A Multi-Center, Randomized, Double-Blind Placebo Controlled Multiple-Ascending Dose Study to Evaluate the Safety and Tolerability of QRL-201 in Amyotrophic Lateral Sclerosis

Minimum Age:

18 Years

Maximum Age:

80 Years

Inclusion Criteria:

- Male or female participants aged 18 to 80 years diagnosed with ALS

- Slow vital capacity >50%

- Clinical 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