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Refractory Anemia
ALN-HPN: Refractory Anemia
ALN-HPN is a systemically delivered RNAi therapeutic targeting the hepcidin pathway via transferrin receptor subtype 2 (TFR2) for the treatment of refractory anemia. Pre-clinical data, presented at the 2011 merican Society of Hematology (ASH) annual meeting, were presented documenting TFR2 as the ideal target for hepcidin pathway antagonism and demonstrating efficacy in anemia models. Alnylam plans to partner its ALN-HPN program prior to initiating a Phase I study.
ALN-HPN Clinical Timeline
Anemias of chronic disease (ACD) occur across multiple populations, including end-stage renal disease (ESRD), cancer, and chronic inflammatory disorders. There are also additional genetic causes, such as iron-refractory iron deficiency anemia (IRIDA). In the U.S., there are ~500,000 patients with ESRD and ~50,000 with refractory anemia.
