Pre-Clinical Data on RNAi Therapeutics Targeting Transthyretin (TTR)

Pre-Clinical Data on RNAi Therapeutics Targeting Transthyretin (TTR)

We have presented pre-clinical data with RNAi therapeutics targeting 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.  These data showed that the degree of TTR knockdown in a mouse disease model was highly correlated with regression of TTR tissue deposits.  Further, comparative studies performed with the TTR stabilizer tafamidis and a TTR-specific antisense oligonucleotide (ASO) showed RNAi therapeutics targeting TTR to have superior pharmacologic profiles.



These data suggest that the 80% TTR knockdown target level achieved in clinical studies with patisiran and ALN-TTRsc could facilitate a reduced pathogenic accumulation of TTR amyloid, and possibly even a regression of TTR amyloid, in patients with ATTR.  The comparative data demonstrate the superior activity of RNAi therapeutics over stabilizers toward regression of TTR deposits, and establish greater TTR knockdown with over 100-fold lower tissue exposure for RNAi therapeutics versus ASOs. Taken together, we believe that these new pre-clinical data highlight the potential for RNAi therapeutics targeting TTR to emerge as an optimal approach for the treatment of ATTR.



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