Revusiran (ALN-TTRSC) is a first-generation short interfering RNA (siRNA) that specifically targets transthyretin (TTR) mRNA. It is utilized in TTR-mediated amyloidosis research.
Eprodisate is a novel compound specifically developed to disrupt the interactions between amyloidogenic proteins and glycosaminoglycans, effectively impeding the polymerization of amyloid fibrils and their deposition in tissues. This compound shows potential for delaying the advancement of AA amyloidosis-related renal disease and could apply to various forms of amyloidosis.
Anselamimab(安赛埃单抗)是一种人源化IgG1κ单克隆抗体,靶向血清淀粉样蛋白A1(SAA1)。它通过特异性结合淀粉样纤维,触发巨噬细胞和中性粒细胞的吞噬作用,从而清除淀粉样沉积物。Anselamimab在临床期中作为APP抑制剂((β-amyloid A4 Protein Precursor),用于治疗轻链(AL)淀粉样变性。