VTX-802

VTX-802 is an investigational gene therapy designed for the treatment of Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2), a rare genetic liver disorder. The therapy uses an adeno-associated virus (AAV) vector to deliver a codon-optimized version of the BSEP (bile salt export pump, encoded by the ABCB11 gene) specifically to liver cells. By restoring functional BSEP expression in hepatocytes, VTX-802 aims to address the underlying cause of PFIC2 and improve bile flow, potentially offering a disease-modifying approach for affected patients. The drug is currently in preclinical development and has shown efficacy in animal models[1][3][8]. Vivet Therapeutics initially developed VTX-802, and Mirum Pharmaceuticals holds development and commercialization rights[7].