23 Jul, 2011 ALN-TTR Data Presented at Peripheral Nerve Society Meeting
Our flagship effort from the Alnylam 5×15 strategy is our ALN-TTR program, which is currently enrolling patients in a Phase I study for the treatment of a disease called transthyretin-mediated amyloidosis (ATTR).
In June 2011, we reported results from a companion natural history study designed to measure blood levels of wild-type and mutant transthyretin (TTR) over time in both ATTR patients and gene carriers. Results, which are informative in our overall efforts to develop our RNAi therapeutic for this devastating disease, showed that TTR levels were stable over time in both patient groups. Pre-clinical data from our ALN-TTR program was also presented at the meeting, including new data demonstrating improved potency with ALN-TTR01 using a loading dose/maintenance dose regimen.