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UA118248C2 - Інгібітори глюкозилцерамідсинтази - Google Patents

Інгібітори глюкозилцерамідсинтази

Info

Publication number
UA118248C2
UA118248C2 UAA201312420A UAA201312420A UA118248C2 UA 118248 C2 UA118248 C2 UA 118248C2 UA A201312420 A UAA201312420 A UA A201312420A UA A201312420 A UAA201312420 A UA A201312420A UA 118248 C2 UA118248 C2 UA 118248C2
Authority
UA
Ukraine
Prior art keywords
glucosylceramide synthase
synthase inhibitors
treatment
inhibitors
glucosylceramide
Prior art date
Application number
UAA201312420A
Other languages
English (en)
Inventor
Еліз Бурке
Кассандра СЕЛАТКА
Бредфорд Херт
Маркус МЕТЦ
Чжун ЧЖАО
Ренато СКЕРЛДЖ
Ібінь Сян
Кетрін Джансізікс
Джон Маршалл
Сен Чен
Рональд ШОЙЛЕ
Маріо Кабрера-Салазар
Ендрю Гуд
Original Assignee
Джензім Корпорейшн
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 Джензім Корпорейшн filed Critical Джензім Корпорейшн
Publication of UA118248C2 publication Critical patent/UA118248C2/uk

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Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P11/06—Antiasthmatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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)

Landscapes

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

Abstract

Даний винахід стосується інгібіторів глюкозилцерамідсинтази (GCS), які можна застосовувати для лікування метаболічних захворювань, таких як хвороби лізосомного накопичення, або у вигляді монотерапії, або в поєднанні з ферментозамісною терапією, а також для лікування раку.
UAA201312420A 2011-03-18 2012-03-16 Інгібітори глюкозилцерамідсинтази UA118248C2 (uk)

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 (en) 2011-03-18 2012-03-16 Glucosylceramide synthase inhibitors

Publications (1)

Publication Number Publication Date
UA118248C2 true UA118248C2 (uk) 2018-12-26

Family

ID=46879977

Family Applications (1)

Application Number Title Priority Date Filing Date
UAA201312420A UA118248C2 (uk) 2011-03-18 2012-03-16 Інгібітори глюкозилцерамідсинтази

Country Status (38)

Country Link
US (5) US9139580B2 (uk)
EP (3) EP2685986B1 (uk)
JP (2) JP6117708B2 (uk)
KR (1) KR101987736B1 (uk)
CN (2) CN103917094B (uk)
AR (1) AR085425A1 (uk)
AU (2) AU2012231275A1 (uk)
BR (2) BR112013023774B1 (uk)
CA (1) CA2830540C (uk)
CL (1) CL2013002688A1 (uk)
CO (1) CO6801749A2 (uk)
CR (1) CR20190034A (uk)
DK (2) DK3673906T3 (uk)
DO (1) DOP2013000209A (uk)
EC (1) ECSP13012978A (uk)
ES (2) ES3037907T3 (uk)
FI (1) FI3673906T3 (uk)
GT (1) GT201300215A (uk)
HR (2) HRP20200226T1 (uk)
HU (2) HUE072576T2 (uk)
IL (1) IL228514B (uk)
LT (2) LT3673906T (uk)
MA (1) MA35023B1 (uk)
MX (3) MX365245B (uk)
MY (1) MY173998A (uk)
NI (1) NI201300083A (uk)
PE (1) PE20140412A1 (uk)
PH (1) PH12013501944A1 (uk)
PL (2) PL2685986T3 (uk)
PT (2) PT3673906T (uk)
RS (2) RS59963B1 (uk)
RU (2) RU2018104472A (uk)
SI (2) SI2685986T1 (uk)
TW (2) TWI546301B (uk)
UA (1) UA118248C2 (uk)
UY (1) UY33958A (uk)
WO (1) WO2012129084A2 (uk)
ZA (1) ZA201307016B (uk)

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
LT3673906T (lt) * 2011-03-18 2025-08-11 Genzyme Corporation Gliukozilceramido sintazės inhibitoriai
JOP20130273B1 (ar) * 2012-09-11 2021-08-17 Genzyme Corp مثبطات انزيم (سينثاز) غلوكوسيل سيراميد
AU2019222801B2 (en) * 2012-09-11 2021-02-18 Genzyme Corporation Glucosylceramide synthase inhibitors
RU2015144035A (ru) 2013-03-15 2017-04-20 Джензим Корпорейшн Способ получения ингибиторов глюкозилцерамидсинтазы
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
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
ES2871416T3 (es) * 2013-12-11 2021-10-28 Genzyme Corp Inhibidores de glucosilceramida sintasa
WO2015181837A2 (en) * 2014-05-30 2015-12-03 Sphaera Pharma Pvt. Ltd. Novel compounds as anti-tubercular agents
ES2856898T3 (es) 2015-02-02 2021-09-28 Univ Michigan Regents Inhibidores de glucosilceramida sintasa y métodos terapéuticos que usan los mismos
TW201642855A (zh) * 2015-03-10 2016-12-16 健臻公司 用於治療蛋白質病變之方法
WO2017004408A1 (en) * 2015-07-01 2017-01-05 Northwestern University Substituted 4-methyl-pyrrolo[1.2-a]pyrimidine-8-carboxamide compounds and uses thereof for modulating glucocerebrosidase activity
WO2017004405A1 (en) * 2015-07-01 2017-01-05 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
CA3018745A1 (en) * 2016-03-25 2017-09-28 Genzyme Corporation Biomarkers of proteopathies and uses thereof
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
WO2018090054A1 (en) * 2016-11-14 2018-05-17 Berg Llc Methods for treating parkinson's disease
US11440898B2 (en) 2016-12-28 2022-09-13 Minoryx Therapeutics S.L. Isoquinoline compounds, methods for their preparation, and therapeutic uses thereof in conditions associated with the alteration of the activity of beta galactosidase
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
EP3709986B1 (en) * 2017-11-14 2023-11-01 Merck Sharp & Dohme LLC Novel substituted biaryl compounds as indoleamine 2,3-dioxygenase (ido) inhibitors
US20200339587A1 (en) 2017-12-21 2020-10-29 Lysosomal Therapeutics Inc. Crystalline substituted cyclohexyl pyrazolo[1,5-a]pyrimidinyl carboxamide compound and therapeutic uses thereof
WO2019151269A1 (ja) * 2018-01-31 2019-08-08 武田薬品工業株式会社 複素環化合物
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
CN113710249A (zh) 2019-02-04 2021-11-26 建新公司 用于治疗溶酶体贮积症相关的症状和病症的方法
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
WO2021081141A1 (en) 2019-10-23 2021-04-29 The Regents Of The University Of Michigan Glucosylceramide synthase inhibitors and therapeutic methods using the same
CN118955495A (zh) * 2019-11-15 2024-11-15 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
US12338237B2 (en) 2019-11-15 2025-06-24 Yuhan Corporation Derivatives having 1,2,3,4-tetrahydronaphthalene moiety or pharmaceutically acceptable salt thereof and pharmaceutical compositions comprising the same
EP4065563A1 (en) 2019-11-25 2022-10-05 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
AU2021217816A1 (en) 2020-02-03 2022-08-11 Gt Gain Therapeutics Sa Combination therapy for treating MPS1
IL295255A (en) 2020-02-03 2022-10-01 Genzyme Corp 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
JP7725076B2 (ja) 2020-04-28 2025-08-19 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合成酵素のピリジン阻害剤及びそれらを使用する治療方法
MX2023001014A (es) 2020-07-24 2023-03-01 Genzyme Corp Composiciones farmaceuticas que comprenden venglustat.
CA3187086A1 (en) * 2020-07-30 2022-02-03 Tanya Zaremba FISCHER Methods for reducing glycosphingolipid concentration in brain tissue and methods of treatment of neurodegenerative diseases involving the same
BR112023022015A2 (pt) * 2021-05-11 2023-12-26 Green Cross Corp Novos compostos que têm atividade inibidora contra glucosilceramida sintase ou sal farmaceuticamente aceitável da mesma, processos para preparar os mesmos e respectivas composições farmacêuticas
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
JP2025534728A (ja) * 2022-10-24 2025-10-17 ユーハン・コーポレイション ジメチル－２，３－ジヒドロ－１ｈ－インデン誘導体の改良された製造方法
EP4608833A1 (en) * 2022-10-24 2025-09-03 Yuhan Corporation Novel salt of dimethyl-2,3-dihydro-1h-indene derivative and processes for preparing the same
WO2024116127A1 (en) 2022-12-01 2024-06-06 Genzyme Corporation Venglustat in combination with a strong or moderate inhibitor of cyp3a4
WO2024141998A1 (en) 2022-12-30 2024-07-04 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
KR20250117295A (ko) * 2024-01-26 2025-08-04 주식회사유한양행 고셔병의 예방 또는 치료용 약제학적 조성물
WO2025215582A1 (en) 2024-04-11 2025-10-16 Gt Gain Therapeutics Sa Substituted pyrazolopyridine and pyrazolopyrimidine compounds and their use
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
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
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 (uk) 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
EP0286670B1 (en) * 1986-10-03 1990-12-19 Micro Motion Incorporated Density insensitive coriolis mass flow rate meter
IE63474B1 (en) 1987-12-24 1995-04-19 Wyeth John & Brother Ltd Heterocyclic compounds
US5549892A (en) 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant 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
US5968502A (en) 1991-11-05 1999-10-19 Transkaryotic Therapies, Inc. Protein production and protein delivery
AU685225B2 (en) 1994-02-10 1998-01-15 Yamanouchi Pharmaceutical Co., Ltd. Novel carbamate derivative and medicinal composition containing the same
JPH08198751A (ja) * 1995-01-19 1996-08-06 Yamanouchi Pharmaceut Co Ltd カルバメート誘導体
US5668144A (en) * 1995-11-08 1997-09-16 American Home Products Corporation 1-azabicyclopheptane derivatives
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp 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
AU2002228015B2 (en) * 2000-12-22 2007-08-23 Almirall, S.A. Quinuclidine carbamate derivatives and their use as M3 antagonists
DE60216830T2 (de) * 2001-02-06 2007-06-14 Pfizer Products Inc., Groton Pharmazeutische Zusammensetzungen zur Behandlung von Störungen des ZNS oder anderen Erkrankungen
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.
AU2003246942B2 (en) * 2002-07-17 2009-06-25 Idorsia Pharmaceuticals Ltd Piperidinetriol derivatives as inhibitors of glycosylceramide synthase
WO2004011430A1 (ja) * 2002-07-25 2004-02-05 Yamanouchi Pharmaceutical Co., Ltd. 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
US7273872B2 (en) * 2002-12-06 2007-09-25 The Feinstein Institute For Medical Research Inhibition of inflammation using α 7 receptor-binding cholinergic agonists
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
WO2006002375A2 (en) * 2004-06-23 2006-01-05 The Feinstein Institute For Medical Research Method of treating ileus by pharmacological activation of cholinergic receptors
WO2006053043A2 (en) 2004-11-10 2006-05-18 Genzyme Corporation Methods of treating diabetes mellitus
EP1976519A2 (en) * 2006-01-27 2008-10-08 Yale University 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
JP2011505374A (ja) * 2007-11-29 2011-02-24 ザ・ホスピタル・フォー・シック・チルドレン リソソーム障害を処置するための組成物および方法
AU2008345225A1 (en) * 2007-12-21 2009-07-09 University Of Rochester Method for altering the lifespan of eukaryotic organisms
US8389517B2 (en) * 2008-07-28 2013-03-05 Genzyme Corporation Glucosylceramide synthase inhibition for the treatment of collapsing glomerulopathy and other glomerular disease
US20110237538A1 (en) * 2008-08-06 2011-09-29 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
US8821599B2 (en) * 2009-06-09 2014-09-02 Sundrop Fuels, Inc. Systems and methods for biomass gasifier reactor and receiver configuration
BR112012004377A2 (pt) 2009-08-28 2017-12-12 Genzyme Corp terapia de reposição enzimática com progressão de dose para tratamento de deficiência de esfingomielinase ácida.
LT3673906T (lt) * 2011-03-18 2025-08-11 Genzyme Corporation Gliukozilceramido sintazės inhibitoriai

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