JP2022519131A5 - - Google Patents

Info

Publication number
JP2022519131A5
JP2022519131A5 JP2021545384A JP2021545384A JP2022519131A5 JP 2022519131 A5 JP2022519131 A5 JP 2022519131A5 JP 2021545384 A JP2021545384 A JP 2021545384A JP 2021545384 A JP2021545384 A JP 2021545384A JP 2022519131 A5 JP2022519131 A5 JP 2022519131A5
Authority
JP
Japan
Application number
JP2021545384A
Other languages
Japanese (ja)
Other versions
JPWO2020163337A5 (https=
JP7633169B2 (ja
JP2022519131A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/016588 external-priority patent/WO2020163337A1/en
Publication of JP2022519131A publication Critical patent/JP2022519131A/ja
Publication of JP2022519131A5 publication Critical patent/JP2022519131A5/ja
Publication of JPWO2020163337A5 publication Critical patent/JPWO2020163337A5/ja
Application granted granted Critical
Publication of JP7633169B2 publication Critical patent/JP7633169B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021545384A 2019-02-04 2020-02-04 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 Active JP7633169B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962800993P 2019-02-04 2019-02-04
US62/800,993 2019-02-04
US201962851430P 2019-05-22 2019-05-22
US62/851,430 2019-05-22
PCT/US2020/016588 WO2020163337A1 (en) 2019-02-04 2020-02-04 Treatment of ciliopathies using inhibitors of glucosylceramide synthase (gcs)

Publications (4)

Publication Number Publication Date
JP2022519131A JP2022519131A (ja) 2022-03-18
JP2022519131A5 true JP2022519131A5 (https=) 2023-02-10
JPWO2020163337A5 JPWO2020163337A5 (https=) 2023-02-10
JP7633169B2 JP7633169B2 (ja) 2025-02-19

Family

ID=69740838

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021545384A Active JP7633169B2 (ja) 2019-02-04 2020-02-04 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

Country Status (10)

Country Link
US (1) US12527776B2 (https=)
EP (1) EP3920912B1 (https=)
JP (1) JP7633169B2 (https=)
CN (1) CN114040762A (https=)
AU (1) AU2020218185B2 (https=)
ES (1) ES3040555T3 (https=)
HU (1) HUE072626T2 (https=)
PL (1) PL3920912T3 (https=)
PT (1) PT3920912T (https=)
WO (1) WO2020163337A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA37975B2 (fr) 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
CN114040762A (zh) 2019-02-04 2022-02-11 建新公司 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病
TW202142236A (zh) 2020-02-03 2021-11-16 美商健臻公司 用於治療與溶體儲積症相關的神經症狀之方法
KR20230043024A (ko) 2020-07-24 2023-03-30 젠자임 코포레이션 벤글루스타트를 포함하는 제약 조성물
WO2022169004A1 (ko) * 2021-02-05 2022-08-11 연세대학교 산학협력단 신규한 fabp4 억제제를 유효성분으로 포함하는 섬모형성 촉진용 조성물
EP4201403A1 (en) 2021-12-21 2023-06-28 Som Innovation Biotech, S.L. Compounds tirapazamine and quazinone for use in the treatment of gm2 gangliosidoses
CN115350278A (zh) * 2022-07-11 2022-11-18 清华大学 提高细胞内鞘脂类物质含量的试剂在制备纤毛异常相关疾病药物中的用途
CN120417900A (zh) 2022-12-01 2025-08-01 建新公司 文鲁司他与cyp3a4的强或中度抑制剂的组合
WO2025178019A1 (ja) * 2024-02-19 2025-08-28 国立大学法人大阪大学 繊毛病治療用医薬組成物
WO2025262570A1 (en) 2024-06-17 2025-12-26 Genzyme Corporation Venglustat as an inhibitor of glucosylceramide synthase in subjects having hepatic impairment

Family Cites Families (98)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB725228A (en) 1952-06-19 1955-03-02 Roche Products Ltd Substituted carbamic acid esters and process for the manufacture thereof
BE640616A (https=) 1962-12-19
NL130759C (https=) 1965-10-07
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
US4593034A (en) 1984-04-06 1986-06-03 A. H. Robins Company, Inc. 2-alkoxy-N-(1-azabicyclo[2.2.2]oct-3-yl)benzamides and thiobenzamides
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
IE62231B1 (en) 1987-12-24 1995-01-11 Wyeth John & Brother Ltd Heterocyclic compounds
DE3878235T2 (de) 1988-08-04 1993-06-03 Synthelabo Antischizophren-wirksame s-n-(1-azabicyclo (2.2.2.)oct-3-yl)benzamide und thiobenzamide.
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.
DK443489D0 (da) 1989-09-08 1989-09-08 Ferrosan As Substituerede urinstofforbindelser, deres fremstilling og anvendelse
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
GB9106571D0 (en) 1991-03-27 1991-05-15 Erba Carlo Spa Derivatives of substituted imidazol-2-one and process for their preparation
US5733761A (en) 1991-11-05 1998-03-31 Transkaryotic Therapies, Inc. Protein production and protein delivery
DE4326510A1 (de) 1993-08-06 1995-02-09 Bayer Ag Verfahren zur Herstellung von Cyclohexen-Derivaten
WO1995021820A1 (en) 1994-02-10 1995-08-17 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
SE9600683D0 (sv) 1996-02-23 1996-02-23 Astra Ab Azabicyclic esters of carbamic acids useful in therapy
SK4699A3 (en) 1996-07-29 2000-04-10 Pharmacia & Upjohn Ab Arylcycloalkane carboxylic esters, their use, pharmaceutical compositions and preparation
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
US5985836A (en) 1998-07-31 1999-11-16 Bayer Corporation Alpha-1 proteinase inhibitor binding peptides
CN1332726A (zh) 1998-11-02 2002-01-23 卫福有限公司 吡咯烷化合物及其药物用途
US6953855B2 (en) 1998-12-11 2005-10-11 Targacept, Inc. 3-substituted-2(arylalkyl)-1-azabicycloalkanes and methods of use thereof
JP2002540203A (ja) 1999-03-29 2002-11-26 ニューロゲン コーポレイション Nk−3および/またはgaba(a)受容体リガンドとしての4−置換キノリン誘導体
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
JP2003521543A (ja) 2000-02-07 2003-07-15 アボット ゲーエムベーハー ウント カンパニー カーゲー 2−ベンゾチアゾリル尿素誘導体およびそのプロテインキナーゼ阻害剤としての使用
GB0010955D0 (en) 2000-05-05 2000-06-28 Novartis Ag Organic compounds
CZ294251B6 (cs) 2000-06-27 2004-11-10 Laboratorios S. A. L. V. A. T., S. A. Karbamáty a jejich použití pro výrobu farmaceutického prostředku
KR100827937B1 (ko) 2000-07-19 2008-05-09 유니티카 가부시끼가이샤 스핑고당지질을 함유하는 생리학적 기능성 식품 또는화장품 및 이들의 제조방법
US6492386B2 (en) 2000-08-18 2002-12-10 Pharmacia & Upjohn Company Quinuclidine-substituted aryl compounds for treatment of disease
US6599916B2 (en) 2000-08-21 2003-07-29 Pharmacia & Upjohn Company Quinuclidine-substituted heteroaryl moieties for treatment of disease
JP4445704B2 (ja) 2000-12-22 2010-04-07 アルミラル・ソシエダッド・アノニマ キヌクリジンカルバメート誘導体およびm3アンダゴニストとしてのそれらの使用
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
DE10211415A1 (de) 2002-03-15 2003-09-25 Bayer Ag Bicyclische N-Biarylamide
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
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
SE0202430D0 (sv) 2002-08-14 2002-08-14 Astrazeneca Ab New Compounds
WO2004052365A2 (en) 2002-12-06 2004-06-24 North Shore-Long Island Jewish Research Institute Inhibition of inflamation using alpha 7 receptor-binding cholinergic agonists
WO2004056744A1 (en) 2002-12-23 2004-07-08 Janssen Pharmaceutica N.V. Adamantyl acetamides as hydroxysteroid dehydrogenase inhibitors
US20050031683A1 (en) 2003-08-04 2005-02-10 Ashish Kapoor Solid pharmaceutical composition
TW200529860A (en) 2003-12-22 2005-09-16 Astrazeneca Ab Nicotinic acetylcholine receptor ligands
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
US8003617B2 (en) 2004-11-10 2011-08-23 Genzyme Corporation Methods of treating diabetes mellitus
WO2006134318A1 (en) 2005-06-11 2006-12-21 Vernalis R & D Limited Pyrazole-substituted benzimidazole derivatives for use in the treatment of cancer and autoimmune disorders
AU2006295397A1 (en) 2005-09-23 2007-04-05 Memory Pharmaceuticals Corporation Indazoles, benzothiazoles, benzoisothiazoles, benzisoxazoles, pyrazolopyridines, isothiazolopyridines, and preparation and uses thereof
SG131819A1 (en) 2005-11-07 2007-05-28 Chikka Pte Ltd Buddy-based cross-carrier messaging system
US20090170847A1 (en) 2006-01-23 2009-07-02 Seung Chul Lee Imidazopyridine Derivatives Inhibiting Protein Kinase Activity, Method for the Preparation Thereof and Pharmaceutical Composition Containing Same
WO2007100430A2 (en) 2006-01-27 2007-09-07 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
KR20090107567A (ko) 2007-02-09 2009-10-13 아스텔라스세이야쿠 가부시키가이샤 아자 가교환 화합물
CN101627014B (zh) 2007-03-09 2013-04-24 株式会社绿十字 用于预防或治疗帕金森病的(+)-3-羟基吗啡喃的口服可生物利用前药
AU2008232614A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of Fabry disease using pharmacological chaperones
EP2546232A1 (en) 2007-06-20 2013-01-16 Merck Sharp & Dohme Corp. Diphenyl Substituted Alkanes
BRPI0822349A2 (pt) 2007-11-29 2019-09-24 Neuraltus Pharmaceuticals Inc composições e métodos para tratar doenças lisossômicas
CA2731685A1 (en) 2008-07-28 2010-02-04 Genzyme Corporation Glucosylceramide synthase inhibition for the treatment of collapsing glomerulopathy and other glomerular disease
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
WO2010121963A1 (en) 2009-04-21 2010-10-28 Nerviano Medical Sciences S.R.L. Resorcinol derivatives as hsp90 inhibitors
AR077468A1 (es) 2009-07-09 2011-08-31 Array Biopharma Inc Compuestos de pirazolo (1,5 -a) pirimidina sustituidos como inhibidores de trk- quinasa
CN102573842A (zh) 2009-07-23 2012-07-11 诺瓦提斯公司 氮杂双环烷基衍生物或吡咯烷-2-酮衍生物的用途
SI3998078T1 (sl) 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
US20110183938A1 (en) 2009-12-16 2011-07-28 Genentech, Inc. 1,7-diazacarbazoles and methods of use
PT2568970T (pt) 2010-05-10 2019-02-11 Lesvi Laboratorios Sl Formulações farmacêuticas estáveis contendo um anti-histamínico
RU2436782C1 (ru) 2010-06-10 2011-12-20 Новосибирский институт органической химии им. Н.Н. Ворожцова Сибирского отделения Российской академии наук (НИОХ СО РАН) 2-МЕТОКСИ-4[(3aR,7aS)-3,3,6-ТРИМЕТИЛ-1,3,3a,4,5,7a-ГЕКСАГИДРО-2-БЕНЗОФУРАН-1-ИЛ]ФЕНОЛ - НОВОЕ СРЕДСТВО ДЛЯ ЛЕЧЕНИЯ БОЛЕЗНИ ПАРКИНСОНА
RU2607084C2 (ru) 2010-11-12 2017-01-10 Сионоги Энд Ко., Лтд. Кристаллы производных 6,7-ненасыщенного-7-карбамоилморфинана и способ их получения
AU2012231275A1 (en) 2011-03-18 2013-10-17 Genzyme Corporation Glucosylceramide synthase inhibitors
WO2012177997A1 (en) 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies
US20140228575A1 (en) 2011-06-22 2014-08-14 Isochem Process for the Preparation of Solifenacin and Salts Thereof
HK1207006A1 (en) 2012-09-11 2016-01-22 Hospira Australia Pty Ltd Daptomycin formulations and uses thereof
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
TWI649317B (zh) 2013-03-15 2019-02-01 健臻公司 製備葡糖基神經醯胺合成酶抑制劑之方法
PT3079695T (pt) 2013-12-11 2021-05-04 Genzyme Corp Inibidores de glicosilceramida-sintase
TW201642855A (zh) 2015-03-10 2016-12-16 健臻公司 用於治療蛋白質病變之方法
EA201891170A1 (ru) * 2015-11-18 2018-11-30 Джензим Корпорейшн Биомаркер поликистозной болезни почек и варианты его применения
EP3412296B1 (en) 2016-02-05 2025-11-12 Kyungpook National University Industry-Academic Cooperation Foundation Pharmaceutical composition containing stem cell in which vascular endothelial growth factor is overexpressed as effective ingredient for preventing or treating neurodegenerative disease
EP3452045A1 (en) 2016-05-04 2019-03-13 Lysosomal Therapeutics Inc. Methods of treatment and combination therapies using gcase activator heterobicyclic and related compounds
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
US10588888B2 (en) 2017-07-17 2020-03-17 Children's Hospital Medical Center CNS-accessible pharmacological chaperones for treatment of acid β-glucosidase-related disease states
US12472144B2 (en) 2017-08-08 2025-11-18 Amneal Pharmaceuticals Llc Pharmaceutical composition comprising eliglustat
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
EP3920924A4 (en) 2019-02-04 2022-11-16 Prilenia Neurotherapeutics Ltd. LOW-DOSE PRIDOIDIN FOR THE TREATMENT OF PARKINSON'S DISEASE AND OTHER PARKINSONIC-RELATED DISEASES
CN114040762A (zh) 2019-02-04 2022-02-11 建新公司 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病
AU2020218496B2 (en) 2019-02-04 2024-12-19 Genzyme Corporation Methods for treating symptoms and disorders associated with lysosomal storage diseases
EP4034256B1 (en) 2019-09-25 2025-11-19 Assia Chemical Industries Ltd. Solid state forms of lucerastat salts and process for preparation thereof
TW202142236A (zh) 2020-02-03 2021-11-16 美商健臻公司 用於治療與溶體儲積症相關的神經症狀之方法
KR20230043024A (ko) 2020-07-24 2023-03-30 젠자임 코포레이션 벤글루스타트를 포함하는 제약 조성물
EP4188381A1 (en) 2020-07-30 2023-06-07 Genzyme Corporation Methods for reducing glycosphingolipid concentration in brain tissue and methods of treatment of neurodegenerative diseases involving the same

Similar Documents

Publication Publication Date Title
BR112023005462A2 (https=)
BR112023012656A2 (https=)
JP2022519131A5 (https=)
BR112021014123A2 (https=)
BR112023009656A2 (https=)
BR112022009896A2 (https=)
BR112021017747A2 (https=)
BR112022024743A2 (https=)
BR112022026905A2 (https=)
BR112023011738A2 (https=)
BR112023004146A2 (https=)
BR112023006729A2 (https=)
BR102021018859A2 (https=)
BR102021015500A2 (https=)
BR102021007058A2 (https=)
BR102020022030A2 (https=)
BR112023016292A2 (https=)
BR112023011539A2 (https=)
BR112023011610A2 (https=)
BR112023008976A2 (https=)
BR102021020147A2 (https=)
BR102021018926A2 (https=)
BR102021018167A2 (https=)
BR102021017576A2 (https=)
BR102021016837A2 (https=)