The startup develops gene therapy treatments for orphan diseases using a modified adeno-associated virus (AAV) vector to deliver a functional version of the ATP7B gene into liver cells. This approach targets inherited disorders such as Wilson disease, Progressive Familial Intrahepatic Cholestasis, and Citrullinemia Type I, providing a potential therapeutic solution for patients with these conditions.
Funding
$91.9M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Inherited metabolic disorders like Wilson disease, Progressive Familial Intrahepatic Cholestasis (PFIC), Citrullinemia Type I, and Cerebrotendinous Xanthomatosis (CTX) result from genetic mutations that disrupt essential metabolic pathways. Current treatments often involve lifelong medication, dietary restrictions, and, in severe cases, liver transplantation, which are associated with significant side effects, limited efficacy, and high costs. These approaches often fail to fully address the underlying genetic defect, leading to progressive organ damage and reduced quality of life for affected individuals.
Solution
Vivet Therapeutics develops gene therapy treatments designed to address the root cause of rare, inherited metabolic diseases. Their approach utilizes recombinant adeno-associated virus (rAAV) vectors to deliver functional copies of the deficient genes directly into liver cells, restoring normal metabolic function. This therapeutic strategy aims to provide long-lasting benefits with a single intravenous administration, reducing the need for continuous medication and invasive procedures. By correcting the underlying genetic defect, Vivet's gene therapies have the potential to prevent disease progression, alleviate symptoms, and improve patient outcomes across a range of liver-related metabolic disorders.
Target Audience
The primary target audience includes patients diagnosed with Wilson disease, PFIC2, PFIC3, Citrullinemia Type 1, and Cerebrotendinous Xanthomatosis, as well as their families and caregivers, and physicians specializing in hepatology, genetics, and metabolic disorders.
Features
- Utilizes rAAV vectors for targeted delivery of therapeutic genes to liver cells.
- Focuses on liver-directed gene therapies for inherited metabolic disorders.
- VTX-801: An AAV-based gene therapy for Wilson’s disease, currently in Phase 1/2 clinical trials (GATEWAY), delivering a corrective version of the ATP7B gene.
- VTX-802: An AAV-based gene therapy for Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2), delivering a codon-optimized version of the BSEP gene, currently under preclinical testing.
- VTX-803: An AAV-based gene therapy for Progressive Familial Intrahepatic Cholestasis Type 3 (PFIC3), delivering a codon-optimized version of the MDR3 gene, currently under preclinical testing/IND enabling studies.
- VTX-804: An AAV-based gene therapy for Citrullinemia Type 1, delivering a codon-optimized version of the ASS1 gene, currently under preclinical testing.
- VTX-806: An AAV-based gene therapy for Cerebrotendinous Xanthomatosis (CTX), delivering a codon-optimized version of the CYP27A1 gene, currently under preclinical testing.
- VTX-PSM: A non-integrative viral-based technology aimed to allow sustainability of expression in growing organs and tissues by replicating the transgene during mitosis, currently under preclinical testing.
- VTX-PID: A technology aimed to deplete Neutralizing Antibodies (NAbs) levels for a given AAV serotype, to a level allowing proper AAV transduction, currently under preclinical testing.