First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement
RNA, Small Interfering
Vascular Endothelial Growth Factor A
The fi ndings in this report show safety, pharmacokinetics, RNAi mechanism of action, and clinical activity with a novel fi rst-in-class LNP-formulated RNAi therapeutic in patients with cancer. The ability to harness RNAi to facilitate specifi c multitargeting, as well as increase the number of druggable targets, has important implications for future drug development in oncology.