NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week unveiled new Phase II data on its transthyretin-mediated amyloidosis (ATTR) therapy patisiran, which showed that the drug is capable of knocking down its disease-causing protein target by about 80 percent in patients — in line with previous clinical findings.
Release of the findings come as the company moves ahead with a recently initiated Phase III trial of patisiran in patients with a manifestation of ATTR known as familial amyloidotic polyneuropathy that is characterized by damage to the peripheral nervous system.