RPT-2950

RPT-2950 is a potent, selective, and orally bioavailable small molecule inhibitor of the Long Interspersed Nuclear Element-1 (LINE-1) reverse transcriptase (RT), also known as ORF2p. Developed by ROME Therapeutics, the drug is designed to treat autoimmune diseases, specifically systemic lupus erythematosus (SLE), by targeting the 'dark genome.' In patients with certain autoimmune conditions, the accumulation of LINE-1-derived nucleic acids in the cytoplasm triggers innate immune sensors such as the cGAS-STING pathway, leading to a chronic and damaging type I interferon response. By inhibiting the reverse transcription of LINE-1 RNA into DNA, RPT-2950 aims to block this inflammatory signaling cascade at its source. The drug is currently in preclinical development, undergoing IND-enabling studies.