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VTX-801 is a replication-deficient recombinant adeno-associated viral vector (rAAV) gene therapy, which is being evaluated for the treatment of wilson disease.
Lead Product(s): VTX-801
Therapeutic Area: Genetic Disease Product Name: VTX-801
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 08, 2024
Details:
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.
Lead Product(s): VTX-806
Therapeutic Area: Genetic Disease Product Name: VTX-806
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: French Government
Deal Size: $5.2 million Upfront Cash: Undisclosed
Deal Type: Funding February 01, 2024
Details:
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.
Lead Product(s): VTX-801
Therapeutic Area: Genetic Disease Product Name: VTX-801
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Pfizer Inc
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 12, 2021
Details:
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.
Lead Product(s): VTX-803
Therapeutic Area: Genetic Disease Product Name: VTX-803
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Mirum Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration April 12, 2021
Details:
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.
Lead Product(s): VTX-801
Therapeutic Area: Genetic Disease Product Name: VTX-801
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Recipient: Pfizer Inc
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement September 23, 2020
Details:
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.
Lead Product(s): VTX-803
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 01, 2020
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