公开(公告)号：US20200046851A1

公开(公告)日：2020-02-13

申请号：US16503091

申请日：2019-07-03

Applicant: Genzyme Corporation

Inventor： Catherine O'RIORDAN ,  Matthew ADAMOWICZ

IPC: A61K48/00 ,  A61K38/17 ,  A61K9/00 ,  C12N15/86 ,  A61K31/7105 ,  C12N15/113

Abstract: Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.

公开(公告)号：US20220054657A1

公开(公告)日：2022-02-24

申请号：US17383052

申请日：2021-07-22

Applicant: Genzyme Corporation

Inventor： Catherine O'RIORDAN ,  Matthew ADAMOWICZ

IPC: A61K48/00 ,  A61K31/7105 ,  C12N15/86 ,  A61K9/00 ,  C12N15/113 ,  A61K38/17

Abstract: Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.

公开(公告)号：US20170173183A1

公开(公告)日：2017-06-22

申请号：US15127757

申请日：2015-03-20

Applicant: GENZYME CORPORATION

Inventor： Catherine O'RIORDAN ,  Matthew ADAMOWICZ

IPC: A61K48/00 ,  A61K9/00 ,  A61K38/17 ,  C12N15/86 ,  A61K31/7105

CPC classification number: A61K48/0058 ,  A61K9/0019 ,  A61K9/0048 ,  A61K31/7105 ,  A61K38/1709 ,  C12N15/113 ,  C12N15/86 ,  C12N2310/141 ,  C12N2320/34 ,  C12N2330/51 ,  C12N2750/14143

Abstract: Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.