VTX-802 / Vivet Therapeutics - LARVOL DELTA

Liver-directed gene therapy results in amelioration of progressive familial intrahepatic cholestasis type 2 in mice. (PubMed, Hepatol Commun) - "VTX-802 restores hepatic BSEP expression and partially corrects disease phenotype in PFIC2 mice. To our knowledge, VTX-802 is the first gene therapy approach that could potentially benefit PFIC2 patients."

Journal • Preclinical • Cholestasis • Dermatology • Fibrosis • Gene Therapies • Hepatology • Immunology • Pruritus • Rare Diseases • Transplantation • ABCB1

Improvement of Progressive Familial Intrahepatic Cholestasis Type 2 Via AAV-Mediated Hepatic BSEP Expression in Mice (ASGCT 2025) - "After developing VTX-802(8), an AAV8 vector containing a codon-optimized human ABCB11 sequence controlled by a liver-specific promoter, a dose range finding study of the therapeutic vector was performed...Developing a long-lasting cure for PFIC2 by restoring long-term hepatic BSEP expression and physiological bile secretion would provide a transformative option for these pediatric patients with significant unmet medical needs. Disease Focus of Abstract:Inborn Errors in Metabolism"

Preclinical • Cholestasis • Dermatology • Fibrosis • Gene Therapies • Hepatocellular Cancer • Hepatology • Immunology • Oncology • Pediatrics • Pruritus • Solid Tumor • ABCB1

Improvement of Progressive Familiar Intrahepatic Cholestasis Type 2 via AAV-Mediated Hepatic BSEP Expression in Mice (ASGCT 2023) - "We next assessed the therapeutic efficacy of gene therapy in this PFIC2 mouse model using a liver-tropic AAV8 vector carrying a codon-optimized human BSEP cDNA under the control of a liver-specific promoter (VTX-802(8))...However, further development of the AAV vector and/or combination with other pharmacological products, might be investigated to enhance treatment efficacy and further revert the disease biomarkers. The development of a durable cure for PFIC2 and its translation to the clinic would provide a life-changing alternative for these pediatric patients with high unmet medical need."

Preclinical • Cholestasis • Dermatology • Fibrosis • Gastrointestinal Cancer • Gene Therapies • Hepatocellular Cancer • Hepatology • Immunology • Oncology • Pediatrics • Pruritus • Solid Tumor • Transplantation • ABCB1