AU2013225816B2 - Compositions and methods for treating Type III Gaucher Disease - Google Patents

Compositions and methods for treating Type III Gaucher Disease Download PDF

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Publication number
AU2013225816B2
AU2013225816B2 AU2013225816A AU2013225816A AU2013225816B2 AU 2013225816 B2 AU2013225816 B2 AU 2013225816B2 AU 2013225816 A AU2013225816 A AU 2013225816A AU 2013225816 A AU2013225816 A AU 2013225816A AU 2013225816 B2 AU2013225816 B2 AU 2013225816B2
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velaglucerase
treatment
imiglucerase
patients
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AU2013225816A1 (en
AU2013225816A8 (en
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Kiran BHIRANGI
Gabriel Martin Cohn
Eric CROMBEZ
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Takeda Pharmaceutical Co Ltd
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Takeda Pharmaceutical Co Ltd
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    • 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
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    • A61P19/00—Drugs for skeletal disorders
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    • A—HUMAN NECESSITIES
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    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/536—Immunoassay; Biospecific binding assay; Materials therefor with immune complex formed in liquid phase
    • G01N33/537—Immunoassay; Biospecific binding assay; Materials therefor with immune complex formed in liquid phase with separation of immune complex from unbound antigen or antibody
    • G01N33/538—Immunoassay; Biospecific binding assay; Materials therefor with immune complex formed in liquid phase with separation of immune complex from unbound antigen or antibody by sorbent column, particles or resin strip, i.e. sorbent materials
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    • G01N33/564—Immunoassay; Biospecific binding assay; Materials therefor for pre-existing immune complex or autoimmune disease, i.e. systemic lupus erythematosus, rheumatoid arthritis, multiple sclerosis, rheumatoid factors or complement components C1-C9
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    • G01—MEASURING; TESTING
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    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/573—Immunoassay; Biospecific binding assay; Materials therefor for enzymes or isoenzymes
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6854—Immunoglobulins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/04—Endocrine or metabolic disorders
    • G01N2800/044—Hyperlipemia or hypolipemia, e.g. dyslipidaemia, obesity
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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AU2013225816A 2012-03-02 2013-03-01 Compositions and methods for treating Type III Gaucher Disease Active AU2013225816B2 (en)

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

Publications (3)

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AU2013225816A1 AU2013225816A1 (en) 2014-09-25
AU2013225816A8 AU2013225816A8 (en) 2015-06-11
AU2013225816B2 true AU2013225816B2 (en) 2017-10-26

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US (1) US9623090B2 (OSRAM)
EP (1) EP2863941B1 (OSRAM)
JP (2) JP6230160B2 (OSRAM)
KR (2) KR102096752B1 (OSRAM)
CN (1) CN104519905A (OSRAM)
AU (1) AU2013225816B2 (OSRAM)
CA (1) CA2865614A1 (OSRAM)
HK (2) HK1207005A1 (OSRAM)
MX (1) MX361231B (OSRAM)
WO (1) WO2013130963A1 (OSRAM)

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
BR112020003541A2 (pt) 2017-08-24 2020-09-01 Sanofi tratamento de condições ósseas anormais em pacientes com deficiência de esfingomielinase ácida
WO2019060298A1 (en) 2017-09-19 2019-03-28 Neuroenhancement Lab, LLC METHOD AND APPARATUS FOR NEURO-ACTIVATION
EP3700526A4 (en) * 2017-10-26 2021-09-08 Takeda Pharmaceutical Company Limited FORMULATIONS WITH GLUCOCEREBROSIDASE AND ISOFAGOMIN
US11717686B2 (en) 2017-12-04 2023-08-08 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to facilitate learning and performance
US11478603B2 (en) 2017-12-31 2022-10-25 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to enhance emotional response
US12280219B2 (en) 2017-12-31 2025-04-22 NeuroLight, Inc. 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
CN113382683A (zh) 2018-09-14 2021-09-10 纽罗因恒思蒙特实验有限责任公司 改善睡眠的系统和方法
US20220125892A1 (en) * 2019-02-01 2022-04-28 OXYRANE UK Ltd. Glucocerebrosidase polypeptides
US12286400B2 (en) 2019-04-25 2025-04-29 Takeda Pharmaceutical Company Limited Isofagomine salts, methods of use and formulations
KR102356047B1 (ko) * 2019-07-26 2022-02-07 재단법인 아산사회복지재단 효소대체요법 치료 비반응성 고셔병 치료용 약학조성물
EP4117628A4 (en) * 2020-03-10 2024-04-10 University of Cincinnati Materials and methods for the treatment of gaucher disease

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110027254A1 (en) * 2009-07-28 2011-02-03 Peter Francis Daniel Compositions and methods for treating gaucher disease

Family Cites Families (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5272066A (en) 1986-03-07 1993-12-21 Massachusetts Institute Of Technology Synthetic method for enhancing glycoprotein stability
US4925796A (en) 1986-03-07 1990-05-15 Massachusetts Institute Of Technology Method for enhancing glycoprotein stability
EP0393143A4 (en) 1987-12-23 1991-09-11 Us Commerce Cloned dna for synthesizing unique glucocerebrosidase
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
DE68926569T2 (de) 1988-12-23 1996-09-26 Genzyme Corp Cho-zellen befähigt enzymatisch aktive rekombinante-glukocerebrosidase zu produzieren
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
WO1992013067A1 (en) 1991-01-21 1992-08-06 Genzyme Corporation 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
AU5743994A (en) 1992-12-10 1994-07-04 Enzon, Inc. Glycolipid enzyme-polymer conjugates
AU6809494A (en) 1992-12-21 1994-07-19 Enzon, Inc. Purification of proteinaceous material
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
DE69521074T2 (de) 1994-03-30 2001-09-13 Takara Shuzo Co., Ltd. Transglykosylierungsverfahren zur Herstellung eines Kohlenhydrats oder eines Glykokonjugates
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
ATE426031T1 (de) 1995-09-14 2009-04-15 Virginia Tech Intell Prop Produktion von lysosomalen enzymen in pflanzlichen expressionssystemen
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
US6177447B1 (en) 1996-07-15 2001-01-23 Universiteit Van Amsterdam Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
PL190041B1 (pl) 1996-09-13 2005-10-31 Transkaryotic Therapies Zastosowanie ludzkiej komórki, cząsteczka DNA, konstrukt ekspresji, hodowana ludzka komórka, klonujący szczep komórek, klonująca linia komórek, białko, sposób wytwarzania glikozylowanej ludzkiej alfa-galaktozydazy A, oczyszczona glikozylowana ludzkaalfa-galaktozydaza A, sposób oczyszczania ludzkiej alfa-galaktozydazy A, zastosowanie oczyszczonej glikozylowanej ludzkiej alfa-galaktozydazy A, kompozycja terapeutyczna i sposób oczyszczania ludzkiej alfa-galaktozydazy A z próbki
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
DE69835308T2 (de) 1997-08-07 2007-08-09 University Of Utah Research Foundation, Salt Lake City Prodrugs und Konjugate von Selenoenthaltenden Verbindungen und ihre Verwendung
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
JP2001526239A (ja) 1997-12-22 2001-12-18 ヒューマン・ジェノム・サイエンシズ・インコーポレイテッド 角質細胞増殖因子−２製剤
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
AU4045599A (en) 1998-06-05 1999-12-30 Aventis Pharma S.A. Polypeptides with beta-secretase type activity
DK1137789T3 (da) 1998-12-09 2010-11-08 Phyton Holdings Llc Fremgangsmåde til fremstilling af et glycoprotein med glycosylering af human type
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
AU6050600A (en) 1999-07-26 2001-02-13 G.D. Searle & Co. Use of long-chain n-alkyl derivates of deoxynojirimycin and a glucocerebrosidaseenzyme for the manufacture of medicament for the treatment of glycolipid storag e diseases
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
AU2352201A (en) 1999-12-30 2001-07-16 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
EP2444102B1 (en) 2003-01-31 2015-04-08 The 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
US7385028B2 (en) 2004-12-22 2008-06-10 Ambrx, Inc Derivatization of non-natural amino acids and polypeptides
WO2007092829A2 (en) 2006-02-07 2007-08-16 Shire Human Genetic Therapies, Inc. Stabilized compositions of proteins having a free thiol moiety
JP2009529885A (ja) 2006-03-17 2009-08-27 バイオマリン ファーマシューティカル インコーポレイテッド リソソーム酵素に対する抗体の検出のためのアッセイ
DK1860101T3 (da) 2006-05-24 2011-07-25 Amicus Therapeutics Inc Tartrat salt af isofagomin og fremgangsmåder til anvendelse deraf
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
US20130017169A1 (en) * 2010-03-02 2013-01-17 Protalix Ltd. Multimeric forms of therapeutic proteins and uses thereof
US9453847B2 (en) * 2010-07-19 2016-09-27 Shire Human Genetic Therapies, Inc. Mannose receptor C type 1 (MRC1) codon optimized cell line and uses thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110027254A1 (en) * 2009-07-28 2011-02-03 Peter Francis Daniel Compositions and methods for treating gaucher disease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
KRAOUA, I. et al Brain and Development, Vol. 33, page 131-139. Published online March 22 2010. *

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