Vivet Therapeutics is a clinical-stage biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.

Vivet’s lead program, VTX-801, is currently under clinical development for Wilson Disease (ongoing GATEWAY – Phase 1/2 Clinical Trial of VTX-801).

VTX-801 is a novel investigational gene therapy for Wilson Disease, which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC) and Fast Track designation by the FDA. Wilson Disease, a rare genetic disorder, is 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.

Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus (rAAV). Vivet is also working on technological platforms addressing key challenges of gene therapy, such as sustained therapeutic gene expression and the treatment of patients with pre-existing immunity to the viral vector.

Video: Educational material from American Society of Gene and Cell Therapy (ASGCT)