VTX-803

VTX-803 is an investigational gene therapy developed for the treatment of progressive familial intrahepatic cholestasis type 3 (PFIC3), a rare genetic liver disorder. The therapy utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the MDR3 gene, aiming to correct the underlying defect in MDR3 transporter function that causes PFIC3. Preclinical studies have demonstrated that VTX-803 can normalize key serum biomarkers, reduce hepatosplenomegaly, and reverse liver fibrosis in animal models of PFIC3. The product has received Orphan Drug Designation from both the US FDA and European Medicines Agency.