公开(公告)号：WO2022182574A1

公开(公告)日：2022-09-01

申请号：PCT/US2022/016890

申请日：2022-02-18

申请人： ALNYLAM PHARMACEUTICALS, INC. ,  NOETZLI, Leila ,  MCININCH, James, D. ,  TREMBLAY, Frederic ,  SCHLEGEL, Mark, K. ,  CASTORENO, Adam

发明人： NOETZLI, Leila ,  MCININCH, James, D. ,  TREMBLAY, Frederic ,  SCHLEGEL, Mark, K. ,  CASTORENO, Adam

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P3/06 ,  A61P3/08 ,  A61P13/12

摘要： The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a KHK gene and to methods of treating or preventing a KHK-associated disorder in a subject.