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公开(公告)号：US11065307B2
公开(公告)日：2021-07-20
申请号：US15715748
申请日：2017-09-26
发明人： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Huang Yan , Mary Alessandrini , Richard Pfeifer
IPC分类号： A61K8/66 , C12N9/24 , A61K38/47 , A61K35/76 , A61K35/761 , A61K9/19 , A61K9/00 , A61K38/46 , C07K14/65 , C12N9/42 , A61K9/08 , A61K47/02 , A61K47/26
摘要： Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
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公开(公告)号：US20130295077A1
公开(公告)日：2013-11-07
申请号：US13892076
申请日：2013-05-10
发明人： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Huang Yan , Mary Alessandrini , Richard Pfeifer
CPC分类号： A61K38/47 , A61K9/0019 , A61K9/0085 , A61K9/08 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/46 , A61K38/465 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06008 , C12Y301/06013 , C12Y302/01045 , C12Y302/01046 , C12Y302/0105 , C12Y310/01001
摘要： Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
摘要翻译： 除此之外，本发明提供了通过例如鞘内（IT）施用Naglu蛋白来治疗Sanfilippo综合征B型（Sanfilippo B）的方法和组合物。 合适的Naglu蛋白可以是重组的，基因激活的或天然的蛋白质。 在一些实施方案中，合适的Naglu蛋白是重组Naglu蛋白。 在一些实施方案中，重组Naglu蛋白是含有Naglu结构域和溶酶体靶向部分的融合蛋白。 在一些实施方案中，溶酶体靶向结构域是IGF-II部分。
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公开(公告)号：US20220133863A1
公开(公告)日：2022-05-05
申请号：US17350909
申请日：2021-06-17
发明人： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Huang Yan , Mary Alessandrini , Richard Pfeifer
IPC分类号： A61K38/47 , A61K35/76 , A61K35/761 , A61K9/19 , C12N9/24 , A61K9/00 , A61K38/46 , C07K14/65 , C12N9/42 , A61K9/08 , A61K47/02 , A61K47/26
摘要： Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
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公开(公告)号：US09814764B2
公开(公告)日：2017-11-14
申请号：US13892076
申请日：2013-05-10
发明人： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Huang Yan , Mary Alessandrini , Richard Pfeifer
IPC分类号： A61K38/46 , A61K38/47 , A61K9/00 , A61K9/19 , C07K14/65 , C12N9/42 , C12N9/24 , A61K35/76 , A61K35/761 , A61K9/08 , A61K47/02 , A61K47/26
CPC分类号： A61K38/47 , A61K9/0019 , A61K9/0085 , A61K9/08 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/46 , A61K38/465 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06008 , C12Y301/06013 , C12Y302/01045 , C12Y302/01046 , C12Y302/0105 , C12Y310/01001
摘要： Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
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公开(公告)号：US20180085438A1
公开(公告)日：2018-03-29
申请号：US15715748
申请日：2017-09-26
发明人： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Huang Yan , Mary Alessandrini , Richard Pfeifer
IPC分类号： A61K38/47 , A61K9/00 , A61K9/19 , A61K38/46 , C12N9/24 , A61K35/76 , A61K35/761 , C07K14/65 , C12N9/42 , A61K9/08 , A61K47/02 , A61K47/26
CPC分类号： A61K38/47 , A61K9/0019 , A61K9/0085 , A61K9/08 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/46 , A61K38/465 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06008 , C12Y301/06013 , C12Y302/01045 , C12Y302/01046 , C12Y302/0105 , C12Y310/01001
摘要： Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
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