公开(公告)号：US12241078B2

公开(公告)日：2025-03-04

申请号：US17194009

申请日：2021-03-05

Applicant: Genzyme Corporation

Inventor： Abraham Scaria ,  Jennifer Sullivan ,  Lisa M. Stanek ,  Lamya S. Shihabuddin

IPC: C12N15/86 ,  A61K48/00 ,  C07K14/015 ,  C07K14/71 ,  C12N7/00 ,  C12N15/113

Abstract: Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and/or treating a CNS disorder.

公开(公告)号：US10982228B2

公开(公告)日：2021-04-20

申请号：US15308335

申请日：2015-05-02

Applicant: Genzyme Corporation

Inventor： Abraham Scaria ,  Jennifer Sullivan

IPC: C12N15/86 ,  A61K48/00 ,  C07K14/71 ,  C07K14/015 ,  C12N7/00 ,  C12N15/113

Abstract: Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and/or treating a CNS disorder.

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.