JP2007523648A5 - - Google Patents

Download PDF

Info

Publication number
JP2007523648A5
JP2007523648A5 JP2006552376A JP2006552376A JP2007523648A5 JP 2007523648 A5 JP2007523648 A5 JP 2007523648A5 JP 2006552376 A JP2006552376 A JP 2006552376A JP 2006552376 A JP2006552376 A JP 2006552376A JP 2007523648 A5 JP2007523648 A5 JP 2007523648A5
Authority
JP
Japan
Prior art keywords
disease
type
derivative
end3 complementation
enzyme
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.)
Pending
Application number
JP2006552376A
Other languages
English (en)
Japanese (ja)
Other versions
JP2007523648A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2005/004345 external-priority patent/WO2005077093A2/en
Publication of JP2007523648A publication Critical patent/JP2007523648A/ja
Publication of JP2007523648A5 publication Critical patent/JP2007523648A5/ja
Pending legal-status Critical Current

Links

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

Publications (2)

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

Family

ID=34860323

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2006552376A Pending JP2007523648A (ja) 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

Country Status (7)

Country Link
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=)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7723296B2 (en) 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
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
CN105274072A (zh) 2008-01-18 2016-01-27 生物马林药物股份有限公司 制备活性高度磷酸化人溶酶体硫酸酯酶及其应用
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
CA2752373C (en) * 2009-02-20 2017-10-10 To-Bbb Holding B.V. Glutathione-based drug delivery system
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
DK2538968T3 (en) * 2010-02-24 2017-12-11 Chiesi Farm Spa PROCEDURE FOR THE PREPARATION AND PURIFICATION OF RECOMBINANT LYSOSOMAL ALFA MANNOSIDASE
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
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
EP2588132A4 (en) * 2010-06-25 2014-10-15 Shire Human Genetic Therapies METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS
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
JP5665065B2 (ja) 2011-04-28 2015-02-04 国立大学法人大阪大学 リソソーム病治療用医薬組成物
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
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
US9387236B2 (en) * 2011-06-10 2016-07-12 Prothera Inc. Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases
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
ES2647082T3 (es) 2012-07-31 2017-12-19 Bioasis Technologies Inc Proteínas desfosforiladas de la enfermedad de depósito lisosómico y métodos de uso de las mismas
EA201590582A1 (ru) * 2012-12-07 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы и композиции для лечения мукополисахаридоза типа iiia с использованием интратекального введения
JP6586412B2 (ja) 2013-03-13 2019-10-02 バイオアシス テクノロジーズ インコーポレイテッド p97のフラグメントおよびその使用
JP6603227B2 (ja) 2014-02-03 2019-11-06 バイオアシス テクノロジーズ インコーポレイテッド P97融合タンパク質
DK3107562T3 (da) 2014-02-19 2019-12-16 Bioasis Technologies Inc P97-ids-fusionsproteiner
US10058619B2 (en) 2014-05-01 2018-08-28 Bioasis Technologies, Inc. P97-polynucleotide conjugates
WO2016054231A1 (en) * 2014-09-30 2016-04-07 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
KR102510941B1 (ko) * 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
TWI759291B (zh) * 2016-03-30 2022-04-01 美商阿米庫斯醫療股份有限公司 用於選擇高m6p重組蛋白之方法
US10227577B2 (en) * 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
IL325155A (en) 2016-03-30 2026-02-01 Amicus Therapeutics Inc Formulations containing recombinant acid alpha-glucosidase
EA039750B1 (ru) * 2017-03-30 2022-03-10 Амикус Терапьютикс, Инк. Способ отбора рекомбинантных белков с высоким содержанием m6p
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
FI3635009T3 (fi) 2017-06-07 2026-04-07 Regeneron Pharma Koostumuksia ja menetelmiä entsyymien internalisoimiseksi
JP7779653B2 (ja) 2018-02-07 2025-12-03 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 治療用タンパク質送達のための方法および組成物
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
KR20220129547A (ko) 2019-12-20 2022-09-23 코덱시스, 인코포레이티드 조작된 산성 알파-글루코시다제 변이체
WO2025007035A2 (en) * 2023-06-30 2025-01-02 Crosswalk Therapeutics, Inc. Engineered acid alpha-glucosidase variants

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4394448A (en) 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
US4391904A (en) 1979-12-26 1983-07-05 Syva Company Test strip kits in immunoassays and compositions therein
US5186941A (en) 1983-05-06 1993-02-16 Vestar, Inc. Vesicle formulation for the controlled release of therapeutic agents
TW492882B (en) 1997-11-28 2002-07-01 Caleb Pharmaceuticals Inc Cholinergic antagonist plaster composition
US6537785B1 (en) * 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
US6261595B1 (en) * 2000-02-29 2001-07-17 Zars, Inc. Transdermal drug patch with attached pocket for controlled heating device

Similar Documents

Publication Publication Date Title
JP2007523648A5 (https=)
US7232670B2 (en) Targeting proteins to cells expressing mannose receptors via expression in insect cells
US8580922B2 (en) Peptide linkers for polypeptide compositions and methods for using same
US8841322B2 (en) Method for increasing the activity of lysosomal enzymes
Fürst et al. Activator proteins and topology of lysosomal sphingolipid catabolism
JP5990102B2 (ja) ホスホ−6−マンノースへのマンノース−1−ホスホ−6−マンノース結合の加水分解
JP2016037504A5 (https=)
JP2015524662A5 (https=)
JP2022046524A (ja) 治療学的酵素類の結合体
US20090022702A1 (en) Methods for introducing mannose 6-phosphate and other oligosacharides onto glycoproteins
JP2007523648A (ja) 高リン酸化リソソーム酵素製剤及びそれらの使用
JP2013539974A5 (https=)
AU2019202865B2 (en) Peptide linkers for polypeptide compositions and methods for using same
Pohl et al. Glycosylation-and phosphorylation-dependent intracellular transport of lysosomal hydrolases.
Moullier et al. Long-term delivery of a lysosomal enzyme by genetically modified fibroblasts in dogs
JPWO2021072372A5 (https=)
US20160082090A1 (en) Use of Polysaccharides for Promotion of Enzymatic Activity
JP7791517B2 (ja) ライソゾームを標的とした新規ddsの開発
Greiner-Tollersrud et al. Lysosomal storage disorders
Wisselaar et al. Effects of N-hydroxyethyl-1-deoxynojirimycin (BAY m 1099) on the activity of neutral-and acid α-glucosidases in human fibroblasts and HepG2 cells
WO2022145449A1 (ja) ライソゾームを標的とした新規ddsの開発
Hancock et al. Lysosomal degradation of glycoproteins and glycosaminoglycans
Eto et al. Various sulfatase activities in leukocytes and cultured skin fibroblasts from heterozygotes for the multiple sulfatase deficiency (mukosulfatidosis)
Gote Isolation, purification and characterization of thermostable alpha-galactosidase from bacillus stearothermophilus (NCIM-5146)
Wiesmann Disorders of Lysosomal Enzymes: General Considerations