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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
发明人： Daniel J. Wendt , Shinong Long , Sianna Castillo , Christopher P. Price , Mika Aoyagi-Scharber , Michel Claude Vellard , Augustus O. Okhamafe
CPC分类号： 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
摘要： 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
发明人： Daniel J. Wendt , Shinong Long , Sianna Castillo , Christopher P. Price , Mika Aoyagi-Scharber , Michel Claude Vellard , Augustus O. Okhamafe
CPC分类号： 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
摘要： 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).
摘要翻译： 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：US20160074406A1
公开(公告)日：2016-03-17
申请号：US14947482
申请日：2015-11-20
发明人： Tianwei Chou , Augustus O. Okhamafe
IPC分类号： A61K31/519 , A61J1/14 , A61K9/48 , A61K9/00 , A61K31/405
CPC分类号： A61K31/519 , A61J1/1418 , A61K9/0053 , A61K9/009 , A61K9/145 , A61K9/4816 , A61K9/4825 , A61K9/485 , A61K9/4858 , A61K9/4866 , A61K31/405
摘要： Dry blend powder formulations comprising a pharmaceutical formulation containing tetrahydrobiopterin, and methods of making and using the same, are disclosed herein.
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公开(公告)号：US11911446B2
公开(公告)日：2024-02-27
申请号：US16837905
申请日：2020-04-01
发明人： Sherry Bullens , Stuart Bunting , Tianwei Chou , Augustus O. Okhamafe , Christopher P. Price , Daniel J. Wendt , Clarence Yap
IPC分类号： A61K38/22 , A61K38/17 , A61K9/19 , A61K47/12 , A61K47/26 , A61K47/10 , A61K9/00 , A61K47/20 , A61K47/18
CPC分类号： A61K38/2242 , A61K9/0019 , A61K9/19 , A61K38/1709 , A61K47/10 , A61K47/12 , A61K47/183 , A61K47/20 , A61K47/26
摘要： 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
发明人： 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
摘要： 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：US20190216905A1
公开(公告)日：2019-07-18
申请号：US16368091
申请日：2019-03-28
摘要： Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed. Methods of using rhTPP1 in the prevention and treatment of symptoms of Neuronal Ceroid Lipofuscinosis (CLN2) disease are also disclosed. The formulations and methods are effective in halting the progression of CLN2 disease and may be used to treat subjects having CLN2 or a family history of CLN2.
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公开(公告)号：US20240156914A1
公开(公告)日：2024-05-16
申请号：US18425954
申请日：2024-01-29
发明人： Sherry Bullens , Stuart Bunting , Tianwei Chou , Augustus O. Okhamafe , Christopher P. Price , Daniel J. Wendt , Clarence Yap
IPC分类号： A61K38/22 , A61K9/00 , A61K9/19 , A61K38/17 , A61K47/10 , A61K47/12 , A61K47/18 , A61K47/20 , A61K47/26
CPC分类号： A61K38/2242 , A61K9/0019 , A61K9/19 , A61K38/1709 , A61K47/10 , A61K47/12 , A61K47/183 , A61K47/20 , A61K47/26
摘要： 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：US11229687B2
公开(公告)日：2022-01-25
申请号：US16941454
申请日：2020-07-28
摘要： Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed. Methods of using rhTPP1 in the prevention and treatment of symptoms of Neuronal Ceroid Lipofuscinosis (CLN2) disease are also disclosed. The formulations and methods are effective in halting the progression of CLN2 disease and may be used to treat subjects having CLN2 or a family history of CLN2.
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公开(公告)号：US10646550B2
公开(公告)日：2020-05-12
申请号：US15880002
申请日：2018-01-25
发明人： Sherry Bullens , Stuart Bunting , Tianwei Chou , Augustus O. Okhamafe , Christopher P. Price , Daniel J. Wendt , Clarence Yap
IPC分类号： A61K38/22 , A61K38/00 , A61K38/16 , A61K9/19 , A61K47/12 , A61K47/26 , A61K47/18 , A61K47/10 , A61K9/00 , A61K38/17
摘要： 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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10.发明授权Manufacture of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase and uses thereof 有权活性高度磷酸化人N-乙酰半乳糖胺-6-硫酸酯酶的制备及其用途公开(公告)号：US09567572B2
公开(公告)日：2017-02-14
申请号：US14573907
申请日：2014-12-17
CPC分类号： C12N9/16 , A61K9/08 , A61K38/00 , A61K38/465 , C12Y301/06004
摘要： This invention provides compositions of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase (GALNS), and pharmaceutical compositions and formulations thereof, methods of producing and purifying GALNS, and its use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the GALNS enzyme, e.g., Mucopolysaccharidosis IVa (MPS IVa or Morquio A syndrome).
摘要翻译： 本发明提供活性高度磷酸化的人N-乙酰半乳糖胺-6-硫酸酯酶（GALNS）的组合物及其药物组合物及其制剂，制备和纯化GALNS的方法及其在疾病和病症的诊断，预防或治疗中的用途， 包括特别是由GALNS酶的缺陷引起或与之相关的溶酶体贮积病，例如粘多糖贮积症IVa（MPS IVa或Morquio A综合征）。
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