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.

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.

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

IPC: C12N15/86 ,  A61K45/06 ,  A61P7/00

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.

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.

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.