Clinical Research Directory

Inclusion Criteria: * Male or female participants aged 18 to 80 years diagnosed with ALS * ALS symptom onset within 24 months of Screening * Slow vital capacity \>50% * Clinical or electrodiagnostic evidence of lower motor neuron involvement * Not pregnant and not nursing * Willing and able to practice effective contraception * Able to tolerate lumbar puncture * If on approved therapies for the treatment of ALS during the course of the study, must be on a stable dose (at the Sponsor's discretion) Exclusion Criteria: * Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes * Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device * Prior exposure to stem cell or gene therapy products * Any contraindication to intrathecal drug administration * Abnormal laboratory values deemed clinically significant by the Investigator * Significant infection or known inflammatory process * Any sign and/or history of neurological conditions and other neuromuscular disorders that could affect the electrophysiological recordings. * An EEG that shows signs of abnormal electrical activity (e.g., epilepsy)