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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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2.发明申请LIPOPROTEIN LIPASE FOR TREATMENT OF HYPERTRIGLYCERIDEMIC-RELATED CONDITIONS INCLUDING ACUTE PANCREATITIS 有权用于治疗包括急性胰腺炎的高血糖相关病症的利培酮公开(公告)号：US20160051637A1
公开(公告)日：2016-02-25
申请号：US14830481
申请日：2015-08-19
发明人： Omar L. Francone , Lin Guey , Kevin Holmes , Bruce Tangarone , Matthew Traylor , Lenore von Krusenstiern , Tracy Dowie , Lieh Yoon Low , Bohong Zhang , Muthuraman Meiyappan , Angela Norton , Bettina Strack-Logue , Dianna Lundberg , Michael F. Concino
IPC分类号： A61K38/46
CPC分类号： A61K38/465 , A61K9/0019 , A61K9/08 , A61K9/19 , C12N9/20 , C12Y301/01034
摘要： A lipoprotein lipase (LPL) protein for treating and/or preventing HTG and its associated diseases, including but not limited to acute pancreatitis (AP), and in particular, acute pancreatitis secondary to or exacerbated by hypertriglyceridemia, and hypertriglyceridemia and its associated diseases in general, including cardiovascular and metabolic diseases, endocrine disorders, and fat embolism syndrome.
摘要翻译： 用于治疗和/或预防HTG及其相关疾病的脂蛋白脂肪酶（LPL）蛋白，包括但不限于急性胰腺炎（AP），特别是由高甘油三酯血症继发或加重的急性胰腺炎，以及高甘油三酯血症及其相关疾病 一般，包括心血管和代谢疾病，内分泌失调和脂肪栓塞综合征。
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公开(公告)号：US09597376B2
公开(公告)日：2017-03-21
申请号：US14830481
申请日：2015-08-19
发明人： Omar L. Francone , Lin Guey , Kevin Holmes , Bruce Tangarone , Matthew Traylor , Lenore von Krusenstiern , Tracy Dowie , Lieh Yoon Low , Bohong Zhang , Muthuraman Meiyappan , Angela Norton , Bettina Strack-Logue , Dianna Lundberg , Michael F. Concino
IPC分类号： A61K38/46
CPC分类号： A61K38/465 , A61K9/0019 , A61K9/08 , A61K9/19 , C12N9/20 , C12Y301/01034
摘要： A lipoprotein lipase (LPL) protein for treating and/or preventing HTG and its associated diseases, including but not limited to acute pancreatitis (AP), and in particular, acute pancreatitis secondary to or exacerbated by hypertriglyceridemia, and hypertriglyceridemia and its associated diseases in general, including cardiovascular and metabolic diseases, endocrine disorders, and fat embolism syndrome.
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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：US10722559B2
公开(公告)日：2020-07-28
申请号：US15503223
申请日：2015-08-11
发明人： Michael F. Concino , Bettina Strack-Logue , Muthuraman Meiyappan , Angela W. Norton , Bohong Zhang , Andrea Iskenderian , Lieh Yoon Low , Dianna Lundberg , Alla Romashko , Hicham Naimy
IPC分类号： A61K38/47 , A61K38/43 , A61K38/17 , A61K38/20 , A61K38/30 , C07K14/47 , C07K14/54 , C07K14/65 , C12N9/12
摘要： A targeted therapeutic including a lysosomal enzyme and a lysosomal targeting moiety that is a peptide containing at least one N-linked glycosylation site. Methods of producing the targeted therapeutic may include nucleotide acids encoding the same and host cells co-expressing GNPT. Pharmaceutical compositions comprising the targeted therapeutic and methods of using the same to treat a lysosomal storage disease.
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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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9.发明申请公开(公告)号：US20170202929A1
公开(公告)日：2017-07-20
申请号：US15411559
申请日：2017-01-20
发明人： Omar L. Francone , Lin Guey , Kevin Holmes , Bruce Tangarone , Matthew Traylor , Lenore von Krusenstiern , Tracy Dowie , Lieh Yoon Low , Bohong Zhang , Muthuraman Meiyappan , Angela Norton , Bettina Strack-Logue , Dianna Lundberg , Michael F. Concino
CPC分类号： A61K38/465 , A61K9/0019 , A61K9/08 , A61K9/19 , C12N9/20 , C12Y301/01034
摘要： A lipoprotein lipase (LPL) protein for treating and/or preventing HTG and its associated diseases, including but not limited to acute pancreatitis (AP), and in particular, acute pancreatitis secondary to or exacerbated by hypertriglyceridemia, and hypertriglyceridemia and its associated diseases in general, including cardiovascular and metabolic diseases, endocrine disorders, and fat embolism syndrome.
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