Escaping the liver

Having addressed one of the two big problems of RNAi delivery - getting therapeutic levels of the molecules inside cells - Arrowhead Research Corp. now believes it has solved the second, with a modification to its technology that enables targeting beyond the liver. The company has unveiled its newest candidate, ARC-HIF2, which targets cells in the kidney and is in preclinical studies for clear cell renal cell carcinoma (ccRCC).

Arrowhead's Dynamic Polyconjugate (DPC) platform solves the first problem, and allows RNAi cargo to be released into cells following endocytosis, by simultaneously delivering RNAi and a lytic polymer that breaks open the endosome vesicles. But until now, the platform had only been used for diseases of the liver...