In gene replacement therapy, genetic defects resulting in genetic disorders are corrected by introducing a correct copy of the defective gene into the cells of the affected tissue/organs of patients. We focus on the development of treatments for genetic disorders that can be cured by transgene expression in specific easy accessible organs such as the liver or the eye. In most cases we want to treat patients as young as possible. Therefore, re-administration with the same vector should be possible to maintain transgene expression lifelong. Since SV40 vectors are non-immunogenic in humans our SVac platform is the preferred vector for in vivo gene therapy.
Our lead gene therapy project is AMA001, aimed at restoring the serine-pyruvate aminotransferase activity (to prevent excessive oxalic acid secretion) in liver cells of patients with primary hyperoxaluria type 1 (PH1).