic approach for the treatment of cystic fibrosis
US20090048260A1 (en) Use of pyrrolopyrazine derivatives for the production of medicaments for the treatment of mucoviscidosis and diseases related to protein addressing errors in cells
Al-Nakkash et al. A common mechanism for cystic fibrosis transmembrane conductance regulator protein activation by genistein and benzimidazolone analogs
Van Goor et al. Rescue of ΔF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules
US8492420B2 (en) Use of purine derivatives for the production of medicaments for the treatment of mucoviscidosis and diseases related to protein addressing errors in cells
Chang et al. Role of N-linked oligosaccharides in the biosynthetic processing of the cystic fibrosis membrane conductance regulator
Cui et al. Murine and human CFTR exhibit different sensitivities to CFTR potentiators
Norez et al. Proteasome-dependent pharmacological rescue of cystic fibrosis transmembrane conductance regulator revealed by mutation of glycine 622
JP2009529488A (ja) 嚢胞性繊維症及び細胞中のタンパク質アドレッシング欠損症を治療する薬物を製造するためのグアナベンズ及びその誘導体の使用
MD3897655T2 (ro) 3beta-(Benziloxi)-17α-metil-pregn-5-en-20-onă pentru utilizare în tratamentul tulburărilor cognitive
Routaboul et al. Discovery of α-aminoazaheterocycle-methylglyoxal adducts as a new class of high-affinity inhibitors of cystic fibrosis transmembrane conductance regulator chloride channels
US7605153B2 (en) Use of paullone derivatives for the production of medicaments for the treatment of mucoviscidosis and diseases related to protein addressing errors in cells
MXPA06005086A (en) Use of glucosidase inhibitors for therapy of mucovisidosis
Bridges Pharmacology of AF508-CFTR Biosynthesis
Becq et al. Specific pharmacological therapy in cystic fibrosis
Bhattarai Design, Synthesis, and Evaluation of Novel Antimycobacterial Agents With Enhanced Pharmacokinetic Profiles
TW201618787A (zh) 組合物及方法
Sutton Rescuing Dopamine Transporter Deficiency Syndrome
BULLOCK CYSTIC FIBROSIS: A DRIVE FOR ACQUIRING KNOWLEDGE
Pedemonte Block of CFTR-dependent chloride currents by inhibitors of multidrug resistance-associated proteins

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20071004

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20071004

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20100914

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20101213

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20101220

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20110113

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20110412

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20110502

R150 Certificate of patent or registration of utility model

Free format text: JAPANESE INTERMEDIATE CODE: R150

Ref document number: 4740868

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

FPAY Renewal fee payment (event date is renewal date of database)

Free format text: PAYMENT UNTIL: 20140513

Year of fee payment: 3

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees