SLN501 / Silence Therap, Mallinckrodt - LARVOL DELTA

Efficacy of GalNAc C3 siRNAs in factor H-deficient mice with C3 glomerulopathy. (PubMed, Mol Immunol) - "SLN501 effectively prevented liver C3 synthesis, thus limiting AP dysregulation, glomerular C3d deposits and the development of ultrastructural alterations. These data provide firm evidence of the use of siRNA-mediated liver C3 gene silencing as a potential therapy for treating C3G patients with either partial or complete FH loss of function."

Journal • Preclinical • Complement-mediated Rare Disorders • Glomerulonephritis • Nephrology • Renal Disease