Plozasiran (API)
Research Application:
Plozasiran API is a synthetic small interfering RNA (siRNA) developed for the treatment of hypertriglyceridemia and related cardiovascular and metabolic disorders. It targets the APOC3 gene, which encodes apolipoprotein C-III, a key regulator of triglyceride metabolism. In research, Plozasiran is used to study RNAi-based lipid-lowering strategies, gene-silencing specificity, and long-acting treatments for conditions such as familial chylomicronemia syndrome (FCS) and mixed dyslipidemia.
Function:
Plozasiran functions by silencing APOC3 mRNA in the liver, leading to a reduction in apolipoprotein C-III levels. This promotes enhanced lipolysis and clearance of triglyceride-rich lipoproteins from the bloodstream. As an API, Plozasiran enables the development of long-acting therapies aimed at significantly lowering triglyceride levels and reducing the risk of pancreatitis and cardiovascular events in patients with severe or genetic lipid disorders.