Compelling additional preclinical data further support therapeutic potential of gene therapy products to treat Wilson disease (WD) and Progressive Familial Intrahepatic Cholestasis Type 3 (PFIC3).
PARIS--(BUSINESS WIRE)-- Vivet Therapeutics ("Vivet"), a privately held gene therapy biotech company dedicated to developing gene therapy treatments for inherited liver disorders with high unmet medical need, today announced that new data from its lead development program, VTX-801 a novel investigational gene therapy for WD, and VTX-803, Vivet' novel therapeutic in development for the treatment of PFIC3, have been accepted for oral presentation during the ASGCT 22nd Annual Meeting, April 29 - May 2, 2019 in Washington, D.C., USA.
Dr. Gloria González-Aseguinolaza, Vivet Therapeutics CSO & Head of the Gene Therapy Dept. at FIMA will present data from a recently completed study, validating in WD mice the use of fecal excretion of IV-injected 64copper as a pharmacodynamic endpoint in the VTX-801 Phase I/II trial currently under preparation. These data are the first demonstration of copper homeostasis restoration with 64Copper in an animal model of WD through the delivery of a single, intravenous administration of AAV.
Title: Preclinical Validation of 64Copper as a Translational Tool for Evaluating the Pharmacodynamics of VTX-801 Gene Therapy in Wilson disease (Tuesday April 30 at 3:30-3:45pm / Room: Heights Courtyard 2)
New findings from recently completed preclinical study of VTX-803 will be presented by Dr. Nicholas D. Weber, Research Scientist at Vivet. Dr Weber will present a new animal proof of concept study for VTX-803, one of Vivet's portfolio indications: "AAV Expressing MDR3 (VTX-803) Mediates the Correction of Progressive Familial Intrahepatic Cholestasis Type 3 (PFIC3) in a Clinically Relevant Mouse Model" (Tuesday April 30 at 4:15-4:30pm / Room: Heights Courtyard 1). These data expand and confirm earlier observations.
The abstracts listed above has been accepted for an Oral Presentation and can be found online at: https://www.asgct.org/global/documents/asgct19_abstracts_-final
Vivet Therapeutics will also be presenting during the Wilson Disease Association (WDA) 2019 annual conference (May 3-4 / New York, USA) and at the Wilson Aarhus 2019 symposium (May 9-12 / Aarhus, Denmark).
About Vivet Therapeutics
Vivet Therapeutics is an emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.
Vivet is building a diversified gene therapy pipeline based on novel adeno-associated virus (AAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada, University of Navarra based in Pamplona, Spain.
Vivet's lead program, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation by the Food and Drug Administration and the European Commission.
Vivet is supported by international life science investors. For more information please visit us on www.vivet-therapeutics.com and follow us on Twitter at @Vivet_tx and LinkedIn.
Source: Vivet Therapeutics
To read more Press Release articles, click here.