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1.发明申请公开(公告)号：US20210189357A1
公开(公告)日：2021-06-24
申请号：US17188994
申请日：2021-03-01
发明人： Erno Pungor , Charles Hague , Zhi Chen , Melita Dvorak-Ewell , Michel Claude Vellard , Vish Koppaka
摘要： This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their 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 lysosomal sulfatase enzyme.
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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：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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5.发明申请MANUFACTURE OF ACTIVE HIGHLY PHOSPHORYLATED HUMAN N-ACETYLGALACTOSAMINE-6-SULFATASE AND USES THEREOF 有权活性高磷酸化人乙酰胆碱酯酶6硫酸酯的制备及其用途公开(公告)号：US20140341878A1
公开(公告)日：2014-11-20
申请号：US14281538
申请日：2014-05-19
IPC分类号： C12N9/16
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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6.发明授权公开(公告)号：US10934534B2
公开(公告)日：2021-03-02
申请号：US16783589
申请日：2020-02-06
发明人： Erno Pungor , Charles Hague , Zhi Chen , Melita Dvorak-Ewell , Michel Claude Vellard , Vish Koppaka
摘要： This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their 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 lysosomal sulfatase enzyme.
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7.发明申请公开(公告)号：US20200181590A1
公开(公告)日：2020-06-11
申请号：US16783589
申请日：2020-02-06
发明人： Erno Pungor , Charles Hague , Zhi Chen , Melita Dvorak-Ewell , Michel Claude Vellard , Vish Koppaka
摘要： This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their 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 lysosomal sulfatase enzyme.
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8.发明申请公开(公告)号：US20160362675A1
公开(公告)日：2016-12-15
申请号：US15130694
申请日：2016-04-15
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
CPC分类号： C12N9/88 , A61K9/0019 , A61K38/51 , A61K47/60 , C07K1/14 , C12Y403/01024
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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9.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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10.发明申请Manufacture of Active Highly Phosphorylated Human N-Acetylgalactosamine-6-Sulfatase and Uses Thereof 有权活性高度磷酸化人类N-乙酰半乳糖胺-6-磺酸酯酶的制备及其用途公开(公告)号：US20160186149A1
公开(公告)日：2016-06-30
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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