Phase I Clinical Study for ALN-TTRsc

Phase I Clinical Study for ALN-TTRsc

We initiated dosing in our Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).  The Phase I trial of ALN-TTRsc is being conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects.  The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc.

Secondary objectives of the study include assessment of clinical activity of the drug as measured by serum TTR levels.  We expect to present data from this trial in mid-2013.

ALN-TTRsc, which is being developed for the treatment of familial amyloidotic cardiomyopathy (FAC), is a subcutaneously administered RNAi therapeutic that comprises an siRNA conjugated to a GalNAc ligand that enables receptor-mediated delivery to the liver.  ALN-TTRsc is the first GalNAc-siRNA, and the first subcutaneously delivered, systemic RNAi therapeutic to enter clinical development stages.


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