The trial will be conducted in patients for whom pre-existing anti-AAV antibodies are currently a limitation to gene therapy treatment.
Genethon, the Telethon laboratory, and Hansa Biopharma, a pioneer in enzyme technology for rare immunological diseases, today announced the launch of a phase 2 trial in patients suffering from Crigler-Najjar disease, a rare liver disorder, and presenting with pre-existing neutralising antibodies against AAV vectors.
The trial will evaluate the efficacy and safety of a single intravenous injection of the gene therapy developed by Genethon, after pre-treatment with imlifidase, a unique antibody-cleaving enzyme developed by Hansa.
