Arrowhead Pharmaceuticals, Inc.

Bo Chen

Zhen Li

So Wong

Jeffrey Carlson

Rui Zhu

Anthony Nicholas

Xiaokai Li

Matthew Fowler-Watters

Erich Altenhofer

Dongxu Shu

The present disclosure relates to RNAi agents, for example, double stranded RNAi agents, able to inhibit HIF-2 alpha (EPAS1) gene expression. Also disclosed are pharmaceutical compositions that include HIF-2 alpha RNAi agents and methods of use thereof. The HIF-2 alpha RNAi agents disclosed herein may be linked or conjugated to targeting ligands (such as compounds that have affinity for integrins, including alpha-v-beta-3 and alpha-v-beta-5 integrins) and pharmacokinetic (PK) enhancers, to facilitate the delivery to cells and tissues, including to clear cell renal cell carcinoma (ccRCC) cells and tumors. Delivery of compositions comprising the HIF-2 alpha RNAi agents in vivo provides for inhibition of HIF-2 alpha gene expression. The HIF-2 alpha RNAi agents can be used in methods of treatment of various diseases and disorders, including ccRCC.

RNAi agents for inhibiting expression of HIF-2 alpha (), compositions thereof, and methods of use

Growing community of inventors

Scarsdale, NY, United States of America

Bo Chen