Safety and Tolerability of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

Inclusion Criteria: Patients who are judged by professional medical staff to still be able to carry out the clinical trial project cycle; 18 years old ≤ age ≤ 75 years old, males or females; ALS patients with confirmed SOD1 gene mutations document (known SOD1 mutation sites and related disease progression have been reported); Forced vital capacity ≥ 80% of predicted vital capacity during the screening period; Diagnosis of confirmed or probable familial or sporadic ALS in accordance with the revised EI Escorial diagnostic criteria for amyotrophic lateral sclerosis of the World Federation of Neurology; The patient or patient's legal representative clearly understands and voluntarily participates in the study and signs the informed consent form; Subjects (including male subjects) are willing to have no birth plan and voluntarily take effective contraceptive measures during the entire study period and within 3 months after the end of the study, and have no plan to donate sperm or eggs. Exclusion Criteria: Patients with SOD1 mutations occurring at nucleotides 44 to 66 (calculated from the start of SOD1 protein translation), patients with P.F21C mutation; Patients who have previously received or are currently receiving Tofersen treatment; HIV test positive or history of positive tests; Positive hepatitis C virus antibody or history of positive tests; Active hepatitis B infection (positive hepatitis B surface antigen and/or positive hepatitis B core antibody); Have used other investigational drugs within 1 month or within 5 drug half-lives; Diseases and deformities of the lumbar spine; Have other conditions known to be associated with motor neuron dysfunction that may confuse or obscure an ALS diagnosis; Other psychiatric disorders diagnosed according to DSM-V diagnostic criteria, or significant suicide intent; With severe hepatic insufficiency, renal insufficiency or severe cardiac insufficiency (severe hepatic insufficiency refers to ALT value≥2.0 times the upper limit of normal value or AST value≥2.0 times the upper limit of normal value; severe renal insufficiency refers to CRE≥1.5 times the upper limit of normal value or eGFR<40mL/min/1.73m2; severe cardiac insufficiency refers to NYHA class 3-4); Permanently dependent on ventilator-assisted ventilation; History of alcohol and drug abuse; Patients who are pregnant, breast-feeding, or who are likely to become pregnant and plan to become pregnant; Patients participating in other clinical trials or using other biological agents, drugs or devices under investigation; Patients who have received any vaccinations within 28 days; Contraindications to MRI (eg, claustrophobia); Unable to be cooperative and complete the follow-up due to other reasons.