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公开(公告)号：US20180071212A1
公开(公告)日：2018-03-15
申请号：US15684568
申请日：2017-08-23
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias
CPC classification number: 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
Abstract: 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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公开(公告)号：US10660944B2
公开(公告)日：2020-05-26
申请号：US16000557
申请日：2018-06-05
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias , Keethkumar Jain , Sujit Basu
Abstract: The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease), In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.
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公开(公告)号：US20190125841A1
公开(公告)日：2019-05-02
申请号：US16000557
申请日：2018-06-05
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias , Keethkumar Jain , Sujit Basu
Abstract: The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease), In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.
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公开(公告)号：US20140377244A1
公开(公告)日：2014-12-25
申请号：US14368153
申请日：2012-12-21
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias , Keethkumar Jain , Sujit Basu
CPC classification number: A61K38/465 , A61K9/0019 , A61K9/0085 , A61K9/19 , A61K38/51 , A61K47/10 , C12Y301/06008
Abstract: The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease). In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.
Abstract translation: 本发明提供了用于CNS递送溶酶体酶（例如，重组人类芳基硫酸酯酶A（rhASA））用于有效治疗溶酶体贮积病（例如Metachromatic Leukodystrophy Disease）的组合物和方法。 在一些实施方案中，本发明包括用于鞘内施用的稳定制剂，其包含ASA蛋白质和泊洛沙姆，其中小于3％的ASA蛋白以聚集形式存在。
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5.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：US20130295071A1
公开(公告)日：2013-11-07
申请号：US13862187
申请日：2013-04-12
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Nazila Salamat-Miller , Katherine Taylor , Ken Manning , Gaozhong Zhu , Paul Campolieto , Zahra Shahrokh , Pericles Calias , Thomas McCauley
CPC classification number: A61K9/0019 , A61K9/0085 , A61K9/19 , A61K38/465 , A61K38/47 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06013 , C12Y302/01046
Abstract: 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.
Abstract translation: 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：US20200237883A1
公开(公告)日：2020-07-30
申请号：US16851822
申请日：2020-04-17
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias , Keethkumar Jain , Sujit Basu
Abstract: The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g., Metachromatic Leukodystrophy Disease). In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 5% of the ASA protein exists in aggregated form.
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公开(公告)号：US10646554B2
公开(公告)日：2020-05-12
申请号：US15684568
申请日：2017-08-23
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Nazila Salamat-Miller , Katherine Taylor , Paul Campolieto , Zahra Shahrokh , Jing Pan , Lawrence Charnas , Teresa Leah Wright , Pericles Calias
IPC: A61K38/47 , A61K35/76 , A61K35/761 , A61K9/19 , C12N9/24 , A61K9/00 , A61K38/46 , C07K14/65 , C12N9/42 , A61K9/08 , A61K47/02 , A61K47/26
Abstract: 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.
