RNA-144101

RNA-144101 is a small molecule drug that acts as an inhibitor (antagonist) of toll-like receptor 3 (TLR3), a protein involved in the innate immune response. TLR3 is expressed on retinal pigment epithelium (RPE) cells and binds to double-stranded RNA, triggering immune signaling pathways that can lead to RPE cell death. Increased activation of TLR3 has been observed in patients with geographic atrophy, an advanced form of dry age-related macular degeneration (AMD). By inhibiting TLR3, RNA-144101 aims to prevent RPE apoptosis and potentially slow or halt the progression of geographic atrophy. The drug was developed by researchers at the University of Kentucky and entered phase 1 clinical trials for this indication but development was discontinued before completion[1][3][4].