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1.发明公开TARGETED THERAPEUTIC LYSOSOMAL ENZYME FUSION PROTEINS AND USES THEREOF 审中-公开GEZIELTE THERAPEUTISCHE LYSOSOMALE ENZYMFUSIONSPROTEINE UND VERWENDUNGEN DAVON
公开(公告)号：EP3115372A1
公开(公告)日：2017-01-11
申请号：EP16182420.6
申请日：2013-11-27
发明人： AOYAGI-SCHARBER, Mika , CHRISTIANSON, Teresa Margaret , DVORAK-EWELL, Melita , WENDT, Daniel , LONG, Shinong , LEBOWITZ, Jonathan , GOLD, Daniel Solomon
CPC分类号： C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
摘要： The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.
摘要翻译： 本发明一般涉及可用于治疗溶酶体贮积病的治疗融合蛋白和治疗这些疾病的方法。 示例性治疗性融合蛋白包含溶酶体酶，溶酶体靶向部分，例如IGF-II肽和间隔肽。 还提供了用于治疗IIIB型粘多糖贮积症（Sanfilippo B综合征）的组合物和方法，其包含包含α-N-乙酰氨基葡糖苷酶（Naglu），溶酶体靶向部分（例如IGF-II肽）和间隔肽 。
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公开(公告)号：EP4031183A1
公开(公告)日：2022-07-27
申请号：EP20785629.5
申请日：2020-09-16
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公开(公告)号：EP4013453A1
公开(公告)日：2022-06-22
申请号：EP20761459.5
申请日：2020-08-12
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公开(公告)号：EP3075386B1
公开(公告)日：2019-10-16
申请号：EP16153694.1
申请日：2010-06-17
发明人： LEBOWITZ, Jonathan , CHANG, Byeong
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公开(公告)号：EP3115372B1
公开(公告)日：2019-03-06
申请号：EP16182420.6
申请日：2013-11-27
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公开(公告)号：EP3272773B8
公开(公告)日：2020-11-18
申请号：EP17182845.2
申请日：2009-05-07
发明人： LEBOWITZ, Jonathan , MAGA, John
IPC分类号： C07K19/00 , C07K14/475
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公开(公告)号：EP3272773A1
公开(公告)日：2018-01-24
申请号：EP17182845.2
申请日：2009-05-07
发明人： LEBOWITZ, Jonathan , MAGA, John
IPC分类号： C07K19/00 , C07K14/475
摘要： The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.
摘要翻译： 基于GILT技术，本发明提供了用于有效溶酶体靶向的进一步改进的组合物和方法。 除此之外，本发明提供了使用弗林蛋白酶抗性溶酶体靶向肽将溶酶体酶靶向溶酶体的方法和组合物。 本发明还提供了使用对胰岛素受体具有降低或降低的结合亲和力的溶酶体靶向肽将溶酶体酶靶向溶酶体的方法和组合物。
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公开(公告)号：EP3075386A1
公开(公告)日：2016-10-05
申请号：EP16153694.1
申请日：2010-06-17
发明人： LEBOWITZ, Jonathan , CHANG, Byeong
摘要： The present invention provides improved formulations for lysosomal enzymes useful for enzyme replacement therapy. Among other things, the present invention provides formulations that preserve or enhance the stability and/or efficacy of a lysosomal enzyme such as acid alpha-glucosidase.
摘要翻译： 本发明提供了用于酶替代疗法的溶酶体酶的改进制剂。 其中，本发明提供了保持或增强溶酶体酶如酸性α-葡糖苷酶的稳定性和/或功效的制剂。
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公开(公告)号：EP2925776A1
公开(公告)日：2015-10-07
申请号：EP13802834.5
申请日：2013-11-27
发明人： AOYAGI-SCHARBER, Mika , CHRISTIANSON, Teresa, Margaret , DVORAK-EWELL, Melita , WENDT, Daniel, J. , LONG, Shinong , LEBOWITZ, Jonathan , GOLD, Daniel, Solomon
CPC分类号： C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
摘要： The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.
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公开(公告)号：EP3679942A1
公开(公告)日：2020-07-15
申请号：EP19203438.7
申请日：2010-06-17
发明人： LEBOWITZ, Jonathan , CHANG, Byeong
摘要： The present invention provides improved formulations for lysosomal enzymes useful for enzyme replacement therapy. Among other things, the present invention provides formulations that preserve or enhance the stability and/or efficacy of a lysosomal enzyme such as acid alpha-glucosidase.
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