RN1201 Injection for Autoimmune Diseases Refractory to Standard Therapies

Inclusion criteria

• Voluntary signed informed consent demonstrating understanding of the study and willingness/ability to comply with all trial procedures.
• Age ≥18 years; both sexes eligible.
• Documented diagnosis of an autoimmune disease for ≥6 months at screening, including but not limited to immune thrombocytopenia (ITP), autoimmune hemolytic anemia (AIHA), systemic lupus erythematosus (SLE), immune-mediated necrotizing myopathy (IMNM), neuromyelitis optica spectrum disorder (NMOSD), multiple sclerosis (MS), myasthenia gravis (MG), etc.
• Standard-of-care therapy for ≥8 weeks before screening without achieving complete remission or adequate disease control, with stable dose for >2 weeks.
• Subjects on corticosteroid monotherapy at screening must be receiving ≥7.5 mg/day prednisone (or equivalent).
• Disease activity score meeting criteria for moderate-to-severe active disease.
• Adequate bone-marrow reserve, coagulation, cardiopulmonary, hepatic, and renal function.
• Agreement to use effective contraception for 24 months after study enrollment.

Exclusion criteria

Subjects meeting any of the following cannot be enrolled:

• Known hypersensitivity, allergy, intolerance, or contraindication to RN1201 or any study drug component (fludarabine, cyclophosphamide, tocilizumab) or history of severe allergic reactions.
• Severe cardiovascular disease or organ failure.
• Active or uncontrolled infection requiring IV antibiotics or evidence of severe active infection.
• Significant bleeding tendency (e.g., Gastrointestinal bleeding, coagulopathy, hypersplenism).
• Hepatitis C virus, HIV, or syphilis infection.
• History of epilepsy or severe neurological disorders/pathology not attributable to autoimmune disease.
• Malignancy within 2 years before screening, except adequately treated carcinoma in situ of skin, cervix, or lung or other non-active tumors.
• Prior CAR-T therapy or other genetically modified T-cell therapy.
• Prednisone (or equivalent) ≥100 mg/day for ≥14 days within 4 weeks before screening.
• Pregnancy, lactation, or planned pregnancy within 2 years.
• Any condition that, in the investigator's judgment, may increase subject risk or interfere with study results.