In vitro, Rovanersen (WVE-120101) leads to the selective RNase H cleavage of mutant Huntingtin allele (mHTT) over wtHTT, with no complement activation. Rovanersen selectively decreases mHTT mRNA and protein levels compared with wtHTT in multiple cells lines[1].

In non-human primates (NHPs), Rovanersen (WVE-120101) is found in nuclear and perinuclear compartments of neurons in brain areas believed to be involved in HD pathology[1].