公开(公告)号：WO2023059629A1

公开(公告)日：2023-04-13

申请号：PCT/US2022/045664

申请日：2022-10-04

Applicant: AMGEN INC.

Inventor： LU, Jiamiao ,  OLLMANN, Michael ,  COLLINS, Patrick ,  LI, Chi-Ming ,  WANG, Songli

IPC: C12N15/113 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/3515 ,  C12Y203/01051 ,  C12Y306/05002

Abstract: The present invention relates to compositions and methods for enhancing the gene silencing activity of oligonucleotide compounds. In particular, the invention relates to inhibiting the expression or activity of suppressor proteins, such as RAB18, ZW10, STX18, SCFD2, NAPG, SAMD4B, or VPS37A, to increase the efficacy of ligand-conjugated oligonucleotide compounds in reducing the expression of target genes in a cell.

公开(公告)号：WO2021247885A2

公开(公告)日：2021-12-09

申请号：PCT/US2021/035730

申请日：2021-06-03

Applicant: AMGEN INC.

Inventor： LIN, Daniel C. H. ,  OLLMANN, Michael ,  MURRAY, Justin K. ,  HERBERICH, Bradley J. ,  DAS, Amrita ,  COLLINS, Patrick ,  HOMANN, Oliver

IPC: C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3521 ,  C12N2310/3523 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12Y101/00

Abstract: The present invention relates to RNAi constructs for reducing expression of the HSD17B13 gene. Methods of using such RNAi constructs to treat or prevent liver disease, nonalcoholic fatty liver disease (NAFLD) are also described.