One phase 1/2 clinical trial starting in 2024 (NCT06100276) and projected to end in 2027 is examining the safety, tolerability, and efficacy of AMT-162, a gene therapy that utilizes adeno-associated viral vector serotype 5 (AAV9) to deliver miRNA, which silences the SOD1 gene, in patients with ALS with the SOD1 mutation. This evidence concerns the gene SOD1 and amyotrophic lateral sclerosis.