08/30/2022
Hereditary tyrosinemia type 1 (HT1) is a rare inborn error of metabolism of the liver, affecting 1 in 100,000 live births. At Castle Creek, we are developing an in vivo gene therapy program to address this genetic disorder.
Recently published research in Nature Communications found that our lead in vivo gene therapy candidate resolved biochemical and hepatic histological markers of HT1 in a large animal model of the disease. The study was conducted by Mayo Clinic researchers.
Read more here:
The cell and gene therapy company’s lead in vivo candidate uses lentiviral vector delivery to correct metabolic disorder in large animal model of hereditary tyrosinemia type-1