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PE20140412A1 - Inhibidores de glucosilceramida sintasa - Google Patents

Inhibidores de glucosilceramida sintasa

Info

Publication number
PE20140412A1
PE20140412A1 PE2013002075A PE2013002075A PE20140412A1 PE 20140412 A1 PE20140412 A1 PE 20140412A1 PE 2013002075 A PE2013002075 A PE 2013002075A PE 2013002075 A PE2013002075 A PE 2013002075A PE 20140412 A1 PE20140412 A1 PE 20140412A1
Authority
PE
Peru
Prior art keywords
aryl
alkyl
glucosylceramide synthase
compounds
azabicycle
Prior art date
Application number
PE2013002075A
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 PE20140412A1 publication Critical patent/PE20140412A1/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
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    • 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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    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P35/00—Antineoplastic agents
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    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • 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
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    • 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
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    • 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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Abstract

SE REFIERE A COMPUESTOS HETEROCICLICOS DE FORMULA (I) DONDE n ES DE 1 A 3; m Y p SON CADA UNO 0 O 1; t Y z SON CADA UNO DE 0 A 2; y ES 1 O 2; E ES S, O, NH, NOH, NCN, ENTRE OTROS; X1 ES CR1 O N, EN DONDE R1 ES H, CN, ALQUILCARBONILO(C1-C6) O ALQUILO(C1-C6); X2 ES O, NH, CH2, SO2, ENTRE OTROS; X3 ES O, NH, CH2, CO, ENTRE OTROS; X4 ES CR4R5, CH2CR4R5 O CH2-ALQUIL(C1-C6)-CR4R5, EN DONDE R4 Y R5 SON CADA UNO H, ALQUILO(C1-C6), ENTRE OTROS; X5 ES UN ENLACE DIRECTO, O, S, SO2, ALQUILOXI(C1-C6), ENTRE OTROS; R6 ES H, HALOGENO, CN, ARILO(C6-C12), ENTRE OTROS; A1 ES ALQUINILO(C2-C6), CICLOALQUILO(C3-C10), ARILO(C6-C12), ENTRE OTROS; A2 ES H, CICLOALQUILO(C3-C10), ARILO(C6-C12), ENTRE OTROS. SON COMPUESTOS PREFERIDOS: [2-(2,4'-DIFLUOROBIFENIL-4-IL)PROPAN-2-IL]CARBAMATO DE 1-AZABICICLO[2,2,2]OCT-3-ILO; {2-[4-(1,3-BENZOTIAZOL-6-IL)FENIL]PROPAN-2-IL}CARBAMATO DE 1-AZABICICLO[2,2,2]OCT-3-ILO; ENTRE OTROS. DICHOS COMPUESTOS SON INHIBIDORES DE GLUCOSILCERAMIDA SINTASA (GCS) SIENDO UTILES EN EL TRATAMIENTO DE ENFERMEDADES DE ALMACENAMIENTO LISOSOMICO, CANCER
PE2013002075A 2011-03-18 2012-03-16 Inhibidores de glucosilceramida sintasa PE20140412A1 (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
PE20140412A1 true PE20140412A1 (es) 2014-04-01

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Family Applications (1)

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PE2013002075A PE20140412A1 (es) 2011-03-18 2012-03-16 Inhibidores de glucosilceramida sintasa

Country Status (38)

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US (5) US9139580B2 (es)
EP (3) EP3673906B1 (es)
JP (2) JP6117708B2 (es)
KR (1) KR101987736B1 (es)
CN (2) CN107080747B (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) HRP20250907T1 (es)
HU (2) HUE047888T2 (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) PL2685986T3 (es)
PT (2) PT2685986T (es)
RS (2) RS59963B1 (es)
RU (2) RU2018104472A (es)
SI (2) SI2685986T1 (es)
TW (2) TWI546301B (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
RU2018104472A (ru) * 2011-03-18 2019-02-22 Джензим Корпорейшн Ингибиторы глюкозилцерамид-синтазы
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
CA2906581C (en) * 2013-03-15 2022-09-20 Genzyme Corporation A method of preparing glucosylceramide synthase quinuclidine carbamate inhibitors, and thiazole intermediates
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
CN105979945A (zh) * 2013-12-11 2016-09-28 建新公司 葡糖神经酰胺合酶抑制剂
EP3148518B1 (en) * 2014-05-30 2022-11-30 Sphaera Pharma Pvt. Ltd. Novel 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
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
EP3317265A4 (en) * 2015-07-01 2019-04-17 Northwestern University SUBSTITUTED CHINAZOLIN 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
WO2017165766A2 (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
EP3538095A4 (en) * 2016-11-14 2020-06-17 Berg LLC Methods for treating parkinson's disease
WO2018122746A1 (en) 2016-12-28 2018-07-05 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
JP7171057B2 (ja) 2016-12-29 2022-11-15 ミノリックス セラピューティクス エセ．エレ． ヘテロアリール化合物およびその使用
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
US11498904B2 (en) * 2017-11-14 2022-11-15 Merck Sharp & Dohme Llc 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
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
KR20210123352A (ko) * 2019-02-04 2021-10-13 젠자임 코포레이션 리소좀 축적 질환과 연관된 증상 및 장애를 치료하기 위한 방법
AU2020218185B2 (en) 2019-02-04 2025-01-23 Genzyme Corporation Treatment of ciliopathies using inhibitors of glucosylceramide synthase (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
JP7539382B2 (ja) * 2019-07-29 2024-08-23 武田薬品工業株式会社 複素環化合物
EP4048397B1 (en) 2019-10-23 2023-08-16 The Regents Of The University Of Michigan Glucosylceramide synthase inhibitors for therapeutic applications
RS65662B1 (sr) 2019-11-15 2024-07-31 Yuhan Corp Derivati koji imaju 2,3-dihidro-1h-inden ili 2,3-dihidrobenzofuranski deo ili njihovu farmaceutski prihvatljivu so i farmaceutske kompozicije koje ih sadrže
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
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
JP7691753B2 (ja) 2019-11-25 2025-06-12 ジーティー ゲイン セラピューティクス エスアー ベータ－グルコ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ヘテロアリール化合物およびその治療的使用
US20230140216A1 (en) 2020-02-03 2023-05-04 Gain Therapeutics Sa Combination therapy for treating mps1
AU2021215396A1 (en) 2020-02-03 2022-09-29 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
US11857512B2 (en) 2020-07-24 2024-01-02 Genzyme Corporation Pharmaceutical compositions comprising venglustat
WO2022024062A1 (en) * 2020-07-30 2022-02-03 Genzyme Corporation Methods for reducing glycosphingolipid concentration in brain tissue and methods of treatment of neurodegenerative diseases involving the same
CA3216293A1 (en) * 2021-05-11 2022-11-17 Dong-Hoon Kim Novel compounds having inhibitory activity against glucosylceramide synthase or pharmaceutically acceptable salt thereof, processes for preparing the same, and pharmaceutical compositions comprising the sam
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
AU2023368360A1 (en) * 2022-10-24 2025-03-20 Green Cross Corporation Novel salt of dimethyl-2,3-dihydro-1h-indene derivative and processes for preparing the same
CA3268123A1 (en) * 2022-10-24 2024-05-02 Yuhan Corporation IMPROVED PROCESSES FOR PREPARING DIMETHYL-2,3-DIHYDRO-1H-INDENE DERIVATIVES
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
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
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
KR970701174A (ko) 1994-02-10 1997-03-17 오노다 마사요시 신규한 카바메이트 유도체 및 이의 의약 조성물(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
AU7551896A (en) * 1995-11-08 1997-05-29 American Home Products Corporation 1-azabicycloheptane derivatives and their pharmaceutical use
CA2305768A1 (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
HUP0303529A3 (en) * 2000-12-22 2008-03-28 Almirall Ag New quinuclidine carbamate derivatives, process for their preparation and pharmaceutical compositions containing them and their use
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.
JP4575156B2 (ja) * 2002-07-17 2010-11-04 アクテリオン ファーマシューティカルズ リミテッド グルコシルセラミドシンターゼの阻害剤としてのピペリジントリオール誘導体
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
PT1949901E (pt) * 2002-12-06 2014-05-23 The Feinstein Inst Medical Res Método para a determinação de um agonista colinérgico selectivo para um receptor nicotínico alfa 7
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
CA2571584A1 (en) * 2004-06-23 2006-01-05 The Feinstein Institute For Medical Research Method of treating ileus by pharmacological activation of cholinergic receptors
US8003617B2 (en) 2004-11-10 2011-08-23 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
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
EP2320886B1 (en) * 2008-07-28 2017-06-28 Genzyme Corporation Glucosylceramide synthase inhibition for the treatment of collapsing glomerulopathy and other glomerular disease
EP2323652A2 (en) * 2008-08-06 2011-05-25 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
WO2010091164A1 (en) 2009-02-06 2010-08-12 Exelixis, Inc. Inhibitors of glucosylceramide synthase
WO2010091104A1 (en) * 2009-02-06 2010-08-12 Exelixis, Inc. Glucosylceramide synthase inhibitors
US9150803B2 (en) * 2009-06-09 2015-10-06 Sundrop Fuels, Inc. Systems and methods for biomass grinding and feeding
FI3998078T3 (fi) 2009-08-28 2025-12-04 Icahn School Med Mount Sinai Annoksensuurentamisentsyymikorvaushoito happaman sfingomyelinaasin puutoksen hoitamista varten
RU2018104472A (ru) * 2011-03-18 2019-02-22 Джензим Корпорейшн Ингибиторы глюкозилцерамид-синтазы

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