MTL-HBG

MTL-HBG is an investigational small activating RNA (saRNA) therapeutic developed by MiNA Therapeutics for the treatment of sickle cell disease and beta thalassemia. It is designed to increase transcription of the gamma globin gene (HBG), leading to elevated production of fetal hemoglobin (HbF) in patients' erythroid progenitor cells. Elevated HbF levels can compensate for defective adult hemoglobin, protecting against symptoms such as vaso-occlusive crises and organ damage in sickle cell disease. MTL-HBG is administered in vivo using a liposomal delivery system (NOV340), enabling targeted induction of HbF without the need for gene editing, harmful pre-conditioning, or complex cell engineering. Preclinical studies have demonstrated that MTL-HBG can induce high, pancellular levels of HbF—up to 62% as a proportion of total hemoglobin—in patient-derived cells, with durable and specific activity exceeding clinically protective thresholds[1][3][4][5].