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公开(公告)号：US20160031965A1
公开(公告)日：2016-02-04
申请号：US14883203
申请日：2015-10-14
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
CPC classification number: C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
Abstract: 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：US20140161788A1
公开(公告)日：2014-06-12
申请号：US14092336
申请日：2013-11-27
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
CPC classification number: C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
Abstract: 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.
Abstract translation: 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，其包含靶向治疗性融合蛋白，其包含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：US20230192799A1
公开(公告)日：2023-06-22
申请号：US17642150
申请日：2020-09-16
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Daniel J. Wendt , Geoffrey Berguig , Karol Estrada , Jonathan LeBowitz
CPC classification number: C07K14/58 , A61K47/542 , A61P19/08 , A61K38/00
Abstract: The present disclosure, relates, in general, to stable variants of C-type natriuretic peptide (CNP) and uses thereof to treat bone-related disorders.
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公开(公告)号：US11254725B2
公开(公告)日：2022-02-22
申请号：US16378163
申请日：2019-04-08
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
Abstract: 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：US20210069304A1
公开(公告)日：2021-03-11
申请号：US16869862
申请日：2020-05-08
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Jonathan LeBowitz , John Maga
Abstract: 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.
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Patent Agency Ranking
公开(公告)号：US20160039900A1
公开(公告)日：2016-02-11
申请号：US14883211
申请日：2015-10-14
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
CPC classification number: C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
Abstract: 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：US20220280647A1
公开(公告)日：2022-09-08
申请号：US17634034
申请日：2020-08-12
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Jake Ang , Sunil Kumar Battu , Joshua Laber , Karol Estrada , Jonathan LeBowitz , Joyce Chou
Abstract: The present disclosure, relates, in general, to hydrophobic salts of hydrophilic peptides that form low solubility materials in aqueous solutions and are capable of extended or sustained release of the peptide component when administered to a subject. Hydrophobic salts of C-type natriuretic peptides and uses thereof are also disclosed.
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公开(公告)号：US10301369B2
公开(公告)日：2019-05-28
申请号：US15688438
申请日：2017-08-28
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
Abstract: 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：US09834587B2
公开(公告)日：2017-12-05
申请号：US14883193
申请日：2015-10-14
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
CPC classification number: C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
Abstract: 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：US09771408B2
公开(公告)日：2017-09-26
申请号：US14883211
申请日：2015-10-14
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
CPC classification number: C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
Abstract: 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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