Drug Type: RNA-Targeted Therapy
Conditions: ALS, FTD
Mechanism Type: Neuroprotection
Mechanism: IONIS-SOD1Rx is an antisense oligonucleotide specifically designed to inhibit production of C9orf72 repeat-rich RNAs. C9orf72 ALS is the most common genetic form of the disease.
U.S. Status for ALS: Phase I
 A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis ClinicalTrials.gov, 19 Nov 2018. Accessed 25 Jan 2019 from https://clinicaltrials.gov/ct2/show/NCT03626012.
 Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs. Jiang, J et al.
Neuron. 2016 May 4;90(3):535-50.