CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, presented new pre-clinical data from its ALN-TTR program at the XII International Symposium on Amyloidosis in Rome on April 18 - 21, 2010. ALN-TTR01 is a systemically delivered RNAi therapeutic being developed for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR), including familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy (FAC). These new pre-clinical data demonstrate – for the first time – that treatment with an RNAi therapeutic can result in regression of pre-existing pathogenic TTR deposits in peripheral tissues. Additional pre-clinical data presented at the meeting demonstrated the potential application of TTR-specific siRNA for the treatment of ocular disease in ATTR.
