Imlifidase as pretreatment in gene therapy for patients with Crigler–Najjar syndrome who are immune to AAV On October 10, 2025, Genethon, a worldwide pioneer and leader in research and development of gene therapy for rare genetic diseases, and Hansa Biopharma, a Sweden-based leader in IgG cleaving enzyme technology announced that…..
Efficacy of gene therapy demonstrated in patients suffering from Crigler-Najjar Syndrome Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the liver enzyme UGT1A1, can cause significant neurological damage and become fatal. At the current time, the only…..