Alnylam Pharmaceuticals develops RNAi gene-silencing medicines targeting the genetic causes of disease, with programs in TTR amyloidosis, acute hepatic porphyria and primary hyperoxaluria.
Alnylam Pharmaceuticals is the world's leading RNA interference (RNAi) therapeutics company, developing gene-silencing medicines that treat disease by addressing underlying genetic causes. Founded in 2002, it has spent more than two decades advancing RNAi science and employs more than 1,900 people globally. The company is headquartered in Cambridge, Massachusetts, with commercial operations spanning multiple continents.
The company's work spans the full arc of biopharmaceutical activity: discovery, development, biologics manufacturing and the commercialization of RNAi therapeutics. Its pipeline includes RNAi-based programs in transthyretin (TTR) amyloidosis, acute hepatic porphyria and primary hyperoxaluria, within a broader portfolio addressing both rare and common diseases. Technical domains cover RNAi therapeutics, gene silencing, genetic medicine and biopharmaceutical discovery and development.
Alnylam operates in the biotechnology, biopharmaceuticals, rare diseases and genetic medicine sectors. It describes its culture as award-winning, with a commitment to scientific excellence and innovation and an integrated approach across discovery, development, manufacturing and commercialization. The company positions its work as mission-driven, focused on transforming patients' lives.
