Complement factor B mRNA (CFB mRNA)

Complement factor B (CFB) is a critical zymogen in the alternative pathway of the complement system, primarily synthesized by hepatocytes in the liver. Upon activation by factor D, CFB forms the catalytic subunit of the C3 and C5 convertases, which are essential for the amplification of the complement cascade and the subsequent immune response. Dysregulation or overactivation of this pathway is a major driver in the pathogenesis of several diseases, including geographic atrophy (an advanced form of age-related macular degeneration) and various rare kidney diseases like C3 glomerulopathy. Targeting the CFB mRNA in hepatocytes using GalNAc-conjugated antisense oligonucleotides (ASOs) allows for the specific and potent reduction of CFB protein production at its primary source. This therapeutic approach aims to lower systemic CFB levels, thereby dampening the overactive alternative pathway and preventing tissue damage in complement-mediated disorders (Source: UniProt P00751; Ionis Pharmaceuticals; PubMed: 31513865).

Other names

CFBProperdin factor BC3/C5 convertaseGlycine-rich beta glycoproteinBFPBFD

Antisense oligonucleotide-mediated degradation of mRNA via RNase H1, leading to reduced hepatic synthesis and lower circulating levels of Complement factor B protein.