RG0401

RG0401 is an investigational HLA gene-editing therapy being developed by RheumaGen for the treatment of refractory or treatment-resistant rheumatoid arthritis. The therapy uses precision editing of DNA markers within the HLA gene in patient-derived hematopoietic stem cells, aiming to convert harmful HLA alleles into healthy ones. This process is designed to make a patient's HLA molecules resemble those found in individuals resistant to rheumatoid arthritis, thereby preventing T cell activation and halting chronic autoimmune responses that drive disease progression. The approach seeks to provide a one-time, potentially curative treatment by addressing the root cause of autoimmunity while preserving normal immune function. As of early 2025, RG0401 is in preclinical (IND-enabling) studies with plans for Phase 1 clinical trials targeting 10–20% of RA patients who are refractory to existing therapies. RheumaGen also has additional programs under development for other autoimmune diseases where HLA alleles play a critical role[1][2][3][4][5][8].