Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company that specializes in the development of RNA interference (RNAi) therapeutics. The company is based in Cambridge, Massachusetts and was founded in 2002. Alnylam's mission is to develop innovative RNAi therapeutics that target a wide range of diseases, including rare genetic diseases, cardio-metabolic diseases, and infectious diseases.
RNAi is a biological process in which RNA molecules inhibit gene expression or translation by neutralizing targeted mRNA molecules. Alnylam has developed a platform technology for the discovery and development of RNAi therapeutics, which has been validated in numerous clinical trials. The company's lead product, ONPATTRO (patisiran), was approved by the US FDA in 2018 for the treatment of hereditary transthyretin-mediated amyloidosis, a rare genetic disease.
Alnylam has a strong pipeline of RNAi therapeutics in development for a variety of diseases. The company is focused on advancing its pipeline and bringing innovative RNAi therapeutics to market. Alnylam has partnerships with several leading pharmaceutical companies, including Sanofi, Regeneron, and Novartis, which provide funding and resources to support its research and development efforts. With a strong focus on RNAi technology and a commitment to developing innovative therapies for a wide range of diseases, Alnylam is well-positioned to make a significant impact in the biopharmaceutical industry in the years ahead.