公开(公告)号：EP3328416A1

公开(公告)日：2018-06-06

申请号：EP16750584.1

申请日：2016-08-01

申请人： BioMarin Pharmaceutical Inc.

发明人： BULLENS, Sherry ,  BUNTING, Stuart ,  CHOU, Tianwei ,  OKHAMAFE, Augustus, O. ,  PRICE, Christopher, P. ,  WENDT, Daniel, J. ,  YAP, Clarence

IPC分类号： A61K38/22 ,  A61K38/17 ,  A61P19/08

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.

公开(公告)号：EP3794112A1

公开(公告)日：2021-03-24

申请号：EP19728796.4

申请日：2019-05-14

申请人： BioMarin Pharmaceutical Inc.

发明人： BUNTING, Stuart

IPC分类号： C12N9/02 ,  A61K38/37 ,  A61K38/44 ,  A61K38/48 ,  A61K48/00 ,  C12N15/86

公开(公告)号：EP3352787A1

公开(公告)日：2018-08-01

申请号：EP16778589.8

申请日：2016-09-23

申请人： BioMarin Pharmaceutical Inc.

发明人： BUNTING, Stuart ,  COLOSI, Peter, Cameron ,  PUNGOR, Erno

IPC分类号： A61K39/12 ,  C07K14/755

CPC分类号： A61K38/37 ,  A61K39/12 ,  A61K47/02 ,  A61K47/10 ,  A61K47/26 ,  A61K48/0008 ,  A61K48/0075 ,  C07K14/755 ,  C12N7/00 ,  C12N2710/16044 ,  C12N2750/14121 ,  C12N2750/14141 ,  C12N2750/14143 ,  G01N33/6854 ,  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.

公开(公告)号：EP4406599A2

公开(公告)日：2024-07-31

申请号：EP24162497.2

申请日：2016-08-01

申请人： BioMarin Pharmaceutical Inc.

发明人： BULLENS, Sherry ,  BUNTING, Stuart ,  CHOU, Tianwei ,  OKHAMAFE, Augustus O ,  PRICE, Christopher P. ,  WENDT, Daniel J. ,  YAP, Clarence

IPC分类号： A61P19/08

CPC分类号： A61K38/1709 ,  A61K38/2242 ,  A61K9/19 ,  A61K9/0019 ,  A61K47/12 ,  A61K47/26 ,  A61K47/20 ,  A61P19/08

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.

公开(公告)号：EP4406599A3

公开(公告)日：2024-11-06

申请号：EP24162497.2

申请日：2016-08-01

申请人： BioMarin Pharmaceutical Inc.

发明人： BULLENS, Sherry ,  BUNTING, Stuart ,  CHOU, Tianwei ,  OKHAMAFE, Augustus O ,  PRICE, Christopher P. ,  WENDT, Daniel J. ,  YAP, Clarence

IPC分类号： A61K9/00 ,  A61K9/19 ,  A61K38/17 ,  A61K38/22 ,  A61K47/12 ,  A61K47/20 ,  A61K47/26 ,  A61P19/08

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.