Editor’s Note: This interview was conducted at #Bio2018. Please pardon the background noise.
Barry Greene, President of Alnylam, provides an overview of his company and the science of RNA interference (RNAi). Alnylam is developing RNAi (RNA interference) into a new class of medicines to potentially address the needs of patients who have limited or inadequate treatment options. Alnylam’s pipeline of investigational RNAi therapeutics is focused on diseases with unmet medical need that fall under 4 Strategic Therapeutic Areas (STArs): genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS) diseases.
– Patisiran is an investigational RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of hereditary ATTR amyloidosis (hATTR amyloidosis). Patisiran is the only investigational medicine in Alnylam’s pipeline that utilizes a lipid nanoparticle (LNP) formulation and is administered via intravenous infusion.
– Inclisiran (ALN-PCSsc) is a subcutaneously administered, investigational RNAi therapeutic targeting proprotein convertase subtilisin kexin type 9 (PCSK9) in development for the treatment of hypercholesterolemia. Inclisiran utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.
– Givosiran (ALN-AS1) is a subcutaneously administered, investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHPs), including acute intermittent porphyria (AIP), variegate porphyria (VP), and hereditary coproporphyria (HCP).