VTX-801 is an AAV-based gene therapy for Wilson’s disease. VTX-801 contains a corrective version of the ATP7B gene, which is malfunctioning in patients affected with Wilson’s disease, and is currently under clinical evaluation in a Phase 1/2 clinical safety and efficacy study (GATEWAY) with a 12-week readout. VTX-801 received US and EU Orphan Drug Designation (ODD), and fast track designation by FDA.
VTX-PID is a technology aimed to deplete Neutralizing Antibodies (NAbs) levels for a given AAV serotype, to a level allowing proper AAV transduction and therefore desired efficacy. VTX-PID is developed both for patients presenting NAbs because of a previous infection and therefore non eligible for gene therapy; and for patients in need of gene therapy re-administration in case of loss of efficacy.
Vivet Therapeutics has investors from both sides of the ocean, bringing strong support, network and long-term commitment.
Vivet is a strong team of experienced scientists and technical experts who are passionate about innovative science and dedicated to improving patients’ lives by curing genetic disorders. We have a unique pipeline of liver-directed AAV products, as well as solid technological platforms which allow us to unlock the full potential of gene therapy.
We are constantly looking for new ideas. Today, we live in an era of extraordinary progress and innovation where we can drive science to create extraordinary opportunities for patients and their families.
Through the unique design of the GATEWAY trial, we intent to demonstrate the effectiveness of our lead asset, VTX-801, to restore copper homeostasis in Wilson disease patients. The innovative use of a biomarker will allow for a quick readout of treatment response that will trigger standard of care treatment withdrawal.
Vivet is a strong team of experienced scientists and technical experts who are passionate about innovative science and dedicated to improving patients’ lives by curing genetic disorders. We have a unique pipeline of liver-directed AAV products, as well as solid technological platforms which allow us to unlock the full potential of gene therapy.
We are constantly looking for new ideas. Today, we live in an era of extraordinary progress and innovation where we can drive science to create extraordinary opportunities for patients and their families.
Through the unique design of the GATEWAY trial, we intent to demonstrate the effectiveness of our lead asset, VTX-801, to restore copper homeostasis in Wilson disease patients. The innovative use of a biomarker will allow for a quick readout of treatment response that will trigger standard of care treatment withdrawal.
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📢 Leukodystrophy Awareness Month takes place throughout the whole month of September and is a time to raise awareness about these devastating rare conditions! Together
European Commission Grants Orphan Drug Designation for Vivet Therapeutics Gene Therapy Product for the Treatment of Cerebrotendinous Xanthomatosis (CTX) • Pre-clinical data of VTX-806 in
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🎉 25th Anniversary of the Spanish Society of Gene🧬& Cell🦠Therapy! 🎉 Join our Co-Founder & Chief Scientific Officer, Dr @Gloria Gonzalez-Aseguinolaza in celebrating this special
🤝Connect with @Sonia Valero, Head of Clinical Ops & Patient Advocacy Representative at @Vivet Therapeutics and US Patient Advocacy Consultant @Lori Mckenna, at the upcoming
Join our CEO, @Jean-Philippe Combal in the Big🍎Apple for @Chardan’s 8th Annual Genetic Medicines Conference! The premier event will bring together groundbreaking thought leaders to
