AU2003284886A8 - Chemical chaperones and their effect upon the cellular activity of beta-glucosidase - Google Patents

Chemical chaperones and their effect upon the cellular activity of beta-glucosidase

Info

Publication number
AU2003284886A8
AU2003284886A8 AU2003284886A AU2003284886A AU2003284886A8 AU 2003284886 A8 AU2003284886 A8 AU 2003284886A8 AU 2003284886 A AU2003284886 A AU 2003284886A AU 2003284886 A AU2003284886 A AU 2003284886A AU 2003284886 A8 AU2003284886 A8 AU 2003284886A8
Authority
AU
Australia
Prior art keywords
glucosidase
beta
effect
cellular activity
chemical chaperones
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.)
Abandoned
Application number
AU2003284886A
Other versions
AU2003284886A1 (en
Inventor
William E Balch
Anu R Sawkar
Ernest Beutler
Jeffrey W Kelly
Chi-Heuy Wong
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.)
WONG CHI HEUY
Scripps Research Institute
Original Assignee
WONG CHI HEUY
Scripps Research Institute
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 WONG CHI HEUY, Scripps Research Institute filed Critical WONG CHI HEUY
Publication of AU2003284886A8 publication Critical patent/AU2003284886A8/en
Publication of AU2003284886A1 publication Critical patent/AU2003284886A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/02—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements
    • C07D295/027—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements containing only one hetero ring
    • C07D295/03—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements containing only one hetero ring with the ring nitrogen atoms directly attached to acyclic carbon atoms
    • 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
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/02—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D207/04—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D207/10—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D207/16—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/08—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms
    • C07D295/084—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/088—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/04—Ortho-condensed systems
AU2003284886A 2002-10-21 2003-10-21 CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF Beta-GLUCOSIDASE Abandoned AU2003284886A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US42047302P 2002-10-21 2002-10-21
US60/420,473 2002-10-21
PCT/US2003/033670 WO2004037373A2 (en) 2002-10-21 2003-10-21 CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF β-GLUCOSIDASE

Publications (2)

Publication Number Publication Date
AU2003284886A8 true AU2003284886A8 (en) 2004-05-13
AU2003284886A1 AU2003284886A1 (en) 2004-05-13

Family

ID=32176573

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2003284886A Abandoned AU2003284886A1 (en) 2002-10-21 2003-10-21 CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF Beta-GLUCOSIDASE

Country Status (2)

Country Link
AU (1) AU2003284886A1 (en)
WO (1) WO2004037373A2 (en)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040180419A1 (en) 2003-01-31 2004-09-16 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
BRPI0416216A (en) 2003-11-12 2006-12-26 Amicus Therapeutics Inc glycoimidazole and polyhydroxycyclohexenyl compound and method for synthesizing glycoimidazole and polyhydroxycyclohexenyl compound, methods for inhibiting glucocerebrosidase, for enhancing glucocerebrosidase activity, pharmaceutical composition, methods for inhibiting a -glycosity, and for treating gaucher disease, glycoimidazole and polyhydroxycyclohexylamine derivatives
EP2932982B1 (en) 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
WO2009066069A1 (en) * 2007-11-21 2009-05-28 Summit Corporation Plc Treatment of protein folding disorders
WO2012001641A1 (en) * 2010-06-30 2012-01-05 Actelion Pharmaceuticals Ltd Stable pharmaceutical compositions
PT2649075T (en) 2010-12-08 2018-07-30 Us Health Substituted pyrazolopyrimidines as glucocerebrosidase activators
US9956236B2 (en) 2011-02-07 2018-05-01 Cornell University Methods for increasing immune responses using agents that directly bind to and activate IRE-1
WO2013134774A1 (en) 2012-03-09 2013-09-12 Cornell University Modulation of breast cancer growth by modulation of xbp1 activity
ES2472115B1 (en) * 2012-12-26 2015-04-30 Consejo Superior De Investigaciones Científicas USE OF BICYCLIC DERIVATIVES OF 1-DEOXIGALACTONOJIRIMYCIN IN THE PREPARATION OF A MEDICINAL PRODUCT FOR THE TREATMENT OF DISEASES RELATED TO HUMAN MUTANT LYSOSOMIC GALACTOSIDASE ILLNESSES
EP3049521B1 (en) 2013-09-25 2019-03-06 Cornell University Compounds for inducing anti-tumor immunity and methods thereof
EP3088389A1 (en) * 2015-04-28 2016-11-02 Dorphan S.A. 4-epi-isofagomine derivatives
WO2019211205A1 (en) 2018-04-30 2019-11-07 Dorphan S.A. Pharmaceutical compositions for use in the treatment of lysosomal diseases
WO2020046132A1 (en) 2018-08-31 2020-03-05 Leiden University Pharmacological chaperones for enzyme treatment therapy
NL2021840B1 (en) 2018-10-19 2020-05-13 Univ Leiden Pharmacological Chaperones For Enzyme Treatment Therapy

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5231185A (en) * 1985-11-19 1993-07-27 Cornell Research Foundation, Inc. Monosaccharide analog-based glycosidase inhibitors
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
AU6050600A (en) * 1999-07-26 2001-02-13 G.D. Searle & Co. Use of long-chain n-alkyl derivates of deoxynojirimycin and a glucocerebrosidaseenzyme for the manufacture of medicament for the treatment of glycolipid storag e diseases

Also Published As

Publication number Publication date
WO2004037373A2 (en) 2004-05-06
AU2003284886A1 (en) 2004-05-13
WO2004037373A3 (en) 2004-07-15

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Legal Events

Date Code Title Description
MK6 Application lapsed section 142(2)(f)/reg. 8.3(3) - pct applic. not entering national phase