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：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：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.