Drug Type: Gene Therapy
Mechanism Type: SOD1-Lowering Therapy
Mechanism: IONIS-SOD1Rx is a generation 2.0 antisense drug specifically designed to inhibit production of mutant superoxide dismutase (SOD1). SOD1 mutations account for approximately 20% of familial ALS cases.
U.S. Status for ALS: Phase I
 Single and Multiple Dose Study of BIIB067 (Isis-SOD1Rx) in Adults With Amyotrophic Lateral Sclerosis (ALS). ClinicalTrials.gov, 1 Feb 2016. Accessed 9 Mar 2016 from https://clinicaltrials.gov/ct2/show/NCT02623699.
 SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy. Winer, L. et al. JAMA Neurol. 2013 Feb;70(2):201-7.
 An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Miller, TM et al. Lancet Neurol. 2013 May;12(5):435-42.
Last updated June 21st, 2017