JP2014534962A5 - - Google Patents

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Publication number
JP2014534962A5
JP2014534962A5 JP2014535865A JP2014535865A JP2014534962A5 JP 2014534962 A5 JP2014534962 A5 JP 2014534962A5 JP 2014535865 A JP2014535865 A JP 2014535865A JP 2014535865 A JP2014535865 A JP 2014535865A JP 2014534962 A5 JP2014534962 A5 JP 2014534962A5
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JP
Japan
Prior art keywords
composition
naglu
rhnaglu
protein
rhnaglu protein
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
JP2014535865A
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English (en)
Japanese (ja)
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JP6329483B2 (ja
JP2014534962A (ja
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Publication date
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Priority claimed from PCT/US2012/059708 external-priority patent/WO2013055888A2/en
Publication of JP2014534962A publication Critical patent/JP2014534962A/ja
Publication of JP2014534962A5 publication Critical patent/JP2014534962A5/ja
Application granted granted Critical
Publication of JP6329483B2 publication Critical patent/JP6329483B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2014535865A 2011-10-12 2012-10-11 組換えヒトnagluタンパク質およびその利用 Expired - Fee Related JP6329483B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161546248P 2011-10-12 2011-10-12
US61/546,248 2011-10-12
PCT/US2012/059708 WO2013055888A2 (en) 2011-10-12 2012-10-11 Recombinant human naglu protein and uses thereof

Publications (3)

Publication Number Publication Date
JP2014534962A JP2014534962A (ja) 2014-12-25
JP2014534962A5 true JP2014534962A5 (enExample) 2015-11-26
JP6329483B2 JP6329483B2 (ja) 2018-05-23

Family

ID=48082738

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2014535865A Expired - Fee Related JP6329483B2 (ja) 2011-10-12 2012-10-11 組換えヒトnagluタンパク質およびその利用

Country Status (18)

Country Link
US (3) US9579366B2 (enExample)
EP (1) EP2766477B1 (enExample)
JP (1) JP6329483B2 (enExample)
KR (1) KR20140108519A (enExample)
CN (1) CN103958676A (enExample)
AU (1) AU2012322811A1 (enExample)
BR (1) BR112014008873A2 (enExample)
CA (1) CA2852027A1 (enExample)
CL (1) CL2014000927A1 (enExample)
CO (1) CO7010783A2 (enExample)
HK (1) HK1200489A1 (enExample)
IL (1) IL232073A0 (enExample)
IN (1) IN2014MN00740A (enExample)
MX (1) MX2014004359A (enExample)
RU (1) RU2014117291A (enExample)
SG (2) SG11201401457SA (enExample)
TW (1) TW201321515A (enExample)
WO (1) WO2013055888A2 (enExample)

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CA3184105A1 (en) 2007-07-27 2009-02-05 Armagen Inc. Methods and compositions for increasing alpha-l-iduronidase activity in the cns
JP2013507131A (ja) 2009-10-09 2013-03-04 アーメイゲン・テクノロジーズ・インコーポレイテッド Cnsにおけるイズロン酸2−スルファターゼ活性を増加させるための方法および組成物
BR122019021757B1 (pt) 2010-09-09 2020-12-08 Alexion Pharmaceuticals, Inc composição farmacêutica para tratamento de um paciente humano sofrendo de uma deficiência de lipase ácida lisossomal (lal)
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
WO2013055888A2 (en) * 2011-10-12 2013-04-18 Synageva Biopharma Corp. Recombinant human naglu protein and uses thereof
US8486399B2 (en) 2011-12-02 2013-07-16 Armagen Technologies, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
JP2016523835A (ja) 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
TWI793159B (zh) * 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
SG11201609314XA (en) * 2014-05-14 2016-12-29 Esteve Labor Dr Adenoassociated virus vectors for the treatment of lysosomal storage disorders
EP3160494B1 (en) * 2014-06-25 2019-11-20 Shire Human Genetic Therapies, Inc. Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase
WO2016025519A1 (en) * 2014-08-11 2016-02-18 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
AU2015323966A1 (en) 2014-09-29 2017-03-16 Alexion Pharmaceuticals, Inc. Methods of treating mucopolysaccharidosis IIIB (MPSIIIB)
CN104524662B (zh) * 2014-12-31 2017-02-22 迈得医疗工业设备股份有限公司 一种医用针座组装的上料机构
US10538589B2 (en) * 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
US20190083583A1 (en) * 2016-01-29 2019-03-21 Alexion Pharmaceuticals, Inc. Enzyme replacement therapy in mucopolysaccharidosis iiib patients
US11253612B2 (en) 2016-04-15 2022-02-22 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
US10633705B2 (en) 2016-10-14 2020-04-28 Alexion Pharmaceuticals, Inc. N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
EP4100532B1 (en) 2020-03-23 2026-08-12 The University of North Carolina at Chapel Hill Aav-naglu vectors for treatment of mucopolysaccharidosis iiib
CN111718947B (zh) * 2020-06-18 2022-08-23 舒泰神(北京)生物制药股份有限公司 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途

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US6825396B2 (en) 1996-06-12 2004-11-30 Board Of Trustees Operating Michigan State University Methods for tissue specific synthesis of protein in eggs of transgenic hens
US5897998A (en) 1997-08-04 1999-04-27 The University Of Georgia Research Foundation, Inc. Method for manipulating avian eggs
AU752946B2 (en) 1997-10-16 2002-10-03 Avigenics, Inc. Vectors comprising a magnum-specific promoter for avian transgenesis
US7511120B2 (en) 1997-10-16 2009-03-31 Synageva Biopharma Corp. Glycosylated G-CSF obtained from a transgenic chicken
US20040019923A1 (en) 1997-10-16 2004-01-29 Ivarie Robert D. Exogenous proteins expressed in avians and their eggs
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US6534300B1 (en) * 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
US7001994B2 (en) * 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
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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
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CN103269709A (zh) * 2010-06-25 2013-08-28 夏尔人类遗传性治疗公司 Sanfilippo综合症类型B的治疗
CA2803166C (en) * 2010-06-25 2023-03-07 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
EP2596123B1 (en) 2010-07-22 2015-11-25 Shire Human Genetic Therapies, Inc. Crystal structure of human alpha-n-acetylglucosaminidase
BR122019021757B1 (pt) 2010-09-09 2020-12-08 Alexion Pharmaceuticals, Inc composição farmacêutica para tratamento de um paciente humano sofrendo de uma deficiência de lipase ácida lisossomal (lal)
WO2013055888A2 (en) * 2011-10-12 2013-04-18 Synageva Biopharma Corp. Recombinant human naglu protein and uses thereof

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