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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：USRE46707E1
公开(公告)日：2018-02-13
申请号：US14604262
申请日：2015-01-23
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Daniel J. Wendt , Shinong Long , Sianna Castillo , Christopher P. Price , Mika Aoyagi-Scharber , Michel Claude Vellard , Augustus O. Okhamafe
CPC classification number: C07K14/58 , A61K9/08 , A61K38/00 , A61K38/22 , A61K38/2242 , A61K47/10 , A61K47/20 , A61K47/22 , A61K47/26 , A61K47/60 , G01N2333/58 , G01N2800/10 , G01N2800/105 , G01N2800/385 , G01N2800/52
Abstract: The present disclosure provides variants of C-type natriuretic peptide (CNP), pharmaceutical compositions comprising CNP variants, and methods of making CNP variants. The CNP variants are useful as therapeutic agents for the treatment of diseases responsive to CNP, including but not limited to bone-related disorders, such as skeletal dysplasias (e.g., achondroplasia), and vascular smooth muscle disorders (e.g., restenosis and arteriosclerosis).
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公开(公告)号：US20170051033A1
公开(公告)日：2017-02-23
申请号：US15332793
申请日：2016-10-24
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Daniel J. Wendt , Shinong Long , Sianna Castillo , Christopher P. Price , Mika Aoyagi-Scharber , Michel Claude Vellard , Augustus O. Okhamafe
CPC classification number: C07K14/58 , A61K9/08 , A61K38/00 , A61K38/22 , A61K38/2242 , A61K47/10 , A61K47/20 , A61K47/22 , A61K47/26 , A61K47/60 , G01N2333/58 , G01N2800/10 , G01N2800/105 , G01N2800/385 , G01N2800/52
Abstract: The present disclosure provides variants of C-type natriuretic peptide (CNP), pharmaceutical compositions comprising CNP variants, and methods of making CNP variants. The CNP variants are useful as therapeutic agents for the treatment of diseases responsive to CNP, including but not limited to bone-related disorders, such as skeletal dysplasias (e.g., achondroplasia), and vascular smooth muscle disorders (e.g., restenosis and arteriosclerosis).
Abstract translation: 本公开提供了C型利钠肽（CNP），包含CNP变体的药物组合物和制备CNP变体的方法的变体。 CNP变体可用作治疗针对CNP的疾病的治疗剂，包括但不限于骨相关疾病，例如骨骼发育不良（例如软骨发育不良）和血管平滑肌病（例如再狭窄和动脉硬化）。
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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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Patent Agency Ranking
公开(公告)号：US11911446B2
公开(公告)日：2024-02-27
申请号：US16837905
申请日：2020-04-01
Applicant: BioMarin Pharmaceutical Inc.
Inventor： Sherry Bullens , Stuart Bunting , Tianwei Chou , Augustus O. Okhamafe , Christopher P. Price , Daniel J. Wendt , Clarence Yap
IPC: A61K38/22 , A61K38/17 , A61K9/19 , A61K47/12 , A61K47/26 , A61K47/10 , A61K9/00 , A61K47/20 , A61K47/18
CPC classification number: A61K38/2242 , A61K9/0019 , A61K9/19 , A61K38/1709 , A61K47/10 , A61K47/12 , A61K47/183 , A61K47/20 , A61K47/26
Abstract: The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
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公开(公告)号：US11505790B2
公开(公告)日：2022-11-22
申请号：US16246300
申请日：2019-01-11
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Augustus O. Okhamafe , Sean M. Bell , G. Nick Zecherle , Kris Antonsen , Yanhong Zhang , Kieu Ly Tran , Paul A. Fitzpatrick , Emil D. Kakkis , Michel Claude Vellard , Daniel J. Wendt , Mubarack Muthalif
Abstract: Provided herein are phenylalanine ammonia-lyase (PAL) variants produced by prokaryotes, wherein such prokaryotic PAL variant has a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL. Further provided are compositions of prokaryotic PAL and biologically active fragments, mutants, variants or analogs thereof, as well as methods for the production, purification, formulation, and use of such compositions for industrial and therapeutic purposes, e.g., treating hyperphenylalaninemia, including phenylketonuria, and other disorders, including cancer.
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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：US20240374688A1
公开(公告)日：2024-11-14
申请号：US18785340
申请日：2024-07-26
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Sherry Bullens , Stuart Bunting , Tianwei Chou , Augustus O. Okhamafe , Christopher P. Price , Daniel J. Wendt , Clarence Yap
IPC: A61K38/22 , A61K9/00 , A61K9/19 , A61K38/17 , A61K47/10 , A61K47/12 , A61K47/18 , A61K47/20 , A61K47/26
Abstract: The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
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