| 发明名称 |
Transferrin/transferrin receptor-mediated siRNA delivery |
| 摘要 |
The invention provides interfering RNA molecule-ligand conjugates useful as a delivery system for delivering interfering RNA molecules to a cell in vitro or in vivo. The conjugates comprise a ligand that can bind to a transferrin receptor (TfR). Therapeutic uses for the conjugates are also provided. |
| 申请公布号 |
US9562230(B2) |
申请公布日期 |
2017.02.07 |
| 申请号 |
US201514801161 |
申请日期 |
2015.07.16 |
| 申请人 |
Arrowhead Pharmaceuticals, Inc. |
发明人 |
Chatterton Jon E.;Clark Abbot F. |
| 分类号 |
A61K48/00;C07H21/02;C07H21/04;C12N15/113;A61K9/00;A61K47/48;C12N15/87;A61K31/7088 |
主分类号 |
A61K48/00 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method of delivering an interfering RNA molecule to a patient, comprising administering to the patient an interfering RNA molecule-ligand conjugate wherein the ligand comprises fusion protein containing:
a) a transferrin (TfR)-specific antibody or antibody fragment and b) a transferrin (Tf) or a less than full length Tf that contains a TfR-binding domain. |
| 地址 |
Pasadena CA US |
