Mouse and human genetic data indicated that lowering transmembrane serine protease 6 (TMPRSS6) expression could up-regulate hepcidin and ameliorate many of the disease symptoms associated with beta-thalassemia.25–27 A phase 2a, randomized, open-label study to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of ISIS 702843 administered subcutaneously to patients with NTDT is ongoing (Ionis Pharmaceuticals, Inc., NCT04059406). This evidence concerns the gene TMPRSS6 and beta thalassemia.