verbrinacogene setparvovec

Verbrinacogene setparvovec is an investigational, liver-directed gene therapy designed for the treatment of hemophilia B (congenital factor IX deficiency). It utilizes a synthetic, non-replicating adeno-associated virus (AAV) vector of a modified serotype (AAVS3), which encodes a codon-optimized human coagulation factor IX gene containing the gain-of-function Padua mutation (R338L). Upon intravenous administration and transduction of hepatocytes, this therapy enables endogenous production of functional factor IX protein in patients with hemophilia B. The goal is to restore normal blood clotting function and reduce bleeding episodes. The drug was originally developed by Freeline Therapeutics, later acquired by Syncona and rebranded as Spur Therapeutics[1][2][3][4][5].

Other names

verbrinacogene setparvovec