Paris, France, October 23, 2023 (GLOBE NEWSWIRE) – Vivet Therapeutics (“Vivet”), a clinical- stage biotechnology company developing novel gene therapies for rare, inherited metabolic conditions, announced its participation and presentations at the 30th European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress. The congress will be held from October 24 – 27, 2023, in Brussels, Belgium.
Dr. Nicholas Weber, Principal Scientist and Lead for New Programs and Innovations at Vivet, will present in an invited talk about adeno-associated virus (AAV) therapies targeting inherited cholestasis.
Gloria Gonzalez-Aseguinolaza, Vivet Therapeutics Chief Scientific Officer, will also present new preclinical results related to its lead program VTX-801, which utilizes the proprietary recombinant AAV (rAAV) technology to target Wilson disease.
The presentation and poster details are listed below; the full program is available on the ESGCT website.
Session 3c: Gene therapy for metabolic diseases at preclinical stage
Date/Time: Wednesday 25 Oct at 8:30.
Title: INV21 – Advances and pitfalls in AAV therapy targeting inherited cholestasis
Presenter: Nicholas Weber, Ph.D , Principal Scientist, Vivet Therapeutics
Room: Maison de la Poste, PARALLEL
Poster Number: P035
Category: AAV & non integrative vectors
Poster Title: Improvement of progressive familial intrahepatic cholestasis type 2 via AAV- mediated hepatic BSEP expression in mice
Presenter: Angie Molina, Ph.D. (Fundación para la Investigación Medica Aplicada in partnership with Vivet Therapeutics)
Poster Number: P585 Category: Metabolic Diseases
Poster Title: Therapeutic efficacy of VTX-801 Wilson’s disease mice at an advanced stage the disease
Presenter: Gloria Gonzalez-Aseguinolaza, Ph.D ,Chief Scientific Officer, Vivet Therapeutics
Number: P567
Category: Metabolic Diseases
Poster Title: Therapeutic Efficacy of the Combination of Long-Term Penicillamine Treatment with a Single Administration of VTX-801 in Wilson’s Disease Mice
Presenter: Gloria Gonzalez-Aseguinolaza, Ph.D, Chief Scientific Officer, Vivet Therapeutics
About Vivet Therapeutics
Vivet Therapeutics is a biotechnology company developing transformative and novel gene therapy treatments for rare, inherited metabolic diseases.
Vivet’s lead program is VTX-801, a novel investigational gene therapy being evaluated in the GATEWAY clinical trial, a Phase I/II clinical trial to determine the safety, viability, and pharmacological activity of a single intravenous infusion in adults with Wilson disease. Wilson disease is a rare genetic disorder caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurologic symptoms and potentially death. VTX-801 has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) and the European Commission (EC). It has also received Fast Track Designation by the FDA.
Vivet is building a diversified gene therapy pipeline based on novel rAAV (recombinant AAV) with five preclinical programs and new technologies addressing key challenges of AAV-based gene therapy related to the sustainability of expression in young patients and immunomodulation for potential retreatment. Vivet develops these technologies through its partnership 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 (CIMA), University of Navarra based in Pamplona, Spain.
Visit www.vivet-therapeutics.com for more information. If you would like to meet with the Vivet team during the congress, or at any other time, please contact: info@vivet- therapeutics.com.
For more information on the ongoing VTX-801 GATEWAY clinical trial, visit https://www.gatewaytrialwilsondisease.com/.