NBTs targeting C9ORF72-ALS include IONIS-C9Rx, a PS 2MOE ASO, designed to target mutant C9ORF72 RNA that is currently in clinical trials for C9ORF72-ALS conducted by Ionis/Biogen (Table 2) (IONIS, 2020). This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.