Dr. Gloria González-Aseguinolaza, Vivet Therapeutics Co-founder & CSO and Deputy Director of CIMA center of the Fundación para la Investigación Médica Aplicada (FIMA) will co-chair the session “Towards innovative gene therapy trials” (Wednesday October 20, 2021 5.40-7.45pm CET – Studio 1 / Plenary 3). Dr. González-Aseguinolaza will also present during the session “Advances in preclinical studies” (Wednesday October 20, 2021 11.25-1.30pm CET – Studio 1 / Plenary 2), where she will talk about Gene therapy for liver inherited diseases (12pm CET – INV22)

New findings from the recently completed preclinical studies of VTX-803 for Progressive Familial Cholestasis Type 3 (PFIC3) will be presented by Dr. Nicholas D. Weber, Research Scientist at Vivet during an oral presentation. VTX-803 was already shown to normalize key serum biomarkers, hepatosplenomegaly and liver fibrosis in two-week-old (pre-fibrotic liver), five-week-old (fibrotic liver) and 16-week old (severely fibrotic/cirrhotic livers) PFIC3 mice. Long-term studies have shown VTX-803 to be safe and the effect to be durable up to 12 months, with efficacy being comparable between 5-week-old and 16-week-old mice associated with equivalent levels of MDR3 mRNA-positive hepatocytes. These results confirm the strong potential of VTX-803 as a candidate therapy for this devastating childhood disease (Friday October 22, 2021 12.30-12.45pm CET / Parallel 7b Liver & metabolic diseases II / OR61) .

Dr. Weber will also present other animal data : “Re-Administration of AAV expressing MDR3 (VTX-803) treats progressive familial intrahepatic cholestasis type 3 (PFIC3) in juvenile Abcb4−/− mice when co-administered with ImmTOR” (P245 – LIVER & METABOLIC). A 2nd abstract will be presented by Dr. J. Martínez-García, Researcher at FIMA: “A minimal BSEP promoter allows bile acid-driven physiological regulation of transgene expression from an AAV vector” (P247 – LIVER & METABOLIC).

New findings from recently completed preclinical study of VTX-804, one of Vivet’s portfolio indications for Citrullinemia Type 1 (CTLN1), will be presented by Dr. A. Bazo, Researcher at FIMA: “rAAV-mediated gene therapy in combination with short-term nitrogen-scavenger treatment corrects biochemical and behavioral abnormalities and increases lifespan in infant Citrullinemia Type 1 (CTLN-1) mice” (P248 – LIVER & METABOLIC). These data expand earlier observations and confirm the potential of AAV based gene therapy as a promising strategy for the treatment of CTLN-1.

Finally new data from VTX-PID, Vivet’s proprietary immune platform technology, will be presented by Dr. I Ros-Gañán, Research Scientist at Vivet: Optimizing VTX-PID treatment regimen for enhanced AAV transduction in individuals with pre-existing anti-AAV neutralizing antibodies (P004 – AVV IMMUNOLOGY).