公开(公告)号：EP3736330A1

公开(公告)日：2020-11-11

申请号：EP19173365.8

申请日：2019-05-08

申请人： European Molecular Biology Laboratory

发明人： HEPPENSTALL, Paul ,  MAFFEI, Mariano ,  DE CASTRO REIS, Fernanda ,  POUW, Kanyn Morris

IPC分类号： C12N7/00 ,  C12N15/86 ,  A61K48/00

摘要： The present invention relates to improved adeno-associated virus (AAV) particles for gene delivery and gene therapy. Provided are adeno associated virus (AAV) particles that comprise a modified capsid. The present invention further relates to methods for producing the improved AAV particles of this invention by removing natural binding sites in adeno associated virus (AAV) capsids and introducing ligand binding sites into said capsid to provide AAVs that transduce only particular cells of interest. An additional aspect of the present invention relates to modified AAV particles for use in the treatment of a disease and methods for treating a disease, comprising administering the modified AAV particles to a subject in need thereof. Yet a further aspect of this invention relates to the AAV particles of this invention for the transfection of cells, for example as a gene delivery tool in basic research.

公开(公告)号：EP3966319A2

公开(公告)日：2022-03-16

申请号：EP20723864.3

申请日：2020-05-07

申请人： European Molecular Biology Laboratory

发明人： HEPPENSTALL, Paul ,  MAFFEI, Mariano ,  DE CASTRO REIS, Fernanda ,  POUW, Kanyn, Morris

IPC分类号： C12N7/00 ,  C12N15/86 ,  A61K48/00