VTX-803 / Vivet Therapeutics - LARVOL DELTA

Effective Readministration of AAV Gene Therapy Prevents Tumorigenesis in Infant PFIC3 Mice, a Mouse Model Prone to Hepatocellular Carcinoma (ASGCT 2023) - "We have previously demonstrated successful therapeutic reversal of disease manifestations in a mouse model of PFIC3 with a single administration of VTX-803(8), a recombinant AAV8 carrying a codon optimized version of the human ABCB4 gene... These outcomes support AAV-mediated gene therapy as a highly efficient and safe modality for a curative approach to inborn errors of metabolism including in very young patients. It also provides evidence countering the strong-held belief that AAV treatment is liable to increase the risk of hepatocellular carcinoma particularly when given to pediatrics or in patients with liver damage, which is an essential evaluation for the safety determination of this powerful therapy."

Gene therapy • Preclinical • Cholestasis • Fibrosis • Gastroenterology • Gastrointestinal Cancer • Gene Therapies • Hepatocellular Cancer • Hepatology • Immunology • Infectious Disease • Liver Cirrhosis • Metabolic Disorders • Oncology • Pediatrics • Solid Tumor • ABCB4