VTX-804

VTX-804 is an adeno-associated virus (AAV)-based gene therapy developed for the treatment of citrullinemia type 1, a rare inherited metabolic disorder. The therapy delivers a codon-optimized version of the *ASS1* gene to the liver, aiming to restore normal ureagenesis and correct biochemical and behavioral abnormalities associated with citrullinemia type 1. Preclinical studies in animal models have shown that a single intravenous dose of VTX-804 can normalize lifespan and correct disease-related biomarkers and behaviors up to six months post-administration, especially when combined with standard-of-care nitrogen scavenger pretreatment[5][6][8]. The drug is currently in preclinical development.