Programs
Curing Autoimmune Diseases at Their Source
RheumaGen is advancing a new class of HLA-gene editing therapies designed to cure autoimmune diseases at their source. The company is focused on editing the HLA, or “immune gene,” so that the immune system does not attack healthy cells.
Our Pipeline
Our Programs
RG0401 for Rheumatoid Arthritis
RG0401, RheumaGen’s lead program, targets 10-20% of RA patients who are refractory or treatment-resistant and suffer substantial unmet need. The company is currently conducting RG0401 IND-enabling studies and plans to begin the Phase I clinical trial in 2026. RG0401 aims to cure refractory RA by treating its HLA source. With a precision edit to a DNA marker(s) of the HLA gene, RG0401 is designed only to make a patient’s HLA molecules mirror those of a person resistant to RA, while maintaining the rest of the immune system as normal. This therapy changes harmful HLA alleles to healthy ones and prevents T cells from activating chronic autoimmune responses, thereby halting not only cell and tissue degradation but also the entire disease process with this approach.
Additional Programs
RheumaGen is building a pipeline of treatments to address autoimmune diseases in which HLA alleles play a critical epidemiological role, including programs for multiple sclerosis, type 1 diabetes, and ankylosing spondylitis currently in development.