Alnylam pioneered RNA interference (RNAi) into real medicines — tiny strands of RNA that switch off a disease-causing gene before its protein is ever made. Its lead drug Amvuttra now treats the heart and nerve damage of ATTR amyloidosis and is driving the company past $1B in quarterly sales into sustained profit. It also earns royalties on Leqvio (a cholesterol drug it invented and licensed to Novartis) and partners with Roche and Regeneron to push RNAi into common diseases like hypertension.
From RNA strand to patient
Alnylam designs a short RNA strand that silences a disease gene, has the strand (API) made by Agilent and finished in its own Norton, MA plant, then ships the medicine through specialty distributors and payers to the cardiologists and rare-disease centres who treat patients.