MA35023B1 - Inhibiteurs de la glucosylcéramide synthase - Google Patents

Inhibiteurs de la glucosylcéramide synthase

Info

Publication number
MA35023B1
MA35023B1 MA36299A MA36299A MA35023B1 MA 35023 B1 MA35023 B1 MA 35023B1 MA 36299 A MA36299 A MA 36299A MA 36299 A MA36299 A MA 36299A MA 35023 B1 MA35023 B1 MA 35023B1
Authority
MA
Morocco
Prior art keywords
inhibitors
glucosylceramide synthase
treat
glucosylceramide
synthase
Prior art date
Application number
MA36299A
Other languages
English (en)
Inventor
Elyse Bourque
Cassandra Celatka
Bradford Hirth
Markus Metz
Zhong Zhao
Renato Skerlj
Yibin Xiang
Katherine Jancisics
John Marshall
Seng Cheng
Ronald Scheule
Mario Cabrera-Salazar
Andrew Good
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of MA35023B1 publication Critical patent/MA35023B1/fr

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/08Bridged systems
    • 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
    • A61K31/55Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • 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
    • A61K31/4523Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/454Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
    • 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
    • A61K31/468-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
    • 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
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
    • 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
    • A61K31/55Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A61K31/551Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having two nitrogen atoms, e.g. dilazep
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P11/06Antiasthmatics
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    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
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    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
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    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • AHUMAN NECESSITIES
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D453/00Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D455/00Heterocyclic compounds containing quinolizine ring systems, e.g. emetine alkaloids, protoberberine; Alkylenedioxy derivatives of dibenzo [a, g] quinolizines, e.g. berberine
    • C07D455/02Heterocyclic compounds containing quinolizine ring systems, e.g. emetine alkaloids, protoberberine; Alkylenedioxy derivatives of dibenzo [a, g] quinolizines, e.g. berberine containing not further condensed quinolizine ring systems
    • 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
    • A61K31/439Heterocyclic 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 the ring forming part of a bridged ring system, e.g. quinuclidine
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D453/00Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
    • C07D453/02Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids containing not further condensed quinuclidine ring systems
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-galactosidase (3.2.1.22)

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Immunology (AREA)
  • Emergency Medicine (AREA)
  • Rheumatology (AREA)
  • Urology & Nephrology (AREA)
  • Pulmonology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Psychiatry (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Vascular Medicine (AREA)

Abstract

L'invention concerne des inhibiteurs de la glucosylcéramide synthase (GCS) utilisés pour traiter des maladies métaboliques, telles que des maladies du stockage lysosomal, seuls ou en association avec une enzymothérapie substitutive, et pour traiter le cancer.
MA36299A 2011-03-18 2012-03-16 Inhibiteurs de la glucosylcéramide synthase MA35023B1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161454034P 2011-03-18 2011-03-18
US201261590711P 2012-01-25 2012-01-25
PCT/US2012/029417 WO2012129084A2 (fr) 2011-03-18 2012-03-16 Inhibiteurs de la glucosylcéramide synthase

Publications (1)

Publication Number Publication Date
MA35023B1 true MA35023B1 (fr) 2014-04-03

Family

ID=46879977

Family Applications (1)

Application Number Title Priority Date Filing Date
MA36299A MA35023B1 (fr) 2011-03-18 2012-03-16 Inhibiteurs de la glucosylcéramide synthase

Country Status (36)

Country Link
US (5) US9139580B2 (fr)
EP (2) EP3673906A1 (fr)
JP (2) JP6117708B2 (fr)
KR (1) KR101987736B1 (fr)
CN (2) CN107080747B (fr)
AR (1) AR085425A1 (fr)
AU (2) AU2012231275A1 (fr)
BR (2) BR122019017719B1 (fr)
CA (1) CA2830540C (fr)
CL (1) CL2013002688A1 (fr)
CO (1) CO6801749A2 (fr)
CR (1) CR20190034A (fr)
DK (1) DK2685986T3 (fr)
DO (1) DOP2013000209A (fr)
EC (1) ECSP13012978A (fr)
ES (1) ES2774293T3 (fr)
GT (1) GT201300215A (fr)
HR (1) HRP20200226T1 (fr)
HU (1) HUE047888T2 (fr)
IL (1) IL228514B (fr)
LT (1) LT2685986T (fr)
MA (1) MA35023B1 (fr)
MX (3) MX365245B (fr)
MY (1) MY173998A (fr)
NI (1) NI201300083A (fr)
PE (1) PE20140412A1 (fr)
PL (1) PL2685986T3 (fr)
PT (1) PT2685986T (fr)
RS (1) RS59963B1 (fr)
RU (2) RU2645675C2 (fr)
SI (1) SI2685986T1 (fr)
TW (2) TWI634119B (fr)
UA (1) UA118248C2 (fr)
UY (1) UY33958A (fr)
WO (1) WO2012129084A2 (fr)
ZA (1) ZA201307016B (fr)

Families Citing this family (48)

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DK2685986T3 (da) * 2011-03-18 2020-03-02 Genzyme Corp Glucosylceramidsyntasehæmmer
AU2019222801B2 (en) * 2012-09-11 2021-02-18 Genzyme Corporation Glucosylceramide synthase inhibitors
MA37975B2 (fr) * 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
JO3713B1 (ar) * 2013-03-15 2021-01-31 Genzyme Corp أشكال ملح (s)-كوينوكليدين-3-يل(2-(2-(4-فلوروفينيل)ثيازول-4-يل)بروبان-2-يل)كارباميت
EP2970251B1 (fr) * 2013-03-15 2019-02-27 Genzyme Corporation Procédé de préparation d'inhibiteurs de la glucosylcéramide synthase
CN104418851A (zh) * 2013-09-02 2015-03-18 上海龙翔生物医药开发有限公司 奎宁环衍生物的制备方法及纯化方法
PL3079695T3 (pl) * 2013-12-11 2021-09-27 Genzyme Corporation Inhibitory syntazy glukozyloceramidu
EP3148518B1 (fr) * 2014-05-30 2022-11-30 Sphaera Pharma Pvt. Ltd. Nouveaux composés utilisés comme agents anti-tuberculeux
JP6749923B2 (ja) 2015-02-02 2020-09-02 ザ リージェンツ オブ ザ ユニバーシティー オブ ミシガン グルコシルセラミドシンターゼ阻害剤及びそれを使用した治療方法
TW201642855A (zh) * 2015-03-10 2016-12-16 健臻公司 用於治療蛋白質病變之方法
JP2018522872A (ja) * 2015-07-01 2018-08-16 ノースウェスタン ユニバーシティ 置換4−メチルピロロ[1,2−a]ピリミジン−8−カルボキサミド化合物及びグルコセレブロシダーゼ活性の調節のためのその使用
WO2017004405A1 (fr) * 2015-07-01 2017-01-05 Northwestern University Composés de quinazoline substituée et leurs utilisations pour moduler l'activité de la glucocérébrosidase
AR107483A1 (es) * 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
KR20180124971A (ko) 2016-03-25 2018-11-21 젠자임 코포레이션 단백질병증의 바이오마커 및 이의 용도
EP3318277A1 (fr) 2016-11-04 2018-05-09 Institut du Cerveau et de la Moelle Epiniere-ICM Inhibiteurs de la synthase de glucosylcéramide pour le traitement de maladies du motoneurone
US11001891B2 (en) 2016-11-14 2021-05-11 Berg Llc Methods for treating Parkinson's disease
CN110494432B (zh) 2016-12-28 2022-08-12 米尼奥尔克斯治疗有限公司 异喹啉化合物,其制备方法及其在与β-半乳糖苷酶活性改变相关的病症中的治疗用途
US11174242B2 (en) 2016-12-29 2021-11-16 Minoryx Therapeutics S.L. Heteroaryl compounds and their use
CN108341822B (zh) * 2017-01-23 2021-04-16 广州市恒诺康医药科技有限公司 Fxr受体调节剂及其制备方法和应用
WO2019099294A1 (fr) * 2017-11-14 2019-05-23 Merck Sharp & Dohme Corp. Nouveaux composés biaryles substitués utilisés en tant qu'inhibiteurs de l'indoléamine 2,3-dioxygénase (ido)
WO2019126776A1 (fr) 2017-12-21 2019-06-27 Lysosomal Therapeutics Inc. Composé de cyclohexyl pyrazolo[1,5-a]pyrimidinyl carboxamide substitué cristallin et ses utilisations thérapeutiques
WO2019151269A1 (fr) * 2018-01-31 2019-08-08 武田薬品工業株式会社 Composé hétérocyclique
US10519157B2 (en) * 2018-02-09 2019-12-31 X-Cutag Therapeutics, Inc. Deuterated compounds for treating Fabry, Gaucher, Parkinson's and related diseases and conditions, and compositions and methods thereof
KR102342901B1 (ko) * 2018-11-22 2021-12-24 (주)프로스테믹스 엑소좀 및 이의 다양한 용도
KR102342902B1 (ko) * 2018-11-22 2021-12-24 (주)프로스테믹스 엑소좀 및 이의 다양한 용도
KR102233530B1 (ko) * 2018-11-22 2021-03-31 (주)프로스테믹스 엑소좀 및 이의 다양한 용도
JP2022519131A (ja) 2019-02-04 2022-03-18 ジェンザイム・コーポレーション グルコシルセラミドシンターゼ(gcs)阻害剤を使用した繊毛病の処置
JP2022520747A (ja) 2019-02-04 2022-04-01 ジェンザイム・コーポレーション リソソーム蓄積性疾患と関連する症状および障害を処置するための方法
WO2020193746A1 (fr) 2019-03-28 2020-10-01 INSERM (Institut National de la Santé et de la Recherche Médicale) Méthodes de prédiction de la survie de patients atteints de mélanome
WO2021020363A1 (fr) 2019-07-29 2021-02-04 武田薬品工業株式会社 Composé hétérocyclique
WO2021081141A1 (fr) 2019-10-23 2021-04-29 The Regents Of The University Of Michigan Inhibiteurs de la glucosylcéramide synthase et procédés thérapeutiques les utilisant
CN114746422B (zh) * 2019-11-15 2024-03-12 柳韩洋行 具有1,2,3,4-四氢萘部分的衍生物或其药学上可接受的盐以及包含它们的药物组合物
CN114829361B (zh) * 2019-11-15 2024-07-16 柳韩洋行 具有2,3-二氢-1h-茚或2,3-二氢苯并呋喃部分的衍生物或其药学上可接受盐及含其的药物组合物
CA3158288A1 (fr) 2019-11-25 2021-06-03 Ana Maria Garcia Collazo Composes heteroaryle et leurs utilisations therapeutiques dans des conditions associees a l'alteration de l'activite de la beta-glucocerebrosidase
CA3158290A1 (fr) 2019-11-25 2021-06-03 Ana Maria Garcia Collazo Composes aryle et heteroaryle, leurs utilisations therapeutiques dans des conditions associees a l'alteration de l'activite de la galactocerebrosidase
JP2023512281A (ja) 2020-02-03 2023-03-24 ゲイン セラピューティクス エスアー Mps1を処置するための併用療法
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