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Inclusion Criteria (Double-Blind Periods):

* 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 (Double Blind Periods):

* 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)

Inclusion Criteria (Open-Label Extended Dosing Period):

* Male or female participants in the sporadic ALS and C9orf72 ALS Cohorts of QRL-201-01 who have completed through at least Day 253 of safety follow up in the double-blind study period OR have completed Cohort 1 of Cohort 2
* Not pregnant and not nursing
* Willing and able to practice effective contraception
* Can visit study site for required visits
* 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 (Open-Label Extended Dosing Period):

* Currently enrolled in any other clinical study involving either an investigational product (IP) or off label use of a drug or device
* Off-label use of drugs or devices that are being used as disease-modifying therapies in ALS
* Participated in another clinical study within 30 days, or 5 half-lives of the previous investigational product, whichever is greater, prior to the first dose
* Prior exposure to stem cell or gene therapy products
* Is not suitable for inclusion in the Open-Label Extended Dosing period of the study, in the opinion of the Investigator
* An EEG that shows signs of abnormal electrical activity (NOTE: Cohort 1 and Cohort 2 participants only)

• Leuven, Belgium
• Calgary, Alberta, Canada
• Edmonton, Alberta, Canada
• Toronto, Ontario, Canada
• Montreal, Quebec, Canada
• Montreal, Quebec, Canada
• Bonn, North Rhine-Westphalia, Germany
• Berlin, Germany
• Lübeck, Germany
• Ulm, Germany
• Dublin, Ireland
• Utrecht, Netherlands
• Sheffield, United Kingdom, United Kingdom
• London, United Kingdom
• London, United Kingdom