Alnylam preclinical data

In non-human primates, a single dose of 1 mg/kg subcutaneous ALN-TTRsc02 led to TTR knockdown of up to 99% with sustained knockdown of >50% for >84 days. Once-monthly 1 mg/kg ALN-TTRsc02 led to sustained TTR knockdown of up to 99% with mean maximal knockdown of >95%. Data were presented at the Oligonucleotide Therapeutics Society meeting in Leiden. Early next year, Alnylam plans to submit an IND for the subcutaneous formulation of an RNAi therapeutic targeting the TTR gene using the company’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform. Clinical data for ALN-TTRsc02 are slated for late 2016 and a Phase III trial is planned for 2017. ...