Vivet Therapeutics - innovative gene therapy treatments for orphan diseases

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 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 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 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.

Undisclosed.

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 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.