BRPI0507440A - fabricação de enzimas lisossomais altamente fosforiladas e uso das mesmas - Google Patents

fabricação de enzimas lisossomais altamente fosforiladas e uso das mesmas

Info

Publication number
BRPI0507440A
BRPI0507440A BRPI0507440-1A BRPI0507440A BRPI0507440A BR PI0507440 A BRPI0507440 A BR PI0507440A BR PI0507440 A BRPI0507440 A BR PI0507440A BR PI0507440 A BRPI0507440 A BR PI0507440A
Authority
BR
Brazil
Prior art keywords
manufacture
highly phosphorylated
lysosomal enzymes
phosphorylated lysosomal
compositions
Prior art date
Application number
BRPI0507440-1A
Other languages
English (en)
Inventor
Todd Zankel
Emil D Kakkis
Original Assignee
Biomarin Pharm Inc
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 Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of BRPI0507440A publication Critical patent/BRPI0507440A/pt

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/005—Glycopeptides, glycoproteins
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A61P3/06—Antihyperlipidemics
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2434—Glucanases acting on beta-1,4-glucosidic bonds
    • 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)
    • C12Y302/01003—Glucan 1,4-alpha-glucosidase (3.2.1.3), i.e. glucoamylase
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

FABRICAçãO DE ENZIMAS LISOSSOMAIS ALTAMENTE FOSFORILADAS E USO DAS MESMAS Esta invenção fornece composições de enzimas lisossomais altamente fosforiladas, suas composições farmacêuticas, métodos de produzir e purificar tais compostos e composições e seus usos no diagnóestico, profilaxia ou tratamento de doenças e condições, incluindo particularmente doenças de armazenamento lisossomal.
BRPI0507440-1A 2004-02-06 2005-02-07 fabricação de enzimas lisossomais altamente fosforiladas e uso das mesmas BRPI0507440A (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US54258604P 2004-02-06 2004-02-06
PCT/US2005/004345 WO2005077093A2 (en) 2004-02-06 2005-02-07 Manufacture of highly phosphorylated lysosomal enzymes and uses thereof

Publications (1)

Publication Number Publication Date
BRPI0507440A true BRPI0507440A (pt) 2007-07-10

Family

ID=34860323

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0507440-1A BRPI0507440A (pt) 2004-02-06 2005-02-07 fabricação de enzimas lisossomais altamente fosforiladas e uso das mesmas

Country Status (7)

Country Link
US (2) US20080014188A1 (pt)
EP (1) EP1720405A4 (pt)
JP (1) JP2007523648A (pt)
AU (1) AU2005211775B2 (pt)
BR (1) BRPI0507440A (pt)
CA (1) CA2556245A1 (pt)
WO (1) WO2005077093A2 (pt)

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AU2003222568B2 (en) 2002-01-11 2009-05-07 Bioasis Technologies, Inc. Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
HUE033381T2 (en) 2003-01-31 2017-11-28 Mount Sinai School Of Medicine Of New York Univ Combination therapy for the treatment of protein deficiency disorders
EP1877099B1 (en) 2005-04-06 2012-09-19 Genzyme Corporation Therapeutic conjugates comprising a lysosomal enzyme, polysialic acid and a targeting moiety
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
CN101631794B (zh) 2007-01-18 2013-01-02 建新公司 包含氨基氧基基团的寡糖及其轭合物
AU2008290217B2 (en) 2007-08-20 2013-09-05 Protalix Ltd. Saccharide-containing protein conjugates and uses thereof
US7722865B2 (en) 2008-01-18 2010-05-25 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
LT2245145T (lt) 2008-01-18 2017-04-10 Biomarin Pharmaceutical Inc. Aktyvių gausiai fosforilintų žmogaus lizosominės sulfatazės fermentų gamyba ir jų panaudojimas
WO2009131698A2 (en) * 2008-04-23 2009-10-29 Iowa State University Research Foundation, Inc. PHOSPHORYLATED RECOMBINANT N-ACETYL-alpha-D- GLUCOSAMINIDASE (NaGlu) AND USES THEREOF
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
BR122020010601B8 (pt) 2008-12-16 2021-07-27 Genzyme Corp conjugados de proteína-oligossacarídeo, seus usos, e composições farmacêuticas
DK3395372T3 (da) * 2009-02-20 2022-04-19 Enhanx Biopharm Inc System til afgivelse af glutathion-baseret medikament
WO2010117957A2 (en) 2009-04-06 2010-10-14 Mayo Foundation For Medical Education And Research Methods and materials for delivering molecules
ES2569514T3 (es) 2009-06-17 2016-05-11 Biomarin Pharmaceutical Inc. Formulaciones para enzimas lisosómicas
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
PL2538968T3 (pl) 2010-02-24 2018-04-30 Chiesi Farmaceutici S.P.A. Sposób wytwarzania i oczyszczania rekombinowanej lizosomalnej alfamannozydazy
WO2011108451A1 (ja) * 2010-03-01 2011-09-09 日本ケミカルリサーチ株式会社 遺伝子ノックアウト細胞を用いた組換え体リソソーム酵素の製造方法
WO2011163651A2 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. METHODS AND COMPOSITIONS FOR CNS DELIVERY OF β-GALACTOCEREBROSIDASE
RU2626514C2 (ru) 2010-06-25 2017-07-28 Шир Хьюман Дженетик Терапис, Инк. Доставка к цнс лечебных агентов
WO2011163649A2 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc Methods and compositions for cns delivery of iduronate-2-sulfatase
PT3626258T (pt) 2010-06-25 2021-10-19 Shire Human Genetic Therapies Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase
PL3626257T3 (pl) 2010-06-25 2022-03-28 Shire Human Genetic Therapies, Inc. Sposoby i kompozycje do dostarczania arylosulfatazy a do oun
US9320711B2 (en) 2010-06-25 2016-04-26 Shire Human Genetic Therapies, Inc. Methods and compositions for CNS delivery of heparan N-sulfatase
US8956859B1 (en) 2010-08-13 2015-02-17 Aviex Technologies Llc Compositions and methods for determining successful immunization by one or more vaccines
LT3272861T (lt) 2011-01-20 2020-03-25 Protalix Ltd. Alfa-galaktozidazės kompozicijos
US9138465B2 (en) 2011-04-28 2015-09-22 Osaka University Pharmaceutical composition for treating lysosomal storage disease
TW201307563A (zh) * 2011-05-19 2013-02-16 Shire Human Genetic Therapies 純化乙醯肝素－ｎ－硫酸酯酶之方法
JP5959114B2 (ja) * 2011-05-19 2016-08-02 学校法人 明治薬科大学 リン酸化糖鎖を含有する組換えヒトサポシンｂタンパク質及びその用途
US9387236B2 (en) * 2011-06-10 2016-07-12 Prothera Inc. Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases
PL2717917T3 (pl) 2011-07-05 2016-12-30 Koniugaty p97 - przeciwciało
DK2739649T3 (en) 2011-08-05 2018-01-08 Bioasis Technologies Inc P97 FRAGMENTS WITH TRANSFER ACTIVITY
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
CA2866683A1 (en) 2012-03-07 2013-09-12 Amicus Therapeutics, Inc. High concentration alpha-glucosidase compositions for the treatment of pompe disease
PL2844279T3 (pl) 2012-05-03 2021-08-16 Amicus Therapeutics, Inc. Schematy dawkowania w leczeniu choroby pompego
JP6433424B2 (ja) 2012-07-31 2018-12-05 バイオアシス テクノロジーズ インコーポレイテッド 脱リン酸化されたリソソーム蓄積症タンパク質およびその使用方法
US20150313971A1 (en) * 2012-12-07 2015-11-05 Shire Human Genetic Therapies Methods and compositions for intrathecally administered treatment of mucupolysaccharidosis type iiia
BR112015022416A2 (pt) 2013-03-13 2017-10-24 Bioasis Technologies Inc fragmentos de p97 e seus usos
US20160347821A1 (en) 2014-02-03 2016-12-01 Bioasis Technologies, Inc. P97 fusion proteins
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JP6847664B2 (ja) 2014-05-01 2021-03-24 バイオアシス テクノロジーズ インコーポレイテッド Ｐ９７−ポリヌクレオチド複合体
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Also Published As

Publication number Publication date
WO2005077093A2 (en) 2005-08-25
EP1720405A2 (en) 2006-11-15
AU2005211775A1 (en) 2005-08-25
US20090191178A1 (en) 2009-07-30
CA2556245A1 (en) 2005-08-25
AU2005211775B2 (en) 2009-10-08
US20080014188A1 (en) 2008-01-17
JP2007523648A (ja) 2007-08-23
EP1720405A4 (en) 2008-08-27
WO2005077093A3 (en) 2005-12-15

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Legal Events

Date Code Title Description
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

Free format text: REFERENTE A 8A ANUIDADE, CONFORME ART. 10 DA RES. 124/06.

B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: REFERENTE AO DESPACHO 8.6 PUBLICADO NA RPI 2204 DE 02/04/2013.