JP2007523648A - 高リン酸化リソソーム酵素製剤及びそれらの使用 - Google Patents

高リン酸化リソソーム酵素製剤及びそれらの使用 Download PDF

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JP2007523648A
JP2007523648A JP2006552376A JP2006552376A JP2007523648A JP 2007523648 A JP2007523648 A JP 2007523648A JP 2006552376 A JP2006552376 A JP 2006552376A JP 2006552376 A JP2006552376 A JP 2006552376A JP 2007523648 A JP2007523648 A JP 2007523648A
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cells
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ザンケル，トッド
ディー． カッキス，エミル
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Biomarin Pharmaceutical Inc
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    • 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
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    • 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

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  • Hematology (AREA)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2006552376A 2004-02-06 2005-02-07 高リン酸化リソソーム酵素製剤及びそれらの使用 Pending JP2007523648A (ja)

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

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JP2007523648A true JP2007523648A (ja) 2007-08-23
JP2007523648A5 JP2007523648A5 (https=) 2008-03-27

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US (2) US20080014188A1 (https=)
EP (1) EP1720405A4 (https=)
JP (1) JP2007523648A (https=)
AU (1) AU2005211775B2 (https=)
BR (1) BRPI0507440A (https=)
CA (1) CA2556245A1 (https=)
WO (1) WO2005077093A2 (https=)

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JP2011509674A (ja) * 2008-01-18 2011-03-31 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 高度にリン酸化された活性なヒトリソソームスルファターゼ酵素の製造およびそれの使用
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
WO2012157136A1 (ja) * 2011-05-19 2012-11-22 財団法人東京都医学総合研究所 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
JP2013520184A (ja) * 2010-02-24 2013-06-06 ザイムネックス エー／エス 組み換えリソソームα−マンノシダーゼの生産および精製のための方法
JP2014517015A (ja) * 2011-06-10 2014-07-17 プロセラ インコーポレイテッド プロテアーゼを含有する医薬組成物およびリソソーム蓄積症の治療方法
US9138465B2 (en) 2011-04-28 2015-09-22 Osaka University Pharmaceutical composition for treating lysosomal storage disease
JP2016117754A (ja) * 2009-02-20 2016-06-30 ２−ビービービー メディシンズ ベスローテン フェンノートシャップ グルタチオンをベースとする薬物送達システム
JP2017537604A (ja) * 2014-09-30 2017-12-21 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
US12246062B2 (en) 2017-05-15 2025-03-11 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
US12414985B2 (en) 2015-12-30 2025-09-16 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease

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US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
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
PT2441467E (pt) 2003-01-31 2015-10-12 Sinai School Medicine Terapia combinada para o tratamento de transtornos associados a uma deficiência proteica
EP1877099B1 (en) 2005-04-06 2012-09-19 Genzyme Corporation Therapeutic conjugates comprising a lysosomal enzyme, polysialic acid and a targeting moiety
EP2932982B1 (en) 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
SI2457920T1 (en) 2007-01-18 2018-02-28 Genzyme Corporation Oligosaccharides containing the amino acid group and their conjugates
EP2192924B1 (en) 2007-08-20 2017-10-11 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
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
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
CA3040973A1 (en) 2008-12-16 2010-07-01 Genzyme Corporation Oligosaccharide-protein conjugates
WO2010117957A2 (en) 2009-04-06 2010-10-14 Mayo Foundation For Medical Education And Research Methods and materials for delivering molecules
EP2475376B1 (en) * 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
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NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
CN103179980B (zh) 2010-06-25 2016-09-28 夏尔人类遗传性治疗公司 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物
US8956859B1 (en) 2010-08-13 2015-02-17 Aviex Technologies Llc Compositions and methods for determining successful immunization by one or more vaccines
CN103443270B (zh) 2011-01-20 2017-06-06 普罗塔里克斯有限公司 用于在植物和植物细胞中表达α‑半乳糖苷酶的核酸构建体
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
ES2582869T3 (es) 2011-07-05 2016-09-15 Bioasis Technologies Inc Conjugados de P97-anticuerpo
CA2842492A1 (en) 2011-08-05 2013-02-14 Bioasis Technologies, Inc. P97 fragments with transfer activity
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR20200032244A (ko) 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
HRP20210398T1 (hr) 2012-05-03 2021-05-28 Amicus Therapeutics, Inc. Režimi doziranja za liječenje pompeove bolesti
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WO2025007035A2 (en) * 2023-06-30 2025-01-02 Crosswalk Therapeutics, Inc. Engineered acid alpha-glucosidase variants

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Cited By (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011509674A (ja) * 2008-01-18 2011-03-31 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 高度にリン酸化された活性なヒトリソソームスルファターゼ酵素の製造およびそれの使用
JP2016117754A (ja) * 2009-02-20 2016-06-30 ２−ビービービー メディシンズ ベスローテン フェンノートシャップ グルタチオンをベースとする薬物送達システム
US10159718B2 (en) 2010-02-24 2018-12-25 Chiesi Farmaceutici S.P.A Process for production and purification of recombinant lysosomal alpha-mannosidase
JP2013520184A (ja) * 2010-02-24 2013-06-06 ザイムネックス エー／エス 組み換えリソソームα−マンノシダーゼの生産および精製のための方法
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
JPWO2011108451A1 (ja) * 2010-03-01 2013-06-27 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
US9138465B2 (en) 2011-04-28 2015-09-22 Osaka University Pharmaceutical composition for treating lysosomal storage disease
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
WO2012157136A1 (ja) * 2011-05-19 2012-11-22 財団法人東京都医学総合研究所 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
JP2014517015A (ja) * 2011-06-10 2014-07-17 プロセラ インコーポレイテッド プロテアーゼを含有する医薬組成物およびリソソーム蓄積症の治療方法
JP2017537604A (ja) * 2014-09-30 2017-12-21 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
JP2021011489A (ja) * 2014-09-30 2021-02-04 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
JP7225176B2 (ja) 2014-09-30 2023-02-20 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ－グルコシダーゼ
JP2023078118A (ja) * 2014-09-30 2023-06-06 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ－グルコシダーゼ
JP7834671B2 (ja) 2014-09-30 2026-03-24 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ－グルコシダーゼ
US12414985B2 (en) 2015-12-30 2025-09-16 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US12246062B2 (en) 2017-05-15 2025-03-11 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase

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AU2005211775A1 (en) 2005-08-25
CA2556245A1 (en) 2005-08-25
US20090191178A1 (en) 2009-07-30
EP1720405A4 (en) 2008-08-27
US20080014188A1 (en) 2008-01-17
WO2005077093A2 (en) 2005-08-25
EP1720405A2 (en) 2006-11-15
WO2005077093A3 (en) 2005-12-15
BRPI0507440A (pt) 2007-07-10
AU2005211775B2 (en) 2009-10-08

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