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PIPELINE
VTX-801
PHASE 3
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).
VTX-802
POC
VTX-802 is an AAV-based gene therapy for patients with Progressive Familial Intrahepatic Cholestasis Type 2. It delivers a codon-optimized version of the BSEP gene to the liver and is currently under preclinical testing.
VTX-803
PHASE 1/2
VTX-803 is an AAV-based gene therapy for patients with Progressive Familial Intrahepatic Cholestasis Type 3. It delivers a codon-optimized version of the MDR3 gene to the liver and is currently under preclinical testing/IND enabling studies. VTX-803 received US and EU Orphan Drug Designation (ODD).
VTX-804
POC
VTX-804 is an AAV-based gene therapy for patients affected by Citrullinemia Type 1. It delivers a codon-optimized version of the ASS1 gene to the liver and is currently under preclinical testing.
VTX-805
RESEARCH
Undisclosed.
VTX-PSM
POC
VTX-PSM is a non-integrative viral-based technology aimed to allow sustainability of expression in growing organs and tissues. It allows, without integration into the genome, to replicate the transgene during the mitosis. It is currently under preclinical testing.
VTX-PID
IND ENABLING
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.
Strong and Committed Investors
Vivet Therapeutics has investors from both sides of the ocean, bringing strong support, network and long-term commitment.
Building a unique company for unique treatmentsVivet 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.
Science for new pathsWe 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.
Developing Breakthrough MedicinesThrough 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.
https://genetherapy-conference.com/
“2022 has undoubtedly been a landmark year for the space with much-needed approvals in the summer marking the first time we have seen multiple approvals in a calendar year from the FDA. Gene Therapy for Rare Disorders 2023 will benchmark these breakthroughs, leverage strategic insights from recent success stories, and bring in-depth insights from experts”
During the “Defining Value of Rare Gene Therapies” session, Vivet Therapeutic’s CEO & Co-Founder, Jean-Philippe Combal, will take part in the following panel discussion: “Navigating a Competitive Market & Establishing a Differentiated Product” (March 21, 2pm EST).
Topics to be discussed in this panel discussion will include:
👉 Exploring precompetitive collaboration to support progress to approval
👉 Navigating landscapes where multiple gene therapies are being developed for a specific disease
👉 Understanding value demonstration where standard of care exists and use of RWD to better understand disease severity
➡️ https://www.terrapinn.com/conference/pharma-pricing/agenda.stm
Thomas Daniel-Robin, Vivet Therapeutic’s BD Director, will take part in the HTA session and give the following presentation: “Healthcare databases versus current practice – gaps in information for patients, payers and physicians in Wilson Disease” (March 21, 4.25-5.30pm CET).
Topics to be discussed on the potential of national healthcare databases will include:
👉 Generating Real World Data for Rare Diseases: large cohorts and long-term follow-up
👉 Identifying data gaps between Real World Data and current practice: what can and cannot be captured by physicians and patients
👉 Finding the unexpected and understanding the value of enriched studies
Anne Douar, Vivet Therapeutics Chief Development Officer, will take part in the panel «Developing gene therapies for rare disease patients » on November 15 at 2 pm. She will also be a panel speaker on “Key considerations on cell & gene therapy manufacturing for commercialization” on November 17 at 10:10 am
Jean-Philippe Combal, Vivet Therapeutics Co-Founder and CEO, will participate in the panel “Designing first in human gene therapy clinical trials”, discussing gene therapy platform approaches for potential re-dosing strategy on November 15 at 3:10 pm
Contact Us
VIVET THERAPEUTICS
- 80 Boulevard Haussmann
75008 Paris, FRANCE - Av. de Pío XII, 55
31008 Pamplona Spain - info@vivet-therapeutics.com
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