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公开(公告)号：US09283181B2
公开(公告)日：2016-03-15
申请号：US13168961
申请日：2011-06-25
申请人： Pericles Calias , Jing Pan , Jan Powell , Lawrence Charnas , Thomas McCauley , Teresa Leah Wright , Richard Pfeifer , Zahra Shahrokh
发明人： Pericles Calias , Jing Pan , Jan Powell , Lawrence Charnas , Thomas McCauley , Teresa Leah Wright , Richard Pfeifer , Zahra Shahrokh
CPC分类号： A61K38/465 , A61K9/0085 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/47 , C12N9/2402 , C12Y301/06013 , C12Y302/01045 , C12Y302/0105
摘要： The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.
摘要翻译： 本发明提供了用于将治疗剂直接递送至中枢神经系统（CNS）的有效且较少侵入性的方法。 在一些实施方案中，本发明提供了包括以下步骤的方法：鞘内施用患有或易受溶酶体酶降低的水平或活性的溶酶体贮积病的受试者，包含溶酶体酶的替代酶的组合物。
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公开(公告)号：US20120003202A1
公开(公告)日：2012-01-05
申请号：US13168961
申请日：2011-06-25
申请人： Pericles Calias , Jing Pan , Jan Powell , Lawrence Charnas , Thomas McCauley , Teresa Leah Wright , Richard Pfeifer , Zahra Shahrokh
发明人： Pericles Calias , Jing Pan , Jan Powell , Lawrence Charnas , Thomas McCauley , Teresa Leah Wright , Richard Pfeifer , Zahra Shahrokh
CPC分类号： A61K38/465 , A61K9/0085 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/47 , C12N9/2402 , C12Y301/06013 , C12Y302/01045 , C12Y302/0105
摘要： The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.
摘要翻译： 本发明提供了用于将治疗剂直接递送至中枢神经系统（CNS）的有效且较少侵入性的方法。 在一些实施方案中，本发明提供了包括以下步骤的方法：鞘内施用患有或易受溶酶体酶降低的水平或活性的溶酶体贮积病的受试者，包含溶酶体酶的替代酶的组合物。
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公开(公告)号：US09770410B2
公开(公告)日：2017-09-26
申请号：US13168963
申请日：2011-06-25
申请人： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias
发明人： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias
CPC分类号： A61K9/0085 , A61K9/0019 , A61K9/19 , A61K38/465 , A61K47/02 , A61K47/26 , C12Y301/06013
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an arylsulfatase A (ASA) protein, salt, and a polysorbate surfactant for the treatment of Metachromatic Leukodystrophy Disease.
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4.发明申请公开(公告)号：US20120009171A1
公开(公告)日：2012-01-12
申请号：US13168963
申请日：2011-06-25
申请人： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias
发明人： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias
CPC分类号： A61K9/0085 , A61K9/0019 , A61K9/19 , A61K38/465 , A61K47/02 , A61K47/26 , C12Y301/06013
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an arylsulfatase A (ASA) protein, salt, and a polysorbate surfactant for the treatment of Metachromatic Leukodystrophy Disease.
摘要翻译： 本发明尤其提供了用于CNS递送溶酶体酶以有效治疗溶酶体贮积病的组合物和方法。 在一些实施方案中，本发明包括用于直接CNS鞘内施用的稳定制剂，其包含芳基硫酸酯酶A（ASA）蛋白，盐和用于治疗间质性白细胞营养不良疾病的聚山梨醇酯表面活性剂。
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5.发明申请公开(公告)号：US20110318323A1
公开(公告)日：2011-12-29
申请号：US13168966
申请日：2011-06-25
申请人： Gaozhong Zhu , Kris Lowe , Zahra Shahrokh , James Christian , Rick Richard Fahmer , Jing Pan , Teresa Leah Wright , Pericles Calias
发明人： Gaozhong Zhu , Kris Lowe , Zahra Shahrokh , James Christian , Rick Richard Fahmer , Jing Pan , Teresa Leah Wright , Pericles Calias
CPC分类号： A61K9/0085 , A61K9/0019 , A61K9/19 , A61K38/465 , A61K47/02 , A61K47/26 , C12Y301/06013
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.
摘要翻译： 本发明尤其提供了用于CNS递送溶酶体酶以有效治疗溶酶体贮积病的组合物和方法。 在一些实施方案中，本发明包括用于直接CNS鞘内施用的稳定制剂，其包含用于治疗猎人综合征的艾杜糖醛酸-2-硫酸酯酶（I2S）蛋白质，盐和聚山梨醇酯表面活性剂。
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公开(公告)号：US08545837B2
公开(公告)日：2013-10-01
申请号：US13168966
申请日：2011-06-25
申请人： Gaozhong Zhu , Kris Lowe , Zahra Shahrokh , James Christian , Richard Fahmer , Jing Pan , Teresa Leah Wright , Pericles Calias
发明人： Gaozhong Zhu , Kris Lowe , Zahra Shahrokh , James Christian , Richard Fahmer , Jing Pan , Teresa Leah Wright , Pericles Calias
CPC分类号： A61K9/0085 , A61K9/0019 , A61K9/19 , A61K38/465 , A61K47/02 , A61K47/26 , C12Y301/06013
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.
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7.发明申请METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE 有权用于中枢神经系统递送HEPARAN N-SULFATASE的方法和组合物公开(公告)号：US20120014936A1
公开(公告)日：2012-01-19
申请号：US13168957
申请日：2011-06-25
申请人： Farah Natoli , Gaozhong Zhu , Jennifer Terew , Yuan Jiang , Jamie Tsung , Zahra Shahrokh , Brian Vernaglia , Jing Pan , Richard Pfeifer , Pericles Calias
发明人： Farah Natoli , Gaozhong Zhu , Jennifer Terew , Yuan Jiang , Jamie Tsung , Zahra Shahrokh , Brian Vernaglia , Jing Pan , Richard Pfeifer , Pericles Calias
CPC分类号： A61K9/0085 , A61K9/0019 , A61K9/19 , A61K38/465 , A61K47/02 , A61K47/26 , C12N9/14 , C12N9/2402 , C12Y302/0105 , C12Y310/01001
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.
摘要翻译： 本发明尤其提供了用于CNS递送溶酶体酶以有效治疗溶酶体贮积病的组合物和方法。 在一些实施方案中，本发明包括用于直接CNS鞘内施用的稳定制剂，其包含用于治疗Sanfilippo综合征A型的肝素N-硫酸酯酶（HNS）蛋白质，盐和聚山梨醇酯表面活性剂。
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公开(公告)号：US09320711B2
公开(公告)日：2016-04-26
申请号：US13168957
申请日：2011-06-25
申请人： Farah Natoli , Gaozhong Zhu , Jennifer Terew , Yuan Jiang , Jamie Tsung , Zahra Shahrokh , Brian Vernaglia , Jing Pan , Richard Pfeifer , Pericles Calias
发明人： Farah Natoli , Gaozhong Zhu , Jennifer Terew , Yuan Jiang , Jamie Tsung , Zahra Shahrokh , Brian Vernaglia , Jing Pan , Richard Pfeifer , Pericles Calias
IPC分类号： A61K38/46 , C12N9/14 , A61P25/00 , C12Q1/34 , A61K9/00 , A61K9/19 , C12N9/24 , A61K47/02 , A61K47/26
CPC分类号： A61K9/0085 , A61K9/0019 , A61K9/19 , A61K38/465 , A61K47/02 , A61K47/26 , C12N9/14 , C12N9/2402 , C12Y302/0105 , C12Y310/01001
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.
摘要翻译： 本发明尤其提供了用于CNS递送溶酶体酶以有效治疗溶酶体贮积病的组合物和方法。 在一些实施方案中，本发明包括用于直接CNS鞘内施用的稳定制剂，其包含用于治疗Sanfilippo综合征A型的肝素N-硫酸酯酶（HNS）蛋白质，盐和聚山梨醇酯表面活性剂。
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9.发明申请METHODS AND COMPOSITIONS FOR CNS DELIVERY OF B-GALACTOCEREBROSIDASE 审中-公开CNS递送B-GALACTOCEREBROSIDASE的方法和组合物公开(公告)号：US20110318324A1
公开(公告)日：2011-12-29
申请号：US13168970
申请日：2011-06-25
申请人： Nazila Salamat-Miller , Katherine Taylor , Ken Manning , Gaozhong Zhu , Paul Campolieto , Zahra Shahrokh , Pericles Calias , Thomas McCauley
发明人： Nazila Salamat-Miller , Katherine Taylor , Ken Manning , Gaozhong Zhu , Paul Campolieto , Zahra Shahrokh , Pericles Calias , Thomas McCauley
CPC分类号： A61K9/0019 , A61K9/0085 , A61K9/19 , A61K38/465 , A61K38/47 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06013 , C12Y302/01046
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an B-Galactocerebrosidase protein, salt, and a polysorbate surfactant for the treatment of GLD Disease.
摘要翻译： 本发明尤其提供了用于CNS递送溶酶体酶以有效治疗溶酶体贮积病的组合物和方法。 在一些实施方案中，本发明包括用于直接CNS鞘内施用的稳定制剂，其包含用于治疗GLD疾病的B-半乳糖脑苷脂酶蛋白质，盐和聚山梨醇酯表面活性剂。
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公开(公告)号：US20110318327A1
公开(公告)日：2011-12-29
申请号：US13168969
申请日：2011-06-25
申请人： 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 , Yan Huang , Mary Alessandrini , Richard Pfeifer
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 , Yan Huang , Mary Alessandrini , Richard Pfeifer
CPC分类号： A61K9/0085 , A61K9/0019 , A61K9/19 , A61K38/465 , A61K47/02 , A61K47/26 , C12Y301/06013
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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