ECSP13012978A - Inhibidores de glucosilceramida sintasa - Google Patents

Inhibidores de glucosilceramida sintasa

Info

Publication number
ECSP13012978A
ECSP13012978A ECSP13012978A ECSP13012978A EC SP13012978 A ECSP13012978 A EC SP13012978A EC SP13012978 A ECSP13012978 A EC SP13012978A EC SP13012978 A ECSP13012978 A EC SP13012978A
Authority
EC
Ecuador
Prior art keywords
inhibitors
glucosilceramide
sintasa
treatment
glucosylceramidasintase
Prior art date
Application number
Other languages
English (en)
Inventor
John Marshall
Seng Cheng
Elyse Bourque
Cassandra Celatka
Bradford Hirth
Markus Metz
Zhong Zhao
Renato Skerlj
Yibin Xiang
Katherine Jancisics
Ronald Scheule
Salazar Mario Cabrera
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 ECSP13012978A publication Critical patent/ECSP13012978A/es

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic 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
    • C07D487/08—Bridged systems
    • 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
    • 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
    • A61K31/439—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 the ring forming part of a bridged ring system, e.g. quinuclidine
    • 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
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/454—Non 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
    • 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
    • A61K31/46—8-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
    • 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
    • A61K31/551—Heterocyclic 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
    • 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
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D453/00—Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D453/00—Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
    • C07D453/02—Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids containing not further condensed quinuclidine ring systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D455/00—Heterocyclic compounds containing quinolizine ring systems, e.g. emetine alkaloids, protoberberine; Alkylenedioxy derivatives of dibenzo [a, g] quinolizines, e.g. berberine
    • C07D455/02—Heterocyclic 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
    • 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)
    • C12Y302/01022—Alpha-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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Immunology (AREA)
  • Emergency Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Rheumatology (AREA)
  • Pulmonology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Child & Adolescent Psychology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Oncology (AREA)
  • Endocrinology (AREA)
  • Hospice & Palliative Care (AREA)
  • Communicable Diseases (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Psychiatry (AREA)
  • Vascular Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)

Abstract

La invención está relacionada con inhibidores de glucosilceramidasintasa (GCS) útiles para el tratamiento de enfermedades metabólicas, tales como enfermedades de almacenamiento lisosómico, solos o en combinación con terapia de remplazo enzimático, y para el tratamiento de cáncer.
ECSP13012978 2011-03-18 2013-10-17 Inhibidores de glucosilceramida sintasa ECSP13012978A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161454034P 2011-03-18 2011-03-18
US201261590711P 2012-01-25 2012-01-25

Publications (1)

Publication Number Publication Date
ECSP13012978A true ECSP13012978A (es) 2015-04-30

Family

ID=46879977

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSP13012978 ECSP13012978A (es) 2011-03-18 2013-10-17 Inhibidores de glucosilceramida sintasa

Country Status (39)

Country Link
US (5) US9139580B2 (es)
EP (3) EP4559918A3 (es)
JP (2) JP6117708B2 (es)
KR (1) KR101987736B1 (es)
CN (2) CN103917094B (es)
AR (1) AR085425A1 (es)
AU (2) AU2012231275A1 (es)
BR (2) BR122019017719B1 (es)
CA (1) CA2830540C (es)
CL (1) CL2013002688A1 (es)
CO (1) CO6801749A2 (es)
CR (1) CR20190034A (es)
DK (2) DK2685986T3 (es)
DO (1) DOP2013000209A (es)
EC (1) ECSP13012978A (es)
ES (2) ES2774293T3 (es)
FI (1) FI3673906T3 (es)
GT (1) GT201300215A (es)
HR (2) HRP20200226T1 (es)
HU (2) HUE072576T2 (es)
IL (1) IL228514B (es)
LT (2) LT3673906T (es)
MA (1) MA35023B1 (es)
MX (3) MX368340B (es)
MY (1) MY173998A (es)
NI (1) NI201300083A (es)
PE (1) PE20140412A1 (es)
PH (1) PH12013501944A1 (es)
PL (2) PL3673906T3 (es)
PT (2) PT3673906T (es)
PY (1) PY1212975A (es)
RS (2) RS59963B1 (es)
RU (2) RU2018104472A (es)
SI (2) SI2685986T1 (es)
TW (2) TWI634119B (es)
UA (1) UA118248C2 (es)
UY (1) UY33958A (es)
WO (1) WO2012129084A2 (es)
ZA (1) ZA201307016B (es)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MY173998A (en) * 2011-03-18 2020-03-03 Genzyme Corp Glucosylceramide synthase inhibitors
AU2019222801B2 (en) * 2012-09-11 2021-02-18 Genzyme Corporation Glucosylceramide synthase inhibitors
JOP20130273B1 (ar) 2012-09-11 2021-08-17 Genzyme Corp مثبطات انزيم (سينثاز) غلوكوسيل سيراميد
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
CN108658970A (zh) 2013-03-15 2018-10-16 建新公司 制备葡萄糖基神经酰胺合酶抑制剂的方法
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
EP3079695B1 (en) * 2013-12-11 2021-03-17 Genzyme Corporation Glucosylceramide synthase inhibitors
US10100012B2 (en) * 2014-05-30 2018-10-16 Sphaera Pharma Pvt. Ltd. Compounds as anti-tubercular agents
US10202340B2 (en) 2015-02-02 2019-02-12 The Regents Of The University Of Michigan Glucosylceramide synthase inhibitors and therapeutic methods using the same
TW201642855A (zh) * 2015-03-10 2016-12-16 健臻公司 用於治療蛋白質病變之方法
EP3317282B1 (en) 2015-07-01 2020-12-09 Northwestern University Substituted 4-methyl-pyrrolo[1.2-a]pyrimidine-8-carboxamide compounds and uses thereof for modulating glucocerebrosidase activity
US10167270B2 (en) * 2015-07-01 2019-01-01 Northwestern University Substituted quinazoline compounds and uses thereof for modulating glucocerebrosidase activity
AR107483A1 (es) * 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
JP6940515B2 (ja) * 2016-03-25 2021-09-29 ジェンザイム・コーポレーション タンパク質症のバイオマーカーおよびその使用
EP3318277A1 (en) 2016-11-04 2018-05-09 Institut du Cerveau et de la Moelle Epiniere-ICM Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases
US11001891B2 (en) 2016-11-14 2021-05-11 Berg Llc Methods for treating Parkinson's disease
PT3562821T (pt) 2016-12-28 2021-03-03 Minoryx Therapeutics S L Compostos de isoquinolina, métodos para a sua preparação e utilizações terapêuticas dos mesmos em condições associadas à alteração da atividade da beta-galactosidade
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
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 (en) * 2017-11-14 2019-05-23 Merck Sharp & Dohme Corp. Novel substituted biaryl compounds as indoleamine 2,3-dioxygenase (ido) inhibitors
EP3728265A1 (en) 2017-12-21 2020-10-28 BIAL - BioTech Investments, Inc. Crystalline substituted cyclohexyl pyrazolo[1,5-a]pyrimidinyl carboxamide compound and therapeutic uses thereof
US11459325B2 (en) 2018-01-31 2022-10-04 Takeda Pharmaceutical Company Limited Heterocyclic compound
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
KR102233530B1 (ko) * 2018-11-22 2021-03-31 (주)프로스테믹스 엑소좀 및 이의 다양한 용도
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
MX2021009384A (es) * 2019-02-04 2021-09-10 Genzyme Corp Metodos para el tratamiento contra sintomas y trastornos asociados a enfermedades del almacenamiento lisosomico.
PT3920912T (pt) 2019-02-04 2025-09-09 Genzyme Corp Tratamento de ciliopatias usando inibidores da glucosilceramida sintase (gcs)
WO2020193746A1 (en) 2019-03-28 2020-10-01 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for predicting the survival of patients suffering from melanoma
EP4005638A4 (en) * 2019-07-29 2023-07-05 Takeda Pharmaceutical Company Limited Heterocyclic compound
JP7698320B2 (ja) 2019-10-23 2025-06-25 ザ・リージェンツ・オブ・ザ・ユニバーシティ・オブ・ミシガン グルコシルセラミドシンターゼ阻害剤およびそれを使用する治療方法
KR20210059633A (ko) * 2019-11-15 2021-05-25 주식회사유한양행 신규의 1,2,3,4-테트라하이드로나프탈렌 모이어티를 갖는 유도체 또는 이의 약학적으로 허용가능한 염, 이의 제조방법, 및 이를 포함하는 약학 조성물
WO2021096238A1 (en) * 2019-11-15 2021-05-20 Yuhan Corporation Novel derivatives having 2,3-dihydro-1h-indene or 2,3-dihydrobenzofuran moiety or pharmaceutically acceptable salt thereof and pharmaceutical compositions comprising the same
US20230107499A1 (en) 2019-11-25 2023-04-06 Gain Therapeutics Sa Heteroaryl compounds and therapeutic uses thereof in conditions associated with the alteration of the activity of beta-glucocerebrosidase
JP2023503470A (ja) 2019-11-25 2023-01-30 ゲイン セラピューティクス エスアー ガラクトセレブロシダーゼの活性の変質に関連する状態におけるアリールおよびヘテロアリール化合物、ならびにそれらの治療的使用
CN115397423A (zh) 2020-02-03 2022-11-25 增益治疗股份有限公司 用于治疗mps1的组合疗法
EP4100009A1 (en) * 2020-02-03 2022-12-14 Genzyme Corporation Methods for treating neurological symptoms associated with lysosomal storage diseases
WO2021214771A1 (en) 2020-04-23 2021-10-28 The Israel Institute of Biological Research (IIBR) Glucosylceramide synthase inhibitors for prevention and treatment of viral diseases
WO2021221953A1 (en) 2020-04-28 2021-11-04 The Regents Of The University Of Michigan Pyridine inhibitors of glucosylceramide synthase and therapeutic methods using the same
CA3186766A1 (en) 2020-07-24 2022-01-27 Danielle Combessis Pharmaceutical compositions comprising venglustat
AU2021317180A1 (en) * 2020-07-30 2023-03-23 Genzyme Corporation Methods for reducing glycosphingolipid concentration in brain tissue and methods of treatment of neurodegenerative diseases involving the same
WO2022240116A1 (en) * 2021-05-11 2022-11-17 Yuhan Corporation Novel compounds having inhibitory activity against glucosylceramide synthase or pharmaceutically acceptable salt thereof, processes for preparing the same, and pharmaceutical compositions comprising the same
WO2023042177A1 (en) 2021-09-20 2023-03-23 Gt Gain Therapeutics Sa Enantiomers of 5-((7-chloroisoquinolin-1-yl)amino)-n-(6-methoxy-1,2,3,4-tetrahydronaphthalen-2-yl)picolinamide
WO2023239892A1 (en) * 2022-06-10 2023-12-14 Arkuda Therapeutics Compounds for modulation of cd68 and uses thereof
EP4608828A1 (en) * 2022-10-24 2025-09-03 Yuhan Corporation Improved processes for preparing dimethyl-2,3-dihydro-1h-indene derivatives
JP2025537478A (ja) * 2022-10-24 2025-11-18 ユーハン・コーポレイション ジメチル－２，３－ジヒドロ－１ｈ－インデン誘導体の新規塩およびその製造方法
IL321165A (en) 2022-12-01 2025-07-01 Genzyme Corp Vanglustat in combination with a strong or moderate CYP3A4 inhibitor
AU2023418678A1 (en) 2022-12-30 2025-07-17 Gt Gain Therapeutics Sa Method of treating tauopathies
WO2025094117A1 (en) 2023-11-02 2025-05-08 Gt Gain Therapeutics Sa Substituted indolyl compounds, compositions thereof, and therapeutic uses thereof
WO2025159544A1 (en) * 2024-01-26 2025-07-31 Yuhan Corporation Pharmaceutical composition for preventing or treating gaucher disease
WO2025215582A1 (en) 2024-04-11 2025-10-16 Gt Gain Therapeutics Sa Substituted pyrazolopyridine and pyrazolopyrimidine compounds and their use
WO2025219952A1 (en) 2024-04-19 2025-10-23 Genzyme Corporation Venglustat for use in methods for reducing disease related biomarker levels in patients with ganglioside storage disorders
WO2025219951A1 (en) 2024-04-19 2025-10-23 Genzyme Corporation Venglustat for use in methods for reducing disease related biomarker levels in patients with ganglioside storage disorders
WO2025262570A1 (en) 2024-06-17 2025-12-26 Genzyme Corporation Venglustat as an inhibitor of glucosylceramide synthase in subjects having hepatic impairment
CN118496159B (zh) * 2024-07-18 2024-09-24 广州医科大学附属市八医院 一种化合物在制备具有预防和/或治疗肝纤维化作用的药物中的应用

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BE640616A (es) 1962-12-19
US3492397A (en) 1967-04-07 1970-01-27 Warner Lambert Pharmaceutical Sustained release dosage in the pellet form and process thereof
US4060598A (en) 1967-06-28 1977-11-29 Boehringer Mannheim G.M.B.H. Tablets coated with aqueous resin dispersions
US3538214A (en) 1969-04-22 1970-11-03 Merck & Co Inc Controlled release medicinal tablets
US4173626A (en) 1978-12-11 1979-11-06 Merck & Co., Inc. Sustained release indomethacin
JPH0769205B2 (ja) * 1986-10-03 1995-07-26 マイクロ・モーション・インコーポレーテッド 密度変化の影響を受けないコリオリ質量流量計
IE63474B1 (en) 1987-12-24 1995-04-19 Wyeth John & Brother Ltd Heterocyclic compounds
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5549892A (en) 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
IT1228293B (it) * 1989-02-06 1991-06-07 Angeli Inst Spa Benzoderivati di composti eterociclici contenenti azoto.
US5272071A (en) 1989-12-22 1993-12-21 Applied Research Systems Ars Holding N.V. Method for the modification of the expression characteristics of an endogenous gene of a given cell line
US5733761A (en) 1991-11-05 1998-03-31 Transkaryotic Therapies, Inc. Protein production and protein delivery
EP0747355A4 (en) 1994-02-10 1997-04-09 Yamanouchi Pharma Co Ltd NEW CARBAMATES AND MEDICINAL PRODUCTS CONTAINING THEM
JPH08198751A (ja) * 1995-01-19 1996-08-06 Yamanouchi Pharmaceut Co Ltd カルバメート誘導体
KR19990067403A (ko) * 1995-11-08 1999-08-16 이곤 이 버그 1-아자바이시클로헵탄 유도체 및 이의 약제학적용도
WO2000009153A1 (en) 1997-10-29 2000-02-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
US6953855B2 (en) * 1998-12-11 2005-10-11 Targacept, Inc. 3-substituted-2(arylalkyl)-1-azabicycloalkanes and methods of use thereof
FR2791678B1 (fr) * 1999-03-30 2001-05-04 Synthelabo Derives de 1,4-diazabicyclo [3.2.2] nonane-4-carboxylates et -carboxamides, leur preparation et leur application en therapeutique
GB0010955D0 (en) * 2000-05-05 2000-06-28 Novartis Ag Organic compounds
NZ526580A (en) * 2000-12-22 2005-04-29 Almirall Prodesfarma Ag Quinuclidine carbamate derivatives and their use as M3 antagonists
ES2275808T3 (es) * 2001-02-06 2007-06-16 Pfizer Products Inc. Composiciones farmaceuticas para el tratamiento de trastornos del snc y otros trastornos.
JP2003267977A (ja) * 2002-03-14 2003-09-25 Yamanouchi Pharmaceut Co Ltd キヌクリジン誘導体
ES2203327B1 (es) * 2002-06-21 2005-06-16 Almirall Prodesfarma, S.A. Nuevos carbamatos de quinuclidina y composiciones farmaceuticas que los contienen.
EP1534676B1 (en) * 2002-07-17 2012-09-12 Actelion Pharmaceuticals Ltd. Piperidinetriol derivatives as inhibitors of glycosylceramide synthase
JPWO2004011430A1 (ja) 2002-07-25 2005-11-24 アステラス製薬株式会社 ナトリウムチャネル阻害剤
SE0202598D0 (sv) * 2002-09-02 2002-09-02 Astrazeneca Ab Alpha-7 Nicotinic receptor agonists and statins in combination
CN102657651A (zh) * 2002-12-06 2012-09-12 范因斯坦医学研究院 用α 7受体结合胆碱能激动剂抑制炎症
GB0400812D0 (en) 2004-01-14 2004-02-18 Celltech R&D Ltd Novel compounds
JP2007186422A (ja) * 2004-01-28 2007-07-26 Astellas Pharma Inc アリールスルフィド誘導体
AU2005258274A1 (en) * 2004-06-23 2006-01-05 The Feinstein For Medical Research Method of treating ileus by pharmacological activation of cholinergic receptors
CA2586761A1 (en) 2004-11-10 2006-05-18 Genzyme Corporation Methods of treating diabetes mellitus
US20090318491A1 (en) * 2006-01-27 2009-12-24 Yale Univeristy Cytisine and Acetylcholine Analogs and Methods of Treating Mood Disorders
US7840109B2 (en) * 2006-08-14 2010-11-23 Adc Telecommunications, Inc. Factory spliced cable assembly
WO2009073564A1 (en) * 2007-11-29 2009-06-11 The Hospital For Sick Children Compositions and methods for treating lysosomal disorders
US8642660B2 (en) * 2007-12-21 2014-02-04 The University Of Rochester Method for altering the lifespan of eukaryotic organisms
WO2010014554A1 (en) * 2008-07-28 2010-02-04 Genzyme Corporation Glucosylceramide synthase inhibition for the treatment of collapsing glomerulopathy and other glomerular disease
WO2010015816A2 (en) * 2008-08-06 2010-02-11 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
EP2154136A1 (en) * 2008-08-08 2010-02-17 CHIESI FARMACEUTICI S.p.A. Quinuclidine carbonate derivatives and medicinal compositions thereof
WO2010091104A1 (en) * 2009-02-06 2010-08-12 Exelixis, Inc. Glucosylceramide synthase inhibitors
WO2010091164A1 (en) * 2009-02-06 2010-08-12 Exelixis, Inc. Inhibitors of glucosylceramide synthase
US20100242352A1 (en) * 2009-06-09 2010-09-30 Sundrop Fuels, Inc. Systems and methods for reactor and receiver control of flux profile
CN102596232A (zh) 2009-08-28 2012-07-18 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
MY173998A (en) * 2011-03-18 2020-03-03 Genzyme Corp Glucosylceramide synthase inhibitors

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