公开(公告)号：US20210261625A1

公开(公告)日：2021-08-26

申请号：US17162356

申请日：2021-01-29

Applicant: GENZYME CORPORATION

Inventor： Amy Frederick ,  Xiaoying Jin ,  Lin Liu ,  Catherine O'Riordan ,  Jennifer Sullivan

IPC: C07K14/005 ,  C12N15/86

Abstract: Provided herein are modified adeno-associated viral (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, and nucleic acids encoding the capsid proteins. The AAV capsids provided herein confer retinal cell tropism and/or corneal cell tropism, and mediate improved transduction efficiency in clinically relevant ocular cell types such as photoreceptors and/or corneal endothelial cells. Also provided are nucleic acids encoding the capsid proteins, and AAV particles comprising the capsid proteins.

公开(公告)号：US20240401082A1

公开(公告)日：2024-12-05

申请号：US18607056

申请日：2024-03-15

Applicant: Genzyme Corporation

Inventor： Matthew Adamowicz ,  Hao Chen ,  Amy Frederick ,  Christian Mueller ,  Catherine O’Riordan ,  Vaishnavi Rajagopal ,  John C. Reed ,  Michael John Storek

IPC: C12N15/864 ,  A61K9/00 ,  A61K31/711 ,  A61P27/02

Abstract: The present disclosure provides gene therapy that targets complement pathways for treating dry age-related macular degeneration.