Alnylam announces proof-of-concept data from RNAi program Alnylam announced that it has presented pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 for the treatment of porphyria including acute intermittent porphyria. "The new research results support the advancement of RNAi therapeutics as a promising strategy for the prevention and/or treatment of acute attacks in patients with AIP," the company said. It added, "In particular, a prototype GalNAc-siRNA targeting ALAS-1 was shown to be effective in blocking ALA and PBG production in both prophylactic and treatment models of AIP. The company is on track to designate a GalNAc-siRNA development candidate, ALN-AS1, in late 2013 resulting in an investigational new drug (IND) filing in 2014."
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