Hemoglobinopathies

ALN-TMP, our fifth Alnylam 5x15 program, comprises a systemically delivered RNAi therapeutic targeting transmembrane protease, serine 6 (Tmprss6) for the treatment of hemoglobinopathies, including beta-thalassemia and sickle cell anemia. Hemoglobinopathies are associated with chronic anemia, extra-medullary hematopoiesis, and iron overload. Tmprss6, a genetically validated target expressed on hepatocytes, functions by cleaving hemojuvelin, resulting in elevated hepcidin levels and restricted iron mobilization.

In pre-clinical animal model studies, presented at the American Society of Hematology Annual Meeting in December 2011, scientists showed that ALN-TMP demonstrated corrective effects on iron overload in addition to broader disease modifying effects including improvements in hemoglobin levels and spleen histopathology. The company expects to partner this program prior to conducting a Phase I clinical study.