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Alnylam Pharmaceuticals (ALNY) Presents New Pre-Clinical Data On Rnai Therapeutics Targeting Transthyretin (TTR) For The Treatment Of TTR-Mediated Amyloidosis (ATTR)


4/29/2014 9:50:49 AM

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the presentation of new pre-clinical data on RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). These data were presented at the International Symposium on Amyloidosis (ISA) held April 27 – May 1, 2014 in Indianapolis, Indiana. In a poster titled “Preclinical Evaluation of RNAi Therapeutics for the Treatment of ATTR: An Update,” Alnylam scientists presented data confirming that the degree of TTR knockdown in a mouse disease model was highly correlated with regression of TTR tissue deposits. Further, comparative studies were performed with the TTR stabilizer tafamidis and a TTR-specific antisense oligonucleotide (ASO). In these pre-clinical studies, RNAi therapeutics targeting TTR were shown to have superior pharmacologic profiles.

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