Clinical Research Directory

Inclusion Criteria: * Diagnosis of ALS in accordance with the revised El Escorial criteria and TRICALS risk score * An overall disease duration of ≤ 2 years after the participant's first symptoms * No or low circulating anti-AAV9 antibodies (titre ≤ 1:50 * Stable dosing with a standard of care ALS medication (eg, riluzole and edaravone) and other prescription medications for 30 days prior to Screening * Not pregnant or breastfeeding, or willing to cease breastfeeding * All participants must use a barrier method of contraception Exclusion Criteria: * Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical history genetic testing * Any history of myocardial infarction or stroke within 6 months prior to Screening, or uncontrolled diabetes (HbA1C \> 9%) * Positive test for cytomegalovirus, hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody. * Inadequate organ function * Any participant with a current open tracheostomy