公开(公告)号：US20240200096A1

公开(公告)日：2024-06-20

申请号：US18479761

申请日：2023-10-02

申请人： BIOMARIN PHARMACEUTICAL INC.

发明人： Peter COLOSI ,  Justin ISHIDA ,  Silvia RAMIREZ

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

CPC分类号： C12N15/86 ,  A61P7/00 ,  A61K45/06 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/48 ,  C12N2830/85

摘要： 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.