FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS

Key Inclusion Criteria: * Adults aged 18 to 75 years * Diagnosis of ALS (clinically definite, clinically probable, or clinically probable laboratory supported) * ALS symptoms began within the past 24 months * Able to perform breathing tests: slow vital capacity (SVC) with consistent results, with breathing capacity of at least 60% of the expected value * Able and willing to meet all study requirements, including travel to the study site, brain magnetic resonance imaging (MRI) scans, lumbar punctures, and blood draws * Able and willing to use wearable sensors and complete speech assessments at home * On a stable dose of approved ALS medication for at least 4 weeks prior to screening * Capable of providing informed consent Key Exclusion Criteria: * Carries a confirmed SOD1 or FUS gene mutation * Has a tracheostomy or requires continuous assisted ventilation more than 22 hours per day during the preceding 3 months before the first Screening Visit * Has a contraindication to brain MRI (e.g., pacemaker, metal implants) * Has a contraindication to lumbar puncture or spinal injection (e.g., blood clotting disorders, certain blood thinners, signs of increased pressure in the brain) * Has significant abnormal liver, kidney, or blood test results * Is currently enrolled in another clinical trial or has received an investigational treatment within the past 4 weeks * Has previously received gene therapy, stem cell therapy, or another Antisense oligonucleotide (ASO) treatment * Has a c…