For example, tominersen, a 2′-MOE ASO to target huntingtin for Huntington’s disease (ClinicalTrials.gov Identifier: NCT02519036; NCT03761849) (Tabrizi et al., 2019) showed no serious adverse effects in phase 2 trials, but phase 3 trials were stopped due to lack of efficacy. Here, HTT is linked to juvenile Huntington disease.