LV-FVt

LV-FVt is an experimental, preclinical-stage third-generation lentiviral gene therapy vector designed for the treatment of congenital factor V (FV) deficiency. Developed by a collaborative Spanish research consortium including the Universidad Complutense de Madrid and funded by the Association for Research and Cure of Factor V Deficiency (ASDEFAV), the vector delivers a truncated factor V transgene (FVt) lacking the B domain (F5 cDNA). In preclinical in vitro and in vivo models, LV-FVt has demonstrated the ability to restore factor V functional activity, reduce prothrombin time (PT) and activated partial thromboplastin time (aPTT), and integrate into hepatic tissue, offering a potential therapeutic option for this ultra-rare autosomal recessive bleeding disorder.