From a safety perspective, among the 20 patients randomised to receive ALN-APP or placebo, one subject experienced two mild AEs deemed related to both the study drug and procedure, but no serious AEs or deaths were reported.19 These interim results for ALN-APP represent the first-in-human translation of an RNA interference therapeutic for neurodegenerative diseases, with the ongoing ALN-HTT02 clinical trial in HD expected to improve understanding of the approach and potential therapeutic benefits of this strategy. Here, APP is linked to Huntington disease.