公开(公告)号：EP4005603A1

公开(公告)日：2022-06-01

申请号：EP21201945.9

申请日：2012-04-20

申请人： The Regents of the University of California

发明人： SCHAFFER, David V. ,  KLIMCZAK, Ryan R. ,  KOERBER, James, T. ,  FLANNERY, John G. ,  DALKARA MOUROT, Deniz ,  VISEL, Meike ,  BYRNE, Leah C.T.

IPC分类号： A61K48/00 ,  C12N15/63 ,  A61P9/00 ,  A61P3/10 ,  C07K14/005 ,  A61P27/00 ,  A61P43/00 ,  A61P27/02 ,  A61P27/06 ,  A61P9/10 ,  C12N15/86 ,  A61K38/17 ,  A61K9/00 ,  C12N7/00 ,  C12Q1/70 ,  C12N15/113 ,  C12N15/115 ,  G01N33/50

摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.

公开(公告)号：EP3693025A1

公开(公告)日：2020-08-12

申请号：EP20151479.1

申请日：2012-04-20

申请人： The Regents of the University of California

发明人： SCHAFFER, David V. ,  KLIMCZAK, Ryan R. ,  KOERBER, James, T. ,  FLANNERY, John G. ,  DALKARA MOUROT, Deniz ,  VISEL, Meike ,  BYRNE, Leah C.T.

IPC分类号： A61K48/00 ,  C12N15/63 ,  C07K14/005 ,  C12N15/86 ,  A61P3/10 ,  A61P9/00 ,  A61P9/10 ,  A61P27/00 ,  A61P27/02 ,  A61P27/06 ,  A61P43/00

摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.