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Drug intelligence / Profile preview
LV-Stab2-FVIII
- Development stage
- Preclinical
- Lead developer
- Università del Piemonte Orientale
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
LV-Stab2-FVIII is an investigational lentiviral vector-based gene therapy designed for the treatment of hemophilia A. Developed by researchers at the Università del Piemonte Orientale, the therapy delivers a transgene encoding coagulation factor VIII (FVIII) under the transcriptional control of the liver sinusoidal endothelial cell (LSEC)-specific stabilin-2 (STAB2) promoter. By restricting FVIII expression to LSECs, the therapy aims to achieve stable, long-term therapeutic levels of FVIII while promoting immune tolerance and preventing the formation of anti-FVIII neutralizing antibodies (inhibitors). Preclinical studies in hemophilia A mouse models have demonstrated that LV-Stab2-FVIII can restore FVIII activity, stabilize cerebral microvasculature, and rescue bone structure alterations.
- Other names
- LV-Stab2-FVIII-HALV-Stab-2-FVIII-HALV-Stab 2-FVIII-HALV.STAB2-FVIII-HA
02
Targets
F8 (Coagulation Factor VIIIa)STAB2 (Stabilin-2)BB-031 (Von Willebrand factor)
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