Eplontersen

Eplontersen, a triantennary N-acetyl galactosamine (GalNAc3-7a) -conjugated antisense oligonucleotide, targets transthyretin (TTR) mRNA to suppress the synthesis of both variant and wild-type TTR proteins. The accumulation of misfolded TTR proteins contributes to the formation of amyloid fibrils in the heart and peripheral nerves, resulting in amyloid TTR (ATTR) amyloidosis [1] [2] [3].