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公开(公告)号：US20250122530A1
公开(公告)日：2025-04-17
申请号：US18688688
申请日：2022-09-02
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Peter COLOSI , Vincent LEONARD , Silvia RAMIREZ , Justin ISHIDA , Yu-Shan TSENG , Teague STERLING
IPC: C12N15/86 , C07K14/005
Abstract: The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
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公开(公告)号：US20240376495A1
公开(公告)日：2024-11-14
申请号：US18688682
申请日：2022-09-02
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Peter COLOSI , Vincent LEONARD , Silvia RAMIREZ , Justin ISHIDA , Yu-Shan TSENG , Teague STERLING
IPC: C12N15/86 , A61K48/00 , C07K14/005 , C12N7/00
Abstract: The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
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公开(公告)号：US20240200096A1
公开(公告)日：2024-06-20
申请号：US18479761
申请日：2023-10-02
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Peter COLOSI , Justin ISHIDA , Silvia RAMIREZ
CPC classification number: C12N15/86 , A61P7/00 , A61K45/06 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2830/008 , C12N2830/48 , C12N2830/85
Abstract: The present disclosure relates, in general, to methods for readministering, or redosing, a subject having undergone a first gene therapy regimen with a second, or subsequent, administration of a gene therapy regimen, wherein the first gene therapy vector and second gene therapy vector comprise different AAV capsids but carry a transgene or polynucleotide useful to treat the same disease or disorder.
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Patent Agency Ranking
4.发明申请公开(公告)号：US20240408240A1
公开(公告)日：2024-12-12
申请号：US18697587
申请日：2022-09-30
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Peter COLOSI , JooChaun ANG , Saeed MOSHASHAEE , Stephanie VARGAS , Thomas MACHING , Jack BROWNRIGG , Lawrence Mason SHIH , Iris CHEN , Heather Ann WENZEL
IPC: A61K48/00 , A61K9/00 , A61K38/55 , A61K45/06 , A61K47/10 , A61K47/18 , A61K47/26 , A61P7/10 , C12N15/86
Abstract: Provided herein are pharmaceutical compositions and methods for treating hereditary angioedema in a human subject.
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公开(公告)号：US20230340078A1
公开(公告)日：2023-10-26
申请号：US17777690
申请日：2020-11-13
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Peter COLOSI
CPC classification number: C07K14/8121 , A61K45/06 , A61K48/0058 , A61K48/0066 , A61P7/10 , C12N15/86
Abstract: Provided herein are compositions and methods for treating a Cl esterase inhibitor deficiency by normalizing levels of the Cl esterase inhibitor protein in a subject having HAE.
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公开(公告)号：US20230075854A1
公开(公告)日：2023-03-09
申请号：US17760227
申请日：2021-02-09
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Michael LOCHRIE , Peter COLOSI , Barrie CARTER , Tomas CINEK
IPC: C12N7/00
Abstract: The invention is directed to methods of removing adventitious virus from cells and cell lines and virus-free cells and cell lines obtainable by such methods.
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公开(公告)号：US20240384242A1
公开(公告)日：2024-11-21
申请号：US18688691
申请日：2022-09-02
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Peter COLOSI , Vincent LEONARD , Silvia RAMIREZ , Justin ISHIDA , Yu-Shan TSENG , Teague STERLING
IPC: C12N7/00 , A61K48/00 , C07K14/005 , C12N15/86
Abstract: The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
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公开(公告)号：US20240376496A1
公开(公告)日：2024-11-14
申请号：US18688685
申请日：2022-09-02
Applicant: BIOMARIN PHARMACEUTICAL INC.
Inventor： Peter COLOSI , Vincent LEONARD , Silvia RAMIREZ , Justin ISHIDA , Yu-Shan TSENG , Teague STERLING
IPC: C12N15/86 , A61K48/00 , C07K14/005
Abstract: The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
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