Our Science Is Changing the Way Medicine Treats DiseaseTM
Alnylam has led the translation of RNAi (RNA interference) from Nobel Prize-winning discovery into an innovative, entirely new class of medicines. Founded in 2002 by a team of distinguished life sciences leaders, Alnylam’s vision is to harness the potential of RNAi therapeutics to transform the lives of people living with diseases for which there are limited or inadequate treatment options. Our pioneering work has delivered the world’s first and only approved RNAi therapeutics—ONPATTRO® (patisiran) in 2018, GIVLAARI® (givosiran) in 2019, and OXLUMO® (lumasiran) in 2020. We are advancing a deep pipeline of innovative RNAi-based medicines in four therapeutic areas: genetic medicines, cardio-metabolic diseases, infectious diseases, and central nervous system (CNS) and ocular diseases.
What’s in a Name?
AL-NY-LAM. Our name may not be the easiest to pronounce, but once you learn it, you’ll never forget it. Alnylam is derived from “Alnilam,” the bright center star in the constellation Orion’s belt, which has been used by navigators for thousands of years and symbolizes our passion for discovery.
RNAi Therapeutics—An Innovative New Class of Medicines
Medicines based on RNAi work by “silencing” or disabling the production (“expression”) of the genes that cause specific diseases. In doing so, RNAi therapeutics work “upstream” of most other classes of medicines, such as small molecules and monoclonal antibodies, by targeting the “root” genetic cause of a disease rather than its symptoms. To learn more about our science, click here.
RNAi is the core discovery that forms the therapies Alnylam is developing. It is recognized as a major scientific breakthrough—but how does it work, exactly?