With only a passing SBIR involvement when the firm was very young, Alnylam Pharmaceuticals, Inc. has long since outgrown SBIR eligibility. The firm is a biopharmaceutical company organized around RNA interference (RNAi) therapeutics andhaving deeloped a pipeline of investigational RNAi therapeutics focus on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system/ocular diseases. Providing ONPATTRO (patisiran), a lipid complex injection for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, the company's development programs include Vutrisiran, an investigational RNAi therapeutic targeting transthyretin that is in Phase III trials for the treatment of transthyretin-mediated amyloidosis; Givosiran that is in Phase III trials to treat acute hepatic porphyrias; and Lumasiran, an investigational RNAi therapeutic, which is in Phase III clinical trials for glycolate oxidase to treat primary hyperoxaluria type 1. It is also developing Inclisiran, an investigational RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9 that is in Phase III clinical trials for the treatment of hypercholesterolemia; and Fitusiran, an investigational RNAi therapeutic that is in Phase III clinical trials for treating hemophilia A and B with or without inhibitors. In addition, the company engages in the development of Cemdisiran for complement-mediated diseases; ALN-AAT02 to treat alpha-1 anti-trypsin deficiency-associated liver disease; ALN-HBV02 for treating chronic hepatitis B virus infection; and other earlier-stage programs.
