Visit Capella to learn more about ALN-TMP, our RNAi therapeutic for the treatment of hemoglobinopathies.
Beta-thalassemia and Iron-overload disorders
ALN-TMP comprises a systemically delivered RNAi therapeutic targeting transmembrane protease, serine 6 (Tmprss6) for the treatment of beta-thalassemia and iron-overload disorders. Tmprss6, a genetically validated target expressed on hepatocytes, functions by cleaving hemojuvelin, resulting in elevated hepcidin levels and restricted iron mobilization.
In pre-clinical studies, presented at the American Society of Hematology (ASH) Annual Meeting in December 2012, scientists showed that ALN-TMP leads to disease modifying effects in models of ß-thalassemia, including amelioration of anemia, iron overload, extra-medullary hematopoiesis, and ineffective erythropoiesis, in addition to correction of globin gene expression. The company expects to partner this program prior to conducting a Phase I clinical study.