NE-DNA-Stab2-hFVIII

NE-DNA-Stab2-hFVIII is a synthetic, non-viral gene therapy candidate designed for the treatment of hemophilia A. It utilizes a "No-End" (NE) DNA platform, which consists of linear AAV DNA sequences devoid of viral capsid proteins. This architecture is intended to allow for repeat dosing and to bypass pre-existing neutralizing antibodies against AAV serotypes, which are common barriers to traditional AAV-mediated gene therapy. The construct features a B-domain-deleted human clotting factor VIII (hFVIII) transgene under the transcriptional control of the Stabilin 2 (Stab2) promoter. The Stab2 promoter specifically targets expression to liver sinusoidal endothelial cells (LSECs), the natural site of FVIII production and secretion. The vector is formulated within liver-targeted lipid nanoparticles (LNPs) or synthetic liposomes for delivery. It is being developed by researchers at the University of Florida and the Università degli Studi del Piemonte Orientale.

Other names

No-End DNA Stabilin 2 human Factor VIII