公开(公告)号：US20210189430A1

公开(公告)日：2021-06-24

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 ,  C07K14/71 ,  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：US20170096683A1

公开(公告)日：2017-04-06

申请号：US15308335

申请日：2015-05-02

Applicant: Genzyme Corporation

Inventor： Abraham SCARIA ,  Jennifer SULLIVAN ,  Lisa M. STANEK

IPC: C12N15/86 ,  C12N15/113 ,  A61K48/00 ,  C12N7/00

CPC classification number: C12N15/86 ,  A61K48/0075 ,  C07K14/015 ,  C07K14/71 ,  C12N7/00 ,  C12N15/113 ,  C12N2310/141 ,  C12N2320/32 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

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.