WO2010116141A3 - Drug combination for the treatment of proteostatic diseases - Google Patents

Drug combination for the treatment of proteostatic diseases Download PDF

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Publication number
WO2010116141A3
WO2010116141A3 PCT/GB2010/000710 GB2010000710W WO2010116141A3 WO 2010116141 A3 WO2010116141 A3 WO 2010116141A3 GB 2010000710 W GB2010000710 W GB 2010000710W WO 2010116141 A3 WO2010116141 A3 WO 2010116141A3
Authority
WO
WIPO (PCT)
Prior art keywords
enzyme
treatment
site
drug combination
diseases
Prior art date
Application number
PCT/GB2010/000710
Other languages
French (fr)
Other versions
WO2010116141A2 (en
Inventor
Francis Xavier Wilson
Richard Storer
Akane Kawamura
Robert Nash
Alan Geoffrey Roach
Jonathon Mark Tinsley
Original Assignee
Summit Corporation Plc
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 Summit Corporation Plc filed Critical Summit Corporation Plc
Publication of WO2010116141A2 publication Critical patent/WO2010116141A2/en
Publication of WO2010116141A3 publication Critical patent/WO2010116141A3/en

Links

Classifications

    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7016—Disaccharides, e.g. lactose, lactulose
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/702—Oligosaccharides, i.e. having three to five saccharide radicals attached to each other by glycosidic linkages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Abstract

Combinations comprising: (a) an active-site-specific pharmacoperone (ASSC) of an enzyme (e.g. a lysosomal enzyme); and (b) a non-active-site-specific pharmacoperone (non-ASSC) of said enzyme which: (i) does not bind to a catalytic site of said enzyme; or (ii) is not a competitive inhibitor of said enzyme; or (iii) is an activator of said enzyme; or (iv) is a non-competitive inhibitor of said enzyme; or (v) binds (e.g. specifically) to an allosteric site of said enzyme; or (vi) does not bind to said enzyme but binds to a chaperone or co- chaperone of said enzyme are described The combinations have various uses in medicine, including the treatment of protein folding diseases (in particular lysosomal storage disorders).
PCT/GB2010/000710 2009-04-09 2010-04-07 Drug combination for the treatment of proteostatic diseases WO2010116141A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB0906159.9A GB0906159D0 (en) 2009-04-09 2009-04-09 Drug combination for the treatment of proteostatic diseases
GB0906159.9 2009-04-09

Publications (2)

Publication Number Publication Date
WO2010116141A2 WO2010116141A2 (en) 2010-10-14
WO2010116141A3 true WO2010116141A3 (en) 2011-03-31

Family

ID=40750361

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2010/000710 WO2010116141A2 (en) 2009-04-09 2010-04-07 Drug combination for the treatment of proteostatic diseases

Country Status (2)

Country Link
GB (1) GB0906159D0 (en)
WO (1) WO2010116141A2 (en)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3578195T3 (en) 2008-06-26 2023-10-30 Zevra Denmark As Use of Hsp70 as a regulator of enzymatic activity
CA2817773A1 (en) 2010-11-30 2012-06-07 Orphazyme Aps Methods for increasing intracellular activity of hsp70
KR20140029469A (en) 2011-04-29 2014-03-10 셀렉타 바이오사이언시즈, 인크. Tolerogenic synthetic nanocarriers to reduce cytotoxic t lymphocyte responses
KR20160003219A (en) 2013-05-03 2016-01-08 셀렉타 바이오사이언시즈, 인크. Tolerogenic synthetic nanocarriers and therapeutic macromolecules for reduced or enhanced pharmacodynamic effects
EA201790550A1 (en) 2014-09-07 2017-08-31 Селекта Байосайенсиз, Инк. METHODS AND COMPOSITIONS FOR REDUCING IMMUNE RESPONSES TO CORRECTIVE VIRAL TRANSFER VECTOR GENES
KR102582559B1 (en) 2014-09-15 2023-09-26 제브라 덴마크 에이/에스 Arimoclomol formulation
WO2017178029A1 (en) 2016-04-13 2017-10-19 Orphazyme Aps Heat shock proteins and cholesterol homeostasis
CN109069496A (en) 2016-04-29 2018-12-21 奥菲泽米有限公司 For treating the arimoclomol of glucocerebrosidase related disease
KR20190124295A (en) 2017-03-11 2019-11-04 셀렉타 바이오사이언시즈, 인크. Methods and compositions related to combination treatment with synthetic nanocarriers comprising anti-inflammatory agents and immunosuppressants
IL303026A (en) 2020-11-19 2023-07-01 Zevra Denmark As Processes for preparing arimoclomol citrate and intermediates thereof

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050113415A1 (en) * 1998-06-01 2005-05-26 Mount Sinai School Of Medicine Of New York University Method for enhancing mutant enzyme activities in gaucher disease
WO2006133446A2 (en) * 2005-06-08 2006-12-14 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes
WO2007150064A2 (en) * 2006-06-23 2007-12-27 Amicus Therapeutics, Inc. METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE
WO2008054947A2 (en) * 2006-10-02 2008-05-08 The Brigham And Women's Hospital, Inc. Structure of acid beta-glucosidase and methods for identifying therapeutic agents
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2009049421A1 (en) * 2007-10-18 2009-04-23 The Hospital For Sick Children Compositions and methods for enhancing enzyme activity in gaucher, gm1-gangliosidosis/morquio b disease, and parkinson's disease
WO2009066069A1 (en) * 2007-11-21 2009-05-28 Summit Corporation Plc Treatment of protein folding disorders
WO2010015816A2 (en) * 2008-08-06 2010-02-11 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050113415A1 (en) * 1998-06-01 2005-05-26 Mount Sinai School Of Medicine Of New York University Method for enhancing mutant enzyme activities in gaucher disease
US20060100241A1 (en) * 1998-06-01 2006-05-11 Mount Sinai School Of Medicine Of New York University Method for enhancing mutant protein activity
WO2006133446A2 (en) * 2005-06-08 2006-12-14 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes
WO2007150064A2 (en) * 2006-06-23 2007-12-27 Amicus Therapeutics, Inc. METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE
WO2008054947A2 (en) * 2006-10-02 2008-05-08 The Brigham And Women's Hospital, Inc. Structure of acid beta-glucosidase and methods for identifying therapeutic agents
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2009049421A1 (en) * 2007-10-18 2009-04-23 The Hospital For Sick Children Compositions and methods for enhancing enzyme activity in gaucher, gm1-gangliosidosis/morquio b disease, and parkinson's disease
WO2009066069A1 (en) * 2007-11-21 2009-05-28 Summit Corporation Plc Treatment of protein folding disorders
WO2010015816A2 (en) * 2008-08-06 2010-02-11 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ASANO N: "Sugar-mimicking glycosidase inhibitors: bioactivity and application", CMLS CELLULAR AND MOLECULAR LIFE SCIENCES, BIRKHÄUSER-VERLAG, BA, vol. 66, no. 9, 12 January 2009 (2009-01-12), pages 1479 - 1492, XP019700781, ISSN: 1420-9071 *
RICHARD A STEET ET AL: "The iminosugar isofagomine increases the activity of N370S mutant acid beta glucosidase in Gaucher fibroblasts by several mechanisms", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES (PNAS), NATIONAL ACADEMY OF SCIENCE, US, vol. 103, no. 37, 1 January 2006 (2006-01-01), pages 13813 - 13818, XP008129303, ISSN: 0027-8424, [retrieved on 20060831], DOI: DOI:10.1073/PNAS.0605928103 *

Also Published As

Publication number Publication date
WO2010116141A2 (en) 2010-10-14
GB0906159D0 (en) 2009-05-20

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