A Study Evaluating the Safety and Tolerability of QRL-201 in ALS

The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS

Inclusion Criteria:Male or female participants aged 18 to 80 years diagnosed with ALSSlow vital capacity >50%Clinical evidence of lower motor neuron involvementNot pregnant and not nursingWilling and able to practice effective contraceptionAble to tolerate lumbar punctureIf 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) genesCurrently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or devicePrior exposure to stem cell or gene therapy productsAny contraindication to intrathecal drug administrationAbnormal laboratory values deemed clinically significant by the InvestigatorSignificant infection, or known inflammatory process