LV-FAH

**LV-FAH** is a lentiviral vector-based gene therapy designed to treat hereditary tyrosinemia type 1 (HT1), a severe metabolic liver disease caused by deficiency of the fumarylacetoacetate hydrolase (FAH) enzyme. It delivers a functional copy of the human **FAH** gene to hepatocytes via portal vein administration, enabling stable, long-term FAH expression and complete repopulation of the diseased liver by corrected cells in preclinical pig models. Preclinical studies demonstrate curative efficacy, with NTBC-independent survival, normalized liver function (e.g., reduced bilirubin, ammonia, tyrosine), absence of fibrosis or tumors, and a benign genomic integration profile favoring safe hepatocyte selection and expansion. Castle Creek Biosciences (formerly via Novavita Thera acquisition) is advancing it toward clinical development with plans for an IND submission.[2][3][4][5][9]

Other names

Lentiviral Vector Expressing Human Fumarylacetoacetate Hydrolase