CA2699864C - Method of treating gaucher disease - Google Patents

Method of treating gaucher disease Download PDF

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Publication number
CA2699864C
CA2699864C CA2699864A CA2699864A CA2699864C CA 2699864 C CA2699864 C CA 2699864C CA 2699864 A CA2699864 A CA 2699864A CA 2699864 A CA2699864 A CA 2699864A CA 2699864 C CA2699864 C CA 2699864C
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CA
Canada
Prior art keywords
compound
ambroxol
glucocerebrosidase
gcase
patient
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Active
Application number
CA2699864A
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English (en)
French (fr)
Other versions
CA2699864A1 (en
Inventor
Jan E. Blanchard
Eric D. Brown
Michael B. Tropak
Don J. Mahuran
Justin D. Buttner
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.)
McMaster University
Hospital for Sick Children HSC
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McMaster University
Hospital for Sick Children HSC
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Publication of CA2699864A1 publication Critical patent/CA2699864A1/en
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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
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/137—Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine or methadone
    • 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
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/136—Amines having aromatic rings, e.g. ketamine, nortriptyline having the amino group directly attached to the aromatic ring, e.g. benzeneamine
    • 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
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/138—Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
    • 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
    • A61K31/16—Amides, e.g. hydroxamic acids
    • A61K31/165—Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
    • A61K31/167—Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the nitrogen of a carboxamide group directly attached to the aromatic ring, e.g. lidocaine, paracetamol
    • 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
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/235—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids having an aromatic ring attached to a carboxyl group
    • 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
    • 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
    • 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
    • A61K31/54—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame
    • A61K31/5415—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame ortho- or peri-condensed with carbocyclic ring systems, e.g. phenothiazine, chlorpromazine, piroxicam
    • 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
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A61K31/553—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having at least one nitrogen and one oxygen as ring hetero atoms, e.g. loxapine, staurosporine
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Emergency Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CA2699864A 2007-09-17 2008-09-16 Method of treating gaucher disease Active CA2699864C (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US97296807P 2007-09-17 2007-09-17
US60/972,968 2007-09-17
US6555008P 2008-02-12 2008-02-12
US61/065,550 2008-02-12
US6568408P 2008-02-13 2008-02-13
US61/065,684 2008-02-13
PCT/US2008/010768 WO2009038695A1 (en) 2007-09-17 2008-09-16 Method of treating gaucher disease

Publications (2)

Publication Number Publication Date
CA2699864A1 CA2699864A1 (en) 2009-03-26
CA2699864C true CA2699864C (en) 2016-08-02

Family

ID=39875224

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2699864A Active CA2699864C (en) 2007-09-17 2008-09-16 Method of treating gaucher disease

Country Status (6)

Country Link
US (10) US8124597B2 (https=)
EP (1) EP2200596B1 (https=)
JP (1) JP5337405B2 (https=)
CA (1) CA2699864C (https=)
IL (1) IL204285A (https=)
WO (1) WO2009038695A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102004021992A1 (de) * 2004-05-03 2005-11-24 Boehringer Ingelheim Pharma Gmbh & Co. Kg Topische Zubereitung enthaltend Ambroxol
EP3366688B1 (en) 2010-12-08 2022-02-09 The U.S.A. as represented by the Secretary, Department of Health and Human Services Substituted pyrazolopyrimidines as glucocerebrosidase activators
KR102076585B1 (ko) * 2011-12-22 2020-02-12 센토제너 아이피 게엠베하 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물
KR102227473B1 (ko) 2013-04-15 2021-03-15 삼성디스플레이 주식회사 박막 증착 장치 및 이를 이용한 박막 형성 방법
CN106455661A (zh) * 2014-05-21 2017-02-22 雀巢产品技术援助有限公司 营养物质的个性化补充
US20190254992A1 (en) * 2016-06-29 2019-08-22 Hadasit Medical Research Services & Development Limited Combinations of beta-glycolipides and 4-[(2-amino-3,5-dibromophenyl)methylamino]cyclohexan-1-ol, compositions and uses thereof in the treatment of disorders associated with protein misfolding and protein aggregations
WO2018089797A1 (en) 2016-11-14 2018-05-17 Mingwu Wang Formulations for the treatment of ocular surface diseases and related methods
CN110325516A (zh) 2016-12-29 2019-10-11 米尼奥尔克斯治疗有限公司 杂芳基化合物和它们的用途
JP7369716B2 (ja) * 2018-01-26 2023-10-26 ノイレ， エルエルシー 皮膚バリア機能を改善するためのアンブロキソールの使用
WO2019211205A1 (en) 2018-04-30 2019-11-07 Dorphan S.A. Pharmaceutical compositions for use in the treatment of lysosomal diseases
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
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
KR20210148101A (ko) 2019-02-04 2021-12-07 프리라인 테라퓨틱스 리미티드 폴리뉴클레오티드
WO2025153714A1 (en) * 2024-01-19 2025-07-24 Scenic Biotech Bv Pla2g15 inhibitors for use in the treatment of lysosomal storage diseases, hiv, alzheimer's disease or parkinson's disease
CN118086341B (zh) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ID28460A (id) 1998-07-08 2001-05-24 Lipogenics Inc Komposisi dan metoda untuk perlakuan dan pencegahan penyakit tulang dengan menggunakan tocotrienol
MXPA04002066A (es) * 2001-09-04 2005-02-17 Techno Network Shinoku Co Ltd Farmacos contra influenza.
US20030171391A1 (en) * 2002-01-25 2003-09-11 Boehringer Ingelheim Pharma Gmbh & Co. Kg Ambroxol for the treatment of chronic pain
DE10303229B4 (de) * 2003-01-28 2007-07-26 Keyneurotek Ag Ambroxol und Inhibitoren des Angiotensin Converting-Enzyms (ACE) umfassende Arzneimittel Arzneimittel-Zubereitung und ihre Verwendung zur Behandlung von neurodegenerativen Erkrankungen
DE10332472A1 (de) * 2003-07-16 2005-02-03 Boehringer Ingelheim Pharma Gmbh & Co. Kg Ambroxol für die Behandlung von Tinnitus
US7488721B2 (en) * 2004-05-13 2009-02-10 Mcmaster University Real time methylumbelliferone-based assay
EP1850840A2 (en) * 2005-01-13 2007-11-07 Sirtris Pharmaceuticals, Inc. Novel compositions for preventing and treating neurodegenerative and blood coagulation disorders
JP2008528510A (ja) * 2005-01-20 2008-07-31 サートリス ファーマシューティカルズ， インコーポレイテッド 紅潮および／または薬物誘発性体重増加を処置するためのサーチュイン活性化化合物の使用
US20070149466A1 (en) * 2005-07-07 2007-06-28 Michael Milburn Methods and related compositions for treating or preventing obesity, insulin resistance disorders, and mitochondrial-associated disorders
US8795668B2 (en) * 2005-12-23 2014-08-05 The Regents Of The University Of Michigan Methods for treating pulmonary fibrosis
US20070244184A1 (en) * 2006-01-09 2007-10-18 Simon Fraser University Glycosidase inhibitors and methods of synthesizing same

Also Published As

Publication number Publication date
US8937059B2 (en) 2015-01-20
US11160773B2 (en) 2021-11-02
EP2200596A1 (en) 2010-06-30
IL204285A (en) 2013-10-31
US20160113892A1 (en) 2016-04-28
EP2200596B1 (en) 2012-06-13
US20130090388A1 (en) 2013-04-11
US9415025B2 (en) 2016-08-16
JP2009137933A (ja) 2009-06-25
US9006223B2 (en) 2015-04-14
CA2699864A1 (en) 2009-03-26
US20160331701A1 (en) 2016-11-17
US20090075960A1 (en) 2009-03-19
US20180296502A1 (en) 2018-10-18
WO2009038695A1 (en) 2009-03-26
US9233083B2 (en) 2016-01-12
US20130184352A1 (en) 2013-07-18
US10028922B2 (en) 2018-07-24
US8124597B2 (en) 2012-02-28
US20140235713A1 (en) 2014-08-21
US20200246282A1 (en) 2020-08-06
US10653645B2 (en) 2020-05-19
US8951994B2 (en) 2015-02-10
JP5337405B2 (ja) 2013-11-06
US20140249233A1 (en) 2014-09-04
US20120021487A1 (en) 2012-01-26
US8404668B2 (en) 2013-03-26

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