Study on the Safety and Efficacy of a Novel CAR-T Cell Therapy (CTX112) for Severe Autoimmune Diseases

Autoimmune diseases occur when the immune system mistakenly attacks the body’s own tissues. These include systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and idiopathic inflammatory myopathies (IIM). In these diseases, certain white blood cells—known as B cells—play a central role. They produce antibodies that trigger inflammation in organs such as the kidneys, skin, lungs, or muscles. If the immune system cannot be sufficiently suppressed with medication, the condition is referred to as refractory (therapy-resistant). This study is investigating the investigational drug CTX112, which is a so-called CAR-T cell therapy. Unlike conventional CAR-T cell therapies, in which the patient’s own immune cells are modified in the laboratory, CTX112 uses cells from healthy donors (allogeneic T cells). The donor cells are edited using the CRISPR-Cas9 “gene scissors” so that they specifically recognize the CD19 surface antigen on the patient’s disease-causing B cells and are designed to eliminate them, thereby “resetting” the immune system and alleviate disease symptoms in the long term.

The goal of this Phase 1 study is to investigate the safety and efficacy of the CTX112 CAR-T cell therapy. The study aims to determine the safe dose that can be administered and to identify any side effects (e.g., immune system reactions). Before treatment, patients receive chemotherapy (lymphodepletion), which reduces the number of the body’s own immune cells to make room for the new cells and temporarily suppress the body’s own immune system so that the CTX112 cells are not rejected. CTX112 is then administered as a single intravenous infusion. After treatment, patients are closely monitored so that any potential side effects can be treated immediately. The study also investigates how long the cells survive in the body and how disease activity (e.g., autoantibody levels, skin or organ changes) changes over time. Follow-up will continue for up to 5 years.

Eligible participants include women and men between the ages of 18 and 70 with a confirmed diagnosis of SLE (including lupus nephritis), systemic sclerosis (with active skin or lung involvement), or idiopathic inflammatory myopathy (with moderate to severe involvement) who have not responded to standard therapies. Excluded, among others, are patients with severe central nervous system disorders, severe infections, or a history of cell or gene therapy.