As you will remember (SNALP Works!), the RNAi Therapeutics candidate ALN-TTR01 last November showed a mean reduction of 41% of serum TTR from baseline following a single 1.0mg/kg infusion. In light of yesterday’s results with ALN-PCS02 where a supposedly less potent RNAi trigger was employed and a ~60% knockdown was achieved with 0.25mg/kg, it seems likely that the Tekmira SNALP-enabled ALN-TTR02 which is about to enter clinical development and which employs a formulation similar to PCS02 will show the type of 70-80% knockdown at 0.25mg/kg.
We have yet to learn more about the safety profile of ISIS-TTRRx which is being developed under an option agreement with GSK.
As you know, these days I’m strongly advocating RNAi Therapeutics as the technology is currently being sold at much below the worth of the science which has progressed steadily over the years despite the up and downs in public perceptions. Today’s results by