JP2015509961A5 - - Google Patents

Download PDF

Info

Publication number
JP2015509961A5
JP2015509961A5 JP2014560086A JP2014560086A JP2015509961A5 JP 2015509961 A5 JP2015509961 A5 JP 2015509961A5 JP 2014560086 A JP2014560086 A JP 2014560086A JP 2014560086 A JP2014560086 A JP 2014560086A JP 2015509961 A5 JP2015509961 A5 JP 2015509961A5
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
subject
replacement therapy
gaucher disease
enzyme replacement
Prior art date
Legal status (The legal status 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 status listed.)
Granted
Application number
JP2014560086A
Other languages
English (en)
Japanese (ja)
Other versions
JP2015509961A (ja
JP6230160B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2013/028608 external-priority patent/WO2013130963A1/en
Publication of JP2015509961A publication Critical patent/JP2015509961A/ja
Publication of JP2015509961A5 publication Critical patent/JP2015509961A5/ja
Application granted granted Critical
Publication of JP6230160B2 publication Critical patent/JP6230160B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2014560086A 2012-03-02 2013-03-01 Iii型ゴーシェ病を治療するための組成物および方法 Active JP6230160B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261606089P 2012-03-02 2012-03-02
US61/606,089 2012-03-02
PCT/US2013/028608 WO2013130963A1 (en) 2012-03-02 2013-03-01 Compositions and methods for treating type iii gaucher disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2017150619A Division JP6475293B2 (ja) 2012-03-02 2017-08-03 Iii型ゴーシェ病を治療するための組成物および方法

Publications (3)

Publication Number Publication Date
JP2015509961A JP2015509961A (ja) 2015-04-02
JP2015509961A5 true JP2015509961A5 (enExample) 2016-04-21
JP6230160B2 JP6230160B2 (ja) 2017-11-15

Family

ID=49083333

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2014560086A Active JP6230160B2 (ja) 2012-03-02 2013-03-01 Iii型ゴーシェ病を治療するための組成物および方法
JP2017150619A Active JP6475293B2 (ja) 2012-03-02 2017-08-03 Iii型ゴーシェ病を治療するための組成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2017150619A Active JP6475293B2 (ja) 2012-03-02 2017-08-03 Iii型ゴーシェ病を治療するための組成物および方法

Country Status (10)

Country Link
US (1) US9623090B2 (enExample)
EP (1) EP2863941B1 (enExample)
JP (2) JP6230160B2 (enExample)
KR (2) KR20140138850A (enExample)
CN (1) CN104519905A (enExample)
AU (1) AU2013225816B2 (enExample)
CA (1) CA2865614A1 (enExample)
HK (2) HK1207005A1 (enExample)
MX (1) MX361231B (enExample)
WO (1) WO2013130963A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101658357B1 (ko) 2015-02-17 2016-09-21 한림대학교 산학협력단 레스베라트롤을 함유하는 ⅲ형 고셔병 치료용 약학 조성물
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
SG11202001544VA (en) 2017-08-24 2020-03-30 Sanofi Sa Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients
US11723579B2 (en) 2017-09-19 2023-08-15 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement
EA202091032A1 (ru) 2017-10-26 2020-07-17 Шайр Хьюман Дженетик Терапиз, Инк. Составы, содержащие глюкоцереброзидазу и изофагомин
US11717686B2 (en) 2017-12-04 2023-08-08 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to facilitate learning and performance
US12280219B2 (en) 2017-12-31 2025-04-22 NeuroLight, Inc. Method and apparatus for neuroenhancement to enhance emotional response
US11478603B2 (en) 2017-12-31 2022-10-25 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to enhance emotional response
US11364361B2 (en) 2018-04-20 2022-06-21 Neuroenhancement Lab, LLC System and method for inducing sleep by transplanting mental states
WO2020056418A1 (en) 2018-09-14 2020-03-19 Neuroenhancement Lab, LLC System and method of improving sleep
US20220125892A1 (en) * 2019-02-01 2022-04-28 OXYRANE UK Ltd. Glucocerebrosidase polypeptides
EP3959196A4 (en) 2019-04-25 2023-04-12 Takeda Pharmaceutical Company Limited ISOFAGOMIN SALTS, METHODS OF USE AND FORMULATIONS
KR102356047B1 (ko) * 2019-07-26 2022-02-07 재단법인 아산사회복지재단 효소대체요법 치료 비반응성 고셔병 치료용 약학조성물
US20230103407A1 (en) * 2020-03-10 2023-04-06 University Of Cincinnati Materials and methods for the treatment of gaucher disease

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4925796A (en) 1986-03-07 1990-05-15 Massachusetts Institute Of Technology Method for enhancing glycoprotein stability
US5272066A (en) 1986-03-07 1993-12-21 Massachusetts Institute Of Technology Synthetic method for enhancing glycoprotein stability
WO1989005850A1 (en) 1987-12-23 1989-06-29 The United States Of America, As Represented By Th Cloned dna for synthesizing unique glucocerebrosidase
DE68926569T2 (de) 1988-12-23 1996-09-26 Genzyme Corp Cho-zellen befähigt enzymatisch aktive rekombinante-glukocerebrosidase zu produzieren
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
US5401650A (en) 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
CA2099876C (en) 1991-01-21 2002-03-26 Michael L. Hayes Production of enzymatically active glucocerebrosidase from recombinant cells
US6270989B1 (en) 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
US5641670A (en) 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US5911983A (en) 1992-06-26 1999-06-15 University Of Pittsburgh Gene therapy for Gaucher disease using retroviral vectors
JPH08507677A (ja) 1992-12-10 1996-08-20 エンゾン,インコーポレーテッド 糖脂質酵素−ポリマー結合体
AU6809494A (en) 1992-12-21 1994-07-19 Enzon, Inc. Purification of proteinaceous material
DE69521074T2 (de) 1994-03-30 2001-09-13 Takara Shuzo Co., Ltd. Transglykosylierungsverfahren zur Herstellung eines Kohlenhydrats oder eines Glykokonjugates
JP3002113B2 (ja) 1994-03-30 2000-01-24 寳酒造株式会社 糖質又は複合糖質の製造方法
US5670132A (en) 1994-09-20 1997-09-23 Immunomedics, Inc. Modified radioantibody fragments for reduced renal uptake
AU693329B2 (en) 1995-04-13 1998-06-25 Corning Incorporated Dispersion managed optical waveguide
US5929304A (en) 1995-09-14 1999-07-27 Croptech Development Corporation Production of lysosomal enzymes in plant-based expression systems
JP2002504083A (ja) 1996-03-05 2002-02-05 オーケスト インコーポレイテッド ヒアルロン酸および増殖因子による骨の増殖を促進する方法
AU732552B2 (en) 1996-07-15 2001-04-26 Genzyme Corporation Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
DE69732129T2 (de) 1996-09-13 2005-12-08 Transkaryotic Therapies, Inc., Cambridge THERAPIE FÜR alpha GALACTOSIDASE A INSUFFIZIENZ
IL155588A0 (en) 2003-04-27 2003-11-23 Metabogal Ltd Methods for expression of enzymatically active recombinant lysosomal enzymes in transgenic plant root cells and vectors used thereby
EP0852951A1 (de) 1996-11-19 1998-07-15 Roche Diagnostics GmbH Stabile lyophilisierte pharmazeutische Zubereitungen von mono- oder polyklonalen Antikörpern
JPH10273500A (ja) 1997-03-03 1998-10-13 Noguchi Inst 複合糖ペプチド及びその製造法
JPH10306099A (ja) 1997-03-04 1998-11-17 Noguchi Inst 新規複合糖ペプチドおよびその製法
TW542721B (en) 1997-08-06 2003-07-21 Melaleuca Inc Dietary supplements containing natural ingredients
US6340746B1 (en) 1997-08-07 2002-01-22 University Of Utah Prodrugs and conjugates of thiol- and selenol- containing compounds and methods of use thereof
US5939279A (en) 1997-09-18 1999-08-17 The Board Of Trustees Of The University Of Arkansas Inhibition of bacterial binding by high-mannose oligosaccharides
CN1283997A (zh) 1997-12-22 2001-02-14 人类基因组科学公司 角质形成细胞生长因子-2制剂
GB9802249D0 (en) 1998-02-03 1998-04-01 Ciba Geigy Ag Organic compounds
GB9807464D0 (en) 1998-04-07 1998-06-10 Pharming Bv Purification of human acid µ-glucosidase
EP0976838A1 (en) 1998-05-06 2000-02-02 Rhone-Poulenc Nutrition Animale Enzymes mixture
JPH11318441A (ja) 1998-05-14 1999-11-24 Nagase & Co Ltd 超耐熱耐酸性アミロプルラナーゼ
KR19990086271A (ko) 1998-05-27 1999-12-15 손경식 면역세포의 신규한 엔도뉴클레아제 및 이를 사용한 면역보조제
KR20010052499A (ko) 1998-06-05 2001-06-25 아방티 파르마 소시에테 아노님 베타-시크리타제형 활성을 지닌 폴리펩타이드
ATE475714T1 (de) 1998-12-09 2010-08-15 Phyton Holdings Llc Verfahren zur herstellung von glycoprotein mit menschlichem glycosylierungsbild
GB9909066D0 (en) 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
AU5483600A (en) 1999-06-11 2001-01-02 Jean A. Chmielewski Pharmaceutical materials and methods for their preparation and use
ATE234626T1 (de) 1999-07-26 2003-04-15 Searle & Co Verwendung von langkettigen n-alkyl derivaten von deoxynojirimycin mit glucocerebrosidase enzyme zur herstellung eines medikaments zur behandlung von mit glykolipiden akkumulation zusammenhängenden krankheiten
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
IL150314A0 (en) 1999-12-30 2002-12-01 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
CN1156702C (zh) * 2001-07-11 2004-07-07 上海晶泰生物技术有限公司 采用标记链霉亲和素-生物素技术的蛋白质芯片
US20040202666A1 (en) 2003-01-24 2004-10-14 Immunomedics, Inc. Anti-cancer anthracycline drug-antibody conjugates
WO2004069190A2 (en) 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
US7951557B2 (en) 2003-04-27 2011-05-31 Protalix Ltd. Human lysosomal proteins from plant cell culture
US20060008415A1 (en) 2004-06-25 2006-01-12 Protein Design Labs, Inc. Stable liquid and lyophilized formulation of proteins
GB2438982A (en) 2004-12-22 2007-12-12 Ambrx Inc Compostions containing, methods involving, and uses of non-natural amino acids and polypeptides
JP5364382B2 (ja) 2006-02-07 2013-12-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 遊離チオール部分を有するタンパク質の安定化された組成物
US7713709B2 (en) 2006-03-17 2010-05-11 Biomarin Pharmaceutical Inc. Assay for detection of antibodies to lysosomal enzymes
PT1860101E (pt) 2006-05-24 2011-07-12 Amicus Therapeutics Inc Sal de tartrato da isofagomina e métodos de uso
JP5364362B2 (ja) 2008-12-24 2013-12-11 株式会社城南製作所 ワイヤドラム式のウインドウレギュレータ
PL3354277T3 (pl) * 2009-07-28 2021-12-13 Takeda Pharmaceutical Company Limited Kompozycje i sposoby leczenia choroby gauchera
WO2011107991A1 (en) 2010-03-02 2011-09-09 Protalix Ltd. Glucocerebrosidase multimers and uses thereof
WO2012012461A2 (en) 2010-07-19 2012-01-26 Shire Human Genetic Therapies, Inc. Mannose receptor c type 1 (mrc1) codon optimized cell line and uses thereof

Similar Documents

Publication Publication Date Title
JP2015509961A5 (enExample)
EP4600271A3 (en) Bioactive molecule conjugate, preparation method and use thereof
BR112014014262A2 (pt) nanopartículas; composição farmacêutica; método de administrar um ou mais agentes terapêuticos, profiláticos, e/ou diagnósticos a um paciente em necessidade dos mesmos e método de preparação das partículas.
JP2014528901A5 (enExample)
HK1200714A1 (en) Fgfr-binder-active agent conjugates
WO2015031698A8 (en) Site-specific antibody conjugation methods and compositions
FI20135152A7 (fi) Farmaseuttinen yhdistelmäkoostumus ja menetelmiä diabeteksen ja metabolisten sairauksien hoidossa
EA201401028A1 (ru) Фармацевтическая композиция, содержащая эмпаглифлозин и лекарственное средство от ожирения
JP2013503165A5 (enExample)
UY30450A1 (es) Aplicacinn de alta frecuencia de terapia con toxina botulenica
EA201270221A1 (ru) Лечение болезни крона с применением лаквинимода
JP2015523321A5 (enExample)
HK1199820A1 (en) Treatment of multiple sclerosis with combination of laquinimod and fingolimod
HK1198279A1 (en) Treatment of multiple sclerosis with combination of laquinimod and glatiramer acetate
EA201490378A1 (ru) ЛЕЧЕНИЕ РАССЕЯННОГО СКЛЕРОЗА С ИСПОЛЬЗОВАНИЕМ КОМБИНАЦИИ ЛАХИНИМОДА И ИНТЕРФЕРОНА-β
WO2011094467A3 (en) Methods of treating diabetes with dll4 antagonists
AU2016204185A1 (en) Methods of administering beta7 integrin antagonists
HK1218865A1 (zh) 用於降低多发性硬化症中丘脑损伤的拉喹莫德
MX358680B (es) Usos de inmunoconjugados dirigidos a cd138.
SG10201906172XA (en) Regulation of glucose metabolism using anti-cgrp antibodies
BR112013027010A2 (pt) método de produzir substâncias com gás supersaturado, dispositivo para sua entrega trasdérmica, e seus usos
MX370898B (es) Agente terapéutico y/o preventivo que comprende un derivado de 1-indansulfamida para el dolor.
HRP20230070T1 (hr) Tasimelteon za liječenje smith-magenis sindroma
EA201590191A1 (ru) Лечение рассеянного склероза комбинацией лаквинимода и фампридина
AU2012334804A8 (en) Modulators of C3a receptors