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.
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
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.
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