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Find Clinical Drug Pipeline Developments & Deals by Vivet Therapeutics
VTX-801 is a replication-deficient recombinant adeno-associated viral vector (rAAV) gene therapy, which is being evaluated for the treatment of wilson disease.
Vivet will use the funds to further develop its gene therapy product VTX-806 as an effective treatment option to stop or reverse disease progression over the long-term, or potentially cure cerebrotendinous xanthomatosis in patients.
VTX-801 is a novel investigational gene therapy to be evaluated in a Phase 1/2 clinical trial to determine the safety, tolerability, and pharmacological activity of a single intravenous infusion in adult patients with Wilson Disease.
Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs.
Under the terms of the agreement, Pfizer will provide clinical supply for a Phase 1/2 clinical trial evaluating Vivet’s proprietary, investigational gene therapy, VTX-801, for the potential treatment of Wilson disease, a rare and potentially life-threatening liver disorder.
VTX-803 is Vivet’s second gene therapy product to be granted Orphan Drug Designation. VTX-803 is currently under IND-enabling studies and initiation of clinical development is planned for the second part of 2021 in both the US and the EU.