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verbrinacogene setparvovec
- Development stage
- Discontinued
- Lead developer
- Spur Therapeutics
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
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
02
Targets
F9 (Coagulation Factor IX)
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