基本信息:
- 专利标题： MUCIN 5B (MUC5B) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
- 申请号：PCT/US2021/056131 申请日：2021-10-22
- 公开(公告)号：WO2022087329A8 公开(公告)日：2022-04-28
- 发明人： NOETZLI, Leila , PARKER, Margaret , MCININCH, James, D. , SCHLEGEL, Mark, K. , CASTORENO, Adam , ZUBER, Jeffrey
- 申请人： ALNYLAM PHARMACEUTICALS, INC.
- 申请人地址： 675 West Kendall Street
- 专利权人： ALNYLAM PHARMACEUTICALS, INC.
- 当前专利权人： ALNYLAM PHARMACEUTICALS, INC.
- 当前专利权人地址： 675 West Kendall Street
- 代理机构： ZACHARAKIS, Maria, Laccotripe et al.
- 优先权： US63/104,557 2020-10-23
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; C12N2310/14 ; C12N2310/312 ; C12N2310/315 ; C12N2310/343 ; C12N2310/346 ; C12N2310/3515 ; C12N2320/11
摘要：
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the mucin 5B (MUC5B) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a MUC5B gene and to methods of treating or preventing a MUC5B-associated disease, such as a lung disease, e.g., cystic fibrosis, chronic obstructive pulmonary disease (COPD), and/or pulmonary fibrosis, e.g., idiopathic pulmonary fibrosis (IPF), in a subject.