公开(公告)号：US10383953B2

公开(公告)日：2019-08-20

申请号：US15127757

申请日：2015-03-20

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：US12201698B2

公开(公告)日：2025-01-21

申请号：US17383052

申请日：2021-07-22

Applicant: Genzyme Corporation

Inventor： Catherine O'Riordan ,  Matthew Adamowicz

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

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：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.

公开(公告)号：US11103598B2

公开(公告)日：2021-08-31

申请号：US16503091

申请日：2019-07-03

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.