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公开(公告)号：US20200069819A1
公开(公告)日：2020-03-05
申请号：US16411841
申请日：2019-05-14
发明人： Stuart Bunting
摘要： The invention relates to the use of adeno-associated virus (AAV) vectors to achieve long term expression of a transgene in the liver of a juvenile subject. The invention includes the stable long-term amelioration of disease symptoms of the subjection following a single administration of an AAV vector to a juvenile subject, wherein the AAV vector delivers the transgene to the subject's liver.
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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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3.发明申请公开(公告)号：US20170087219A1
公开(公告)日：2017-03-30
申请号：US15274046
申请日：2016-09-23
摘要： The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
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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：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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公开(公告)号：US12076372B2
公开(公告)日：2024-09-03
申请号：US18157573
申请日：2023-01-20
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 , A61K38/00 , A61K38/16 , 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：US11690898B2
公开(公告)日：2023-07-04
申请号：US16675841
申请日：2019-11-06
IPC分类号： C12N15/864 , A61K38/37 , A61K48/00 , A61K47/10 , A61K47/26 , A61K39/12 , C07K14/755 , A61K47/02 , C12N7/00 , G01N33/68
CPC分类号： A61K38/37 , A61K39/12 , A61K47/02 , A61K47/10 , A61K47/26 , A61K48/0008 , A61K48/0075 , C07K14/755 , C12N7/00 , G01N33/6854 , C12N2710/16044 , C12N2750/14121 , C12N2750/14141 , C12N2750/14143 , G01N2333/015
摘要： The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
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公开(公告)号：US20200246433A1
公开(公告)日：2020-08-06
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
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：US10512675B2
公开(公告)日：2019-12-24
申请号：US15274046
申请日：2016-09-23
IPC分类号： A61K38/37 , A61K48/00 , C07K14/755 , C12N7/00 , C12N15/867 , C12N15/00 , A61K47/02 , A61K47/10 , A61K47/26 , G01N33/68 , A61K39/12
摘要： The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
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公开(公告)号：US09907834B2
公开(公告)日：2018-03-06
申请号：US15225355
申请日：2016-08-01
发明人： Sherry Bullens , Stuart Bunting , Tianwei Chou , Augustus O. Okhamafe , Christopher P. Price , Daniel J. Wendt , Clarence Yap
IPC分类号： A61K38/00 , A61K38/22 , A61K9/19 , A61K47/12 , A61K47/26 , A61K47/18 , A61K47/10 , A61K9/00 , A61K38/17
CPC分类号： A61K38/2242 , A61K9/0019 , A61K9/19 , A61K38/1709 , A61K47/10 , A61K47/12 , A61K47/183 , 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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