VTX-806

VTX-806 is a gene therapy product developed as an adeno-associated virus (AAV) vector encoding the human CYP27A1 enzyme. It is designed to restore CYP27A1 enzymatic activity, which is deficient in patients with cerebrotendinous xanthomatosis (CTX), a rare autosomal recessive neurodegenerative disorder affecting bile acid synthesis. The therapy aims to normalize toxic bile acid metabolites in blood, liver, tendons, and brain, potentially stabilizing or reversing disease progression and offering a curative treatment option beyond current standard of care that only slows or stabilizes symptoms. Preclinical studies demonstrated reduction of hepatomegaly, normalization of compensatory enzymes and bile acids, and improvement in motor function in CTX mouse models. VTX-806 has received Orphan Drug Designation from the European Commission for CTX treatment[1][2][3][4][5][6][7].

Other names

VTX 806VTX806VTX-806