RTM-1000

RTM-1000 is an orally bioavailable, brain-penetrant small molecule inhibitor of cyclic GMP-AMP synthase (cGAS) developed by Riltide Medicines. By targeting cGAS, the most upstream node of the cGAS-STING pathway, RTM-1000 blocks downstream STING-driven inflammatory cascades, including Type I interferon (IFN) production and NF-κB-mediated pro-inflammatory cytokine expression. In preclinical studies, RTM-1000 demonstrated cross-species activity against both human and mouse cGAS, excellent blood-brain barrier penetration (with a rat unbound brain-to-plasma ratio of approximately 40%), and dose-dependent suppression of interferon-stimulated gene (ISG) expression in brain tissue. It is being developed for the treatment of systemic and central nervous system (CNS) autoimmune diseases, such as systemic lupus erythematosus (SLE) and systemic sclerosis (SSc).