New Pre-Clinical Data Presented from our ALN-AS1 Program for the Treatment of Acute Intermittent Porphyria

New Pre-Clinical Data Presented from our ALN-AS1 Program for the Treatment of Acute Intermittent Porphyria

We presented pre-clinical proof-of-concept data from our ALN-AS1 program for the treatment of acute intermittent porphyria (AIP) at the International Congress of Porphyrins and Porphyrias being held May 16 – 18, 2013. The research, presented by Alnylam scientists and collaborators at the Icahn School of Medicine at Mount Sinai in New York City, showed that administration of a GalNAc-conjugated siRNA targeting aminolevulinate synthase-1 (ALAS-1) blocked production of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic precursors that cause disease symptoms and pathophysiology, in a mouse model of AIP.



This effect was shown to be dose-responsive and durable, with administration of a single dose resulting in ALA and PBG reduction that lasted for at least two weeks. In addition, our collaborators presented preliminary comparative data showing that ALAS-1 siRNA administration was more effective than heme administration in treating an acute porphyria attack. We are excited by these new data, as they provide support for the clinical advancement of an RNAi therapeutic targeting ALAS-1 to treat patients that suffer from this debilitating disease.  ALN-AS1 is a key program in our “Alnylam 5×15” product development strategy, aimed at bringing innovative medicines to patients with a core focus on RNAi therapeutics toward genetically defined targets for the treatment of life-threatening diseases with a high unmet need.



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