AAV8-HLP-FVIII-V3

AAV8-HLP-FVIII-V3 is an experimental gene therapy candidate designed for the treatment of Hemophilia A. It utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional human Factor VIII (FVIII) gene specifically to hepatocytes. The construct features a synthetic hybrid liver-specific promoter (HLP) designed to maximize expression while minimizing the size of the expression cassette to fit within the limited packaging capacity of AAV vectors. The transgene, designated FVIII-V3, is a B-domain deleted version of human Factor VIII that has been codon-optimized and modified with a specific linker to enhance secretion efficiency and stability. Developed through a collaboration between University College London and Freeline Therapeutics, the therapy aimed to provide sustained, endogenous production of Factor VIII to eliminate the need for regular factor replacement therapy. However, development was deprioritized by Freeline Therapeutics in favor of other pipeline assets.

Other names

AAV8-HLP-hFVIII-V3AAV-8-HLP-hFVIII-V3AAV 8-HLP-hFVIII-V3