Drug Profile

VTX 804

Alternative Names: VTX-804

Latest Information Update: 28 Jul 2024

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At a glance

Originator Vivet Therapeutics
Developer CIMA- Universidad de Navarra; Vivet Therapeutics
Class Gene therapies
Mechanism of Action Argininosuccinate synthase replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

28 Jul 2024 No recent reports of development identified for preclinical development in Citrullinemia in France (IV)
28 Jul 2024 No recent reports of development identified for preclinical development in Citrullinemia in Spain (IV)
16 Jun 2020 Preclinical trials in Citrullinemia in France (Parenteral), prior to June 2020 (Vivet Therapeutics pipeline, June 2020)

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