FAH lentiviral vector gene therapy

FAH lentiviral vector gene therapy is an experimental in vivo gene therapy approach being studied for the treatment of hereditary tyrosinemia type 1 (HT1). HT1 is a rare genetic disorder caused by a deficiency in the enzyme fumarylacetoacetate hydrolase (FAH), which leads to the accumulation of toxic metabolites that cause severe liver and kidney damage. This therapy utilizes a lentiviral vector to deliver a functional human FAH transgene directly into the liver, typically via percutaneous portal vein administration. The expression of the transgene is driven by a liver-specific promoter, such as the HCR-AAT (hepatic control region enhancer and alpha-1 antitrypsin promoter). In preclinical studies using a porcine model of HT1, this approach has demonstrated stable, long-term expression of the FAH enzyme and has shown potential superiority over conventional nitisinone (NTBC) therapy and ex vivo cell therapy approaches. It is currently an academic research program rather than a commercial drug product.

Other names

FAH lentiviral gene therapyLentiviral-mediated FAH gene therapy