sefafaxersen

Sefafaxersen is an investigational ligand-conjugated antisense (LICA) oligonucleotide designed to target and reduce the production of complement component 3 (C3). Developed by Ionis Pharmaceuticals in collaboration with Roche, the drug utilizes Ionis' proprietary LICA technology, which conjugates the antisense molecule to N-acetylgalactosamine (GalNAc) to ensure highly specific delivery to hepatocytes in the liver, the primary site of C3 synthesis. By binding to C3 messenger RNA (mRNA) and promoting its degradation, sefafaxersen inhibits the synthesis of the C3 protein, which is the central node of the classical, alternative, and lectin complement pathways. This systemic reduction in C3 levels is intended to suppress complement-mediated inflammation and glomerular damage in patients with IgA Nephropathy (IgAN). Sefafaxersen is currently in Phase 3 clinical development for IgAN and has also been evaluated for other complement-driven conditions such as geographic atrophy.

Other names

sefafersen